oversight

Obesity Drugs: Few Adults Used Prescription Drugs for Weight Loss and Insurance Coverage Varied

Published by the Government Accountability Office on 2019-08-09.

Below is a raw (and likely hideous) rendition of the original report. (PDF)

              United States Government Accountability Office
              Report to Congressional Committees




              OBESITY DRUGS
August 2019




              Few Adults Used
              Prescription Drugs for
              Weight Loss and
              Insurance Coverage
              Varied




GAO-19-577
                                              August 2019

                                              OBESITY DRUGS
                                              Few Adults Used Prescription Drugs for Weight Loss
                                              and Insurance Coverage Varied
Highlights of GAO-19-577, a report to
congressional committees




Why GAO Did This Study                        What GAO Found
Obesity has been associated with an           The prevalence of obesity—that is, body weight higher than what is considered a
increased risk of developing conditions       healthy weight for a given height—was about 38 percent among all U.S. adults,
such as heart disease, stroke, diabetes,      according to the latest available national estimates at the time of GAO’s analysis.
and certain types of cancer. Treatment        This prevalence was similar for adults with different types of health insurance.
options for individuals with obesity
include lifestyle therapy, such as diet,      Prevalence of Obesity among U.S. Adults by Health Insurance Type, 2013–2016
exercise, and behavioral counseling;
obesity drugs; surgery; or a combination
of these efforts. The Bipartisan Budget
Act of 2018 (P.L. 115-123) included a
provision for GAO to review the
prevalence of obesity and the use and
insurance coverage of obesity drugs.
This report examines the prevalence of
obesity in the United States, and what is
known about the use and health
insurance coverage of obesity drugs,
among other objectives.
GAO examined data from agencies
within the Department of Health and
Human Services (HHS) on the
prevalence of obesity (using estimates
for 2013 through 2016) and the use,
spending, and coverage of obesity
drugs; conducted a literature review of
relevant studies published from January
2012 through January 2019 in peer-
reviewed and other publications;              Treatment for adults with obesity may include one or more of nine prescription
reviewed drug formularies for selected        drugs that the Food and Drug Administration has approved for weight
health plans; and reviewed documents          management (i.e., obesity drugs), though relatively few adults have used these
and interviewed officials from federal        drugs. Of an estimated 71.6 million U.S. adults with obesity, an estimated
agencies and stakeholder organizations        660,000 per year, on average, used an obesity drug from 2012 through 2016,
(including medical associations,              according to national estimates. Among adults who reported trying to lose weight,
advocacy groups, pharmacy benefit             about 3 percent reported that they took prescription medication for weight loss
managers, and insurers).                      from 2013 through 2016, according to national estimates.
HHS provided technical comments on a
                                              Coverage of obesity drugs varied across different types of health insurance,
draft of this report, which were
                                              including Medicare and Medicaid. Plans cited factors such as low consumer
incorporated as appropriate.
                                              demand and strong evidence supporting other treatments in their coverage
                                              decisions. GAO’s analysis of Centers for Medicare & Medicaid Services’ data
                                              indicates that some Medicare prescription drug plans and state Medicaid
                                              programs reimbursed for some obesity drugs in 2016 and 2017. Coverage for
                                              private health insurance plans also varied, and plans may require the patient to
                                              obtain prior authorization for the drugs to be covered, according to officials from
                                              insurers and pharmacy benefit managers GAO interviewed. For example,
                                              officials from one insurer said that some of their plans only cover obesity drugs
View GAO-19-577. For more information,
contact John E. Dicken at (202) 512-7114 or   after a patient has tried other treatment options such as behavioral counseling.
dickenj@gao.gov.
                                              ______________________________________ United States Government Accountability Office
Contents


Letter                                                                                 1
               Background                                                              6
               The Prevalence of Obesity Was Close to 40 Percent among All
                 U.S. Adults from 2013 through 2016                                    8
               Few Adults Used Obesity Drugs and Limited Data Are Available
                 on Individuals Who Have Used These Drugs                            10
               Health Insurance Coverage for Obesity Drugs Is Limited and
                 Varied across Types of Insurance                                    19
               Two-Thirds of Obesity Drug Payments Were Made Out of Pocket;
                 Adults Who Used Obesity Drugs Had Higher Average
                 Estimated Medical Spending                                          26
               Agency Comments                                                       30

Appendix I     Objectives, Scope, and Methodology                                    32



Appendix II    List of FDA-Approved Prescription Obesity Drugs                       41



Appendix III   Prevalence of Obesity and Overweight among U.S. Adults                42



Appendix IV    List of Selected Studies Reviewed                                     46



Appendix V     Estimates of New Adult Users of Obesity Drugs, 2008-2017              49



Appendix VI    Reimbursement for Obesity Drugs in Medicare Part D Enhanced
               Alternative Coverage, 2016 and 2017                                   51



Appendix VII   Reimbursement for Obesity Drugs in Medicaid, 2016 and 2017            54




               Page i                                            GAO-19-577 Obesity Drugs
Appendix VIII   Estimates of Medical and Prescription Drug Expenditures for Adults
                Who Used and Did Not Use Obesity Drugs                                 59



Appendix IX     GAO Contact and Staff Acknowledgments                                  63


Tables
                Table 1: Prevalence of Obesity in Adults by Age and Class of
                        Obesity, 2013–2016                                             10
                Table 2: Number of Medicare Part D Plans and Beneficiaries
                        Reimbursed for Obesity Drugs, 2016 and 2017                    21
                Table 3: Numbers of States and Prescriptions with Medicaid
                        Reimbursement for Obesity Drugs, 2016 and 2017                 22
                Table 4: FDA-Approved Prescription Obesity Drugs, as of June
                        2019                                                           41
                Table 5: Prevalence of Obesity among U.S. Adults, 2013–2016,
                        by Age and Class of Obesity                                    43
                Table 6: Prevalence of Obesity among U.S. Adults, 2013–2016,
                        by Insurance Coverage and Class of Obesity                     43
                Table 7: Prevalence of Overweight among U.S. Adults, 2013–
                        2016, by Age and Insurance Coverage                            45
                Table 8: List of Selected Studies Pertaining to Our Research
                        Objectives, by Topic Area                                      46
                Table 9: Duration of Use for New Adult Users of Obesity Drugs,
                        2008-2017                                                      50
                Table 10: New Adult Users of Obesity Drugs by Age and Gender,
                        2008-2017                                                      50
                Table 11: Medicare Part D Number of Claims, Unique Plans,
                        Unique Beneficiaries, Plan Spending, and Beneficiary
                        Liability for the Nine Obesity Drugs, 2016                     52
                Table 12: Medicare Part D Number of Claims, Unique Plans,
                        Unique Beneficiaries, Plan Spending, and Beneficiary
                        Liability for the Nine Obesity Drugs, 2017                     53
                Table 13: Medicaid Amount Reimbursed and Number of
                        Prescriptions for Obesity Drugs in Each State, 2016            54
                Table 14: Medicaid Amount Reimbursed and Total Number of
                        Prescriptions for Obesity Drugs in Each State, 2017            56
                Table 15: Medicaid Amount Reimbursed and Total Number of
                        Prescriptions for Each of the Nine Obesity Drugs, 2016         57




                Page ii                                            GAO-19-577 Obesity Drugs
          Table 16: Medicaid Amount Reimbursed and Total Number of
                  Prescriptions for Each of the Nine Obesity Drugs, 2017                            58
          Table 17: Estimates of Average Annual Expenditures per Adult for
                  All Prescription Drugs, 2012–2016                                                 60
          Table 18: Estimates of Average Annual Medical Expenditures per
                  Adult, 2012 – 2016                                                                61
          Table 19: Estimates of Average Annual and Median Expenditures
                  per Adult for All Obesity Drugs Purchased, 2012–2016                              62

Figures
          Figure 1: Prevalence of Obesity among U.S. Adults by Insurance
                   Type and Class of Obesity, 2013–2016                                              9
          Figure 2: Estimated Average Annual Estimates of Distribution of
                   Payments for Obesity Drugs by Insurance Type, 2012–
                   2016                                                                             27




          Abbreviations

          AHRQ              Agency for Healthcare Research and Quality
          BMI               body mass index
          CDC               Centers for Disease Control and Prevention
          CMS               Centers for Medicare & Medicaid Services
          HHS               Department of Health and Human Services
          FEHBP             Federal Employees Health Benefits Program
          FDA               Food and Drug Administration
          MEPS              Medical Expenditure Panel Survey
          NHANES            National Health and Nutrition Examination Survey


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          Page iii                                                        GAO-19-577 Obesity Drugs
                       Letter




441 G St. N.W.
Washington, DC 20548




                       August 9, 2019

                       Congressional Committees

                       Obesity—that is, body weight higher than what is considered a healthy
                       weight for a given height—has been associated with an increased risk of
                       developing heart disease, stroke, type 2 diabetes, certain types of cancer,
                       and other conditions. It is also associated with an increased risk for death,
                       particularly among adults younger than 65 years old. 1 Obesity and its
                       associated health problems also have a significant economic effect on the
                       U.S. health care system. The medical spending on obesity for adults in
                       the United States was estimated to be about $342 billion in 2013, and the
                       share of health care spending to treat obesity-related illness rose from
                       about 21 percent in 2005 to about 28 percent in 2013, according to a
                       2017 study. 2

                       In 2013, the American Medical Association classified obesity as a disease
                       that requires a range of interventions for its treatment and prevention.
                       Obesity is a complex health issue to address and results from a
                       combination of causes and contributing factors, including individual
                       factors such as behavior and genetics, according to the Centers for
                       Disease Control and Prevention (CDC). Treatment options for individuals
                       with obesity include lifestyle therapy (such as diet, physical activity, and
                       behavioral counseling), bariatric surgery, prescription weight loss
                       medications (i.e., obesity drugs), or a combination of these. 3 As of June
                       2019, there were nine prescription obesity drugs approved by the Food
                       and Drug Administration (FDA); four of them are approved for short-term
                       use and five are approved for long-term use.



                       1
                        U.S. Preventive Services Task Force, “Behavioral Weight Loss Interventions to Prevent
                       Obesity-Related Morbidity and Mortality in Adults: U.S. Preventive Services Task Force
                       Recommendation Statement,” JAMA, vol. 320, no. 11 (2018): 1163–1171.
                       2
                        A. Biener, J. Cawley, and C. Meyerhoefer, “The High and Rising Costs of Obesity to the
                       U.S. Health System,” The Journal of General Internal Medicine, vol. 32 (Suppl 1), (2017):
                       S6-S8.
                       3
                        Bariatric surgery includes a variety of procedures to help individuals lose weight by
                       reducing the size of the stomach and/or rearranging the small intestine. This can help
                       individuals feel fuller sooner and can also lead to metabolic changes that cause weight
                       loss.




                       Page 1                                                          GAO-19-577 Obesity Drugs
The Bipartisan Budget Act of 2018 included a provision for us to review
the prevalence of obesity and the use of obesity drugs, including
spending for and insurance coverage of these drugs. 4 This report
examines

1. the prevalence of obesity among adults in the United States;
2. what is known about the use of obesity drugs and the individuals who
   use them;
3. what is known about health insurance coverage of obesity drugs; and
4. what is known about spending on obesity drugs and about medical
   spending for adults who used obesity drugs compared to those who
   did not.

To examine the prevalence of obesity among adults in the United States,
we examined nationally representative estimates calculated by CDC
using data from the National Health and Nutrition Examination Survey
(NHANES), which uses physical examinations of participants to measure
height and weight to calculate body mass index (BMI). 5 CDC analyzed
NHANES data from 2013 through 2016 to estimate the prevalence of
obesity for all adults by age and health insurance coverage.

To examine what is known about the use of obesity drugs and the
individuals who use them, we conducted a literature review, interviewed
knowledgeable stakeholders, and examined federal agency data:

•   Literature review. We identified relevant peer-reviewed studies
    published from January 2012 through January 2019 through a search
    of bibliographic databases, including ProQuest, Scopus, MEDLINE,
    and International Pharmaceutical Abstracts, using terms such as
    “obesity,” “weight loss,” and “prescriptions.” Of the 765 study citations
    we identified, we reviewed 220 full studies; of those, we determined
    there were 19 relevant studies, which we examined for information
    related to the use of obesity drugs and individuals who use them. We
    also examined available information on the clinical trials conducted to
    obtain FDA’s approval of the prescription obesity drugs for the U.S.


4
Pub. L. No. 115-123, div. E, tit. III, § 50352, 132 Stat. 64, 212.
5
 BMI is a person’s weight in kilograms divided by the square of their height in meters, and
is used as a screening tool for obesity.




Page 2                                                               GAO-19-577 Obesity Drugs
    market. These were either included in our literature review or, for
    publications prior to 2012, we obtained a copy of the study.
•   Stakeholder interviews. We obtained information from officials from
    eight organizations—three medical associations and five advocacy
    groups for obesity research and treatment. 6 We reviewed information
    and studies obtained from these organizations on the use of obesity
    drugs, including any guidelines for using obesity drugs, and also
    obtained their perspectives on what physicians and other health care
    providers take into consideration when prescribing these drugs,
    among other things.
•   Federal agency data. We reviewed data and documents, and
    interviewed officials from federal agencies within the Department of
    Health and Human Services (HHS), including CDC, FDA, the Agency
    for Health Care Research and Quality (AHRQ), the Centers for
    Medicare & Medicaid Services (CMS), and the National Institutes of
    Health. Data and documents we reviewed included AHRQ’s nationally
    representative estimates of the use of obesity drugs from Medical
    Expenditure Panel Survey (MEPS) data from 2012 through 2016;
    CDC’s estimates of adults who reported that they took prescription
    medications for weight loss from NHANES data from 2013 through
    2016; and FDA’s analysis of dispensings of the nine prescription
    obesity drugs using 2008 through 2017 data from the agency’s
    Sentinel System. 7 These data were the most recently available data at
    the time of our review.

To examine what is known about the health insurance coverage of
obesity drugs, we examined relevant laws and regulations and obtained

6
 The stakeholder organizations we contacted were the American Academy of Family
Physicians, American Association of Clinical Endocrinologists, American College of
Cardiology, American Heart Association, Obesity Action Coalition, Obesity Medicine
Association, The Obesity Society, and the Strategies to Overcome and Prevent (STOP)
Obesity Alliance. These stakeholders were selected because of their medical or scientific
expertise, relevant publications, or familiarity with the treatment of obesity and obesity
drugs.
7
 MEPS collects nationally representative data on health care use, expenditures (i.e.,
spending), sources of payment, and insurance coverage for the U.S. civilian,
noninstitutionalized population.

Dispensing is the act of delivering a prescription drug to a patient or an intermediary who
is responsible for administering the drug. FDA’s analysis of its Sentinel System included
data from populations with federal or commercial health insurance from 17 of 18 FDA data
partners, including large insurers and Medicare.




Page 3                                                          GAO-19-577 Obesity Drugs
information and policy documents from federal agencies, including from
CMS (for Medicare and Medicaid) and the Office of Personnel
Management [for the Federal Employees Health Benefits Program
(FEHBP)]. 8 For information on the number of claims for obesity drugs that
were reimbursed and the number of plans that reimbursed these claims
under the Medicare prescription drug program, known as Medicare Part
D, we analyzed Prescription Drug Event data from CMS for 2016 and
2017. 9 For information on the number of state Medicaid programs that
reimbursed claims for obesity drugs and the number of claims for obesity
drugs that they reimbursed, we analyzed CMS’s Medicaid State Drug
Utilization data for 2016 and 2017. 10 For information on FEHBP guidance
on coverage of obesity drugs as of May 2019, we examined documents
and information from the Office of Personnel Management. In addition,
we identified one study in our literature review relevant to insurance
coverage of obesity drugs, and we obtained information from officials
from the three largest pharmacy benefit managers and from four large
insurers, as well as from two organizations knowledgeable about
prescription drug benefits for employer-sponsored health plans. 11 We also
reviewed drug formularies for selected private health insurance plans to
determine if any of the prescription obesity drugs were included. 12

To examine what is known about spending on obesity drugs and about
medical spending for adults who used obesity drugs compared to those
who did not, we examined the latest available AHRQ estimates, which are

8
 The Office of Personnel Management administers FEHBP—the largest employer-
sponsored health insurance program in the United States—providing health insurance
coverage to about 8 million federal employees, retirees, and their dependents in 2016
through contracts with private health insurance plans.
9
 Medicare Part D provides a voluntary outpatient prescription drug benefit for Medicare
beneficiaries. In 2018, there were about 44 million enrollees covered by a Medicare Part D
plan. CMS’s Prescription Drug Event data contains cost and payment data submitted by
plans to CMS for covered Part D drugs to enable CMS to make payments to plans and
administer the Part D benefit.
10
 As of March 2019, there were 65.9 million enrollees covered by Medicaid.
11
  Pharmacy benefit managers administer prescription drug programs for health plans,
including employer-sponsored health plans and Medicare Part D plans.
12
  A formulary is a list of prescription drugs covered by a prescription drug plan or another
insurance plan offering prescription drug benefits. A plan may cover drugs that are not
included on the formulary if, for example, a patient has previously used other prescription
drugs or therapies and the health care provider determines the prescription drug not
included in the formulary is medically necessary for the patient.




Page 4                                                            GAO-19-577 Obesity Drugs
based on 2012 through 2016 MEPS data on payments for obesity drugs
and medical spending, and FDA’s nationally projected estimates on the
prescriptions dispensed for obesity drugs calculated using data from the
2017 IQVIA™ National Prescription Audit and IQVIA™ Total Patient
Tracker. 13 In addition, for the amounts spent on obesity drugs by
Medicare Part D plans and Medicaid, we analyzed CMS’s Prescription
Drug Event data and Medicaid State Drug Utilization data, respectively,
for 2016 and 2017. 14 We also reviewed studies that we identified in our
literature review or obtained from stakeholders.

To determine the reliability of the data we used for all four objectives, we
reviewed documentation on data collection processes and discussed
limitations of the data with the relevant federal agency officials. In
addition, we conducted data reliability checks on the data, when
appropriate. We determined the data used in this report were sufficiently
reliable for our purposes. See appendix I for additional information on our
scope and methodology.

We conducted this performance audit from April 2018 to August 2019 in
accordance with generally accepted government auditing standards.
Those standards require that we plan and perform the audit to obtain
sufficient, appropriate evidence to provide a reasonable basis for our
findings and conclusions based on our audit objectives. We believe that
the evidence obtained provides a reasonable basis for our findings and
conclusions based on our audit objectives.




13
  IQVIA™ is proprietary data that includes data for prescriptions dispensed at
approximately 59,900 U.S. outpatient retail pharmacies. FDA analyzed these data and
provided us with aggregated results for obesity drugs.
14
 This analysis did not include some reimbursement amounts for obesity drugs covered by
Medicaid under a Medicaid managed care plan that received a capitated payment to cover
medical costs, including prescription drugs, as those payments are not captured in CMS’s
Medicaid State Drug Utilization data.




Page 5                                                        GAO-19-577 Obesity Drugs
                                                        BMI is used as a screening tool for obesity. An individual with a BMI of 30
Background                                              or higher is considered to have obesity.

Body Mass Index (BMI) and Classes of                    Over the past two decades, both the prevalence of obesity and estimates
Obesity                                                 of the medical spending associated with individuals with obesity have
BMI is a person’s weight in kilograms divided           increased. For example, a 2018 study estimated that the percentage of
by the square of their height in meters.
                                                        national medical expenditures used to treat obesity-related illnesses in
Obesity (defined as a BMI of 30 or higher) is
frequently subdivided into categories:                  adults increased from 6.13 percent in 2001 to 7.91 percent in 2015, a 29
•     Class 1: BMI of 30 to <35                         percent increase. 15 This study also found that the high medical costs of
•     Class 2: BMI of 35 to <40                         obesity are due to extremely high medical costs among a small
•     Class 3: BMI of 40 or higher. Class 3             percentage of the population who have severe obesity (those with a BMI
      obesity is sometimes categorized as               of 40 or higher). In addition, a 2017 study found that medical expenditures
      extreme or severe obesity.                        rise most rapidly for individuals with a BMI of 40 or higher. 16
Source: Centers for Disease Control and Prevention. |
GAO-19-577

                                                        One option for the treatment of obesity is the use of prescription obesity
                                                        drugs. 17 As of June 2019, there were nine prescription drugs approved by
                                                        FDA to treat obesity. 18 Four obesity drugs—benzphetamine,
                                                        diethylpropion, phendimetrazine, and phentermine—were approved by
                                                        FDA in 1961 or earlier for short-term use, which is generally about 12
                                                        weeks, and are available as generic drugs. The remaining five obesity

                                                        15
                                                          This study presents estimates of the amount by which individuals with obesity have
                                                        higher medical expenditures than individuals who do not have obesity. One important
                                                        limitation noted in this study is that the estimates do not show a causal effect—that is,
                                                        even if individuals with obesity have higher medical costs, it does not mean the obesity
                                                        caused the higher medical costs. See A. Biener, J. Cawley, and C. Meyerhoefer, “The
                                                        Impact of Obesity on Medical Care Costs and Labor Market Outcomes in the US,” Clinical
                                                        Chemistry, vol. 64, no.1 (2018): 108-117.
                                                        16
                                                         Biener, Cawley, and Meyerhoefer, “The High and Rising Costs of Obesity,” S6-S8.
                                                        17
                                                          Obesity drugs are not for everyone with a high BMI; some people who are overweight or
                                                        have obesity may lose weight with a lifestyle program that helps them change their
                                                        behaviors and improve their eating and physical activity habits, according to the National
                                                        Institutes of Health. See National Institutes of Health, Prescription Medications to Treat
                                                        Overweight and Obesity, accessed May 2, 2019, https://www.niddk.nih.gov/health-
                                                        information/weight-management/prescription-medications-treat-overweight-obesity. Also,
                                                        see S.Z. Yanovski and J.A. Yanovski. “Long-term Drug Treatment for Obesity: A
                                                        Systematic and Clinical Review.” JAMA, vol. 311, no. 1 (2014): 74–86. In addition, the
                                                        U.S. Preventive Services Task Force recommends intensive, multicomponent behavioral
                                                        interventions as the primary focus of effective interventions for weight loss in adults. See
                                                        U.S. Preventive Services Task Force, Recommendation Statement, pp. 1163–1171.
                                                        18
                                                          There are two additional FDA-approved prescription obesity drugs, methamphetamine
                                                        and amphetamine, that we excluded from our review because they were not
                                                        recommended for use by any medical society guidelines we reviewed for the treatment of
                                                        obesity. Appendix II provides more detail on the obesity drugs included in our review.




                                                        Page 6                                                           GAO-19-577 Obesity Drugs
drugs were approved by FDA in 1999 or later for long-term use and are
available as brand-name drugs— bupropion/naltrexone (Contrave),
liraglutide (Saxenda), lorcaserin (Belviq), orlistat (Xenical), and
phentermine/topiramate (Qsymia). 19 Each of these five brand-name
obesity drugs underwent one or more randomized, controlled clinical trials
for safety and efficacy prior to FDA approval of the drug—a total of 15
clinical trials across the five drugs. Obesity drugs work in different ways;
some may help an individual feel full sooner or less hungry, while others
may reduce fat absorption in the body. Results vary by medication and by
person, but, according to the National Institutes of Health, on average,
people who take obesity drugs as part of a lifestyle program lose between
3 and 9 percent more of their starting body weight than people in a
lifestyle program who do not take obesity drugs. As with other prescription
drugs, obesity drugs may have side effects such as headache, dizziness,
dry mouth, nausea, and diarrhea. 20 And, as with other prescription drugs,
health care providers may prescribe an obesity drug for off-label use—
that is, for a different medical condition, in a different dosage, or for a
different duration than for which the drug is FDA approved. 21

Obesity drugs should be used as an adjunct to lifestyle therapy (e.g., diet,
physical activity, and behavioral counseling), according to guidelines from
several medical associations. 22 According to these guidelines, the use of
obesity drugs is indicated for individuals with a BMI of 27 or higher with
one or more obesity comorbidities (such as type 2 diabetes), or
individuals with a BMI of 30 or higher who have a history of failure to
achieve clinically meaningful weight loss (that is, weight loss of 5 percent

19
  These five obesity drugs are FDA approved for weight management as an adjunct to a
reduced-calorie diet and, for four of the drugs, as an adjunct to increased physical activity.
20
  These side effects are some of the most common, but they may not apply to all nine
obesity drugs as the different obesity drugs may have different side effects.
21
  Once FDA approves a drug, health care providers generally may prescribe the drug for
an unapproved use when they determine that it is medically appropriate for their patient.
22
  These guidelines include: (1) American College of Cardiology and American Heart
Association Task Force on Practice Guidelines and The Obesity Society, A Guideline for
the Management of Overweight and Obesity in Adults, (2013); (2) C.M. Apovian et al.,
“Pharmacological Management of Obesity: An Endocrine Society Clinical Practice
Guideline,” Journal of Clinical Endocrinology and Metabolism, vol. 100, no. 2, (2015): 342-
362; (3) W.T. Garvey et al., “American Association of Clinical Endocrinologists and
American College of Endocrinology Comprehensive Clinical Practice Guidelines for
Medical Care of Patients with Obesity,” Endocrine Practice, vol. 22, supp. 3, (2016): 1-
203; and (4) Obesity Medicine Association, Obesity Algorithm 2017-2018, (Denver, CO:
June 2018).




Page 7                                                             GAO-19-577 Obesity Drugs
                        or more) or who are unable to sustain weight loss. In addition, the
                        guidelines recommend evaluating the patient’s weight loss after about 12
                        to 16 weeks of treatment with an obesity drug and discontinuing the drug
                        if the patient has not lost a certain amount (e.g., at least 5 percent) of
                        their initial body weight.

                        Although obesity is classified as a disease, some health care providers,
                        including those who specialize in the care of patients with obesity,
                        continue to stigmatize patients with obesity. For example, a 2018 study
                        reported that health care providers may perceive patients with obesity as
                        being less compliant and having less self-discipline than other patients. 23
                        Additionally, health care providers may not initiate discussions about
                        weight loss with patients because of lack of time, other important issues
                        or concerns, a belief that a patient is not motivated or interested in losing
                        weight, or concern over a patient’s emotional state, according to another
                        2018 study. 24


                        The prevalence of obesity was about 38 percent among all U.S. adults
The Prevalence of       (about four of every 10 adults) from 2013 through 2016, according to
Obesity Was Close to    nationally representative estimates from CDC. The estimate of
                        prevalence among adults covered by Medicare was about 40 percent,
40 Percent among All    and among those with Medicaid or other public health insurance
U.S. Adults from 2013   (excluding Medicare) it was about 42 percent. In addition, the prevalence
                        of obesity among adults with private health insurance coverage and
through 2016            among the uninsured was similar, at about 37 percent and 38 percent,
                        respectively. These national estimates also showed that about 24 percent
                        of Medicare beneficiaries had Class 1 obesity, about 10 percent had
                        Class 2 obesity, and about 6 percent had Class 3, or severe, obesity.
                        (See fig. 1.)




                        23
                         D.H. Bessesen and L.F. Van Gaal, “Progress and Challenges in Anti-obesity
                        Pharmacology,” The Lancet Diabetes Endocrinology, vol. 6 (2018): 237-248.
                        24
                         L.M. Kaplan et al., “Perceptions of Barriers to Effective Obesity Care: Results from the
                        National ACTION Study,” Obesity, vol. 26, no. 1 (2018): 61-69.




                        Page 8                                                          GAO-19-577 Obesity Drugs
Figure 1: Prevalence of Obesity among U.S. Adults by Insurance Type and Class of
Obesity, 2013–2016




Notes: Individuals with a body mass index (BMI) of 30 or higher are considered to have obesity. BMI
is a person’s weight in kilograms divided by the square of their height in meters and is used as a
screening tool for obesity. The insurance categories are mutually exclusive. These estimates exclude
coverage by a single service plan, such as a dental or vision plan.
a
 Medicare estimates include all adults who reported having Medicare, including adults who also
reported having private health insurance or other public health insurance; if they reported having
Medicare, they were counted in the Medicare category and not in the private insurance or other public
insurance category.
b
 Adults who reported having private health insurance and another type of insurance, such as
Medicaid, were counted in the private insurance category except if they had Medicare—in which case
they were counted in the Medicare category.
c
 Medicaid accounted for about 46 percent of the Medicaid/other public insurance category in the
NHANES analysis, according to CDC. Other public insurance also includes military health care,
Indian Health Service; state-sponsored health plans, and other government programs. This excludes
Medicare coverage.




Page 9                                                                 GAO-19-577 Obesity Drugs
                       According to CDC estimates, adults age 18 to 64 and adults age 65 and
                       older had a similar prevalence of obesity, about 39 percent and 38
                       percent, respectively. 25 However, a higher percentage of adults age 18 to
                       64 than adults age 65 and older had Class 3 obesity. (See table 1.)
                       Appendix III provides additional information on the prevalence of obesity
                       among adults, as well as on the prevalence of adults who were
                       overweight, which is defined as a BMI of 25 to <30, including 95 percent
                       confidence intervals.

                       Table 1: Prevalence of Obesity in Adults by Age and Class of Obesity, 2013–2016

                        Age groups                    All adults with Class 1 (BMI of Class 2 (BMI of                                 Class 3 (BMI
                                                          body mass        30 to <35)      35 to <40)                                         ≥40)
                                                         index (BMI)
                                                                 ≥30
                        18–64                                         38.5                      20.4                       9.7                       8.4
                        65 and older                                  37.6                      23.6                      10.0                       4.0
                       Source: Centers for Disease Control and Prevention’s estimates from the National Health and Nutrition Examination Survey, 2013-
                       2016. | GAO-19-577




                       Relatively few U.S. adults, including adults with obesity and adults who
Few Adults Used        reported trying to lose weight, used obesity drugs from 2012 through
Obesity Drugs and      2016, according to nationally representative estimates. Guidelines
                       suggest prescribing obesity drugs as an adjunct to other diet and lifestyle
Limited Data Are       changes, or when other approaches have not resulted in clinically
Available on           significant weight loss. Those health care providers who prescribe obesity
                       drugs consider several factors, such as whether there are any
Individuals Who Have   contraindications of the obesity drug for their patients and the cost of the
Used These Drugs       drug. Some limited data are available on individuals who have used
                       obesity drugs, including data on whether these individuals adhered to
                       taking the prescribed obesity drug or maintained their weight loss over
                       time.




                       25
                         Of all U.S. adults with a BMI of 30 or higher, CDC estimated that about 81 percent were
                       age 18 to 64 and about 19 percent were age 65 and older.




                       Page 10                                                                                      GAO-19-577 Obesity Drugs
Relatively Few Adults   Available data indicate that relatively few U.S. adults, including those with
Used Obesity Drugs      obesity, used obesity drugs. 26 Specifically, of the estimated 233 million
                        U.S. adults, fewer than a million used any of the nine obesity drugs,
                        according to AHRQ’s nationally representative estimates from MEPS data
                        for 2012 through 2016. 27 Of the estimated 71.6 million U.S. adults with
                        obesity, an estimated 660,000 per year, on average, used an obesity
                        drug, according to these data. 28 Similarly, among those who reported
                        trying to lose weight, relatively few of them (about 3 percent) reported that
                        they took prescription medication for weight loss, according to CDC’s
                        nationally representative estimates from NHANES for 2013 through
                        2016. 29

                        Additionally, six of the studies we reviewed examined this topic and found
                        that few U.S. adults have used obesity drugs. 30 For example, one study
                        reported that in 2011, 2,554 obesity drug prescriptions were filled per
                        100,000 people, with about 87 percent of those prescriptions for
                        phentermine, a generic obesity drug. 31 Three other studies assessed the
                        use of obesity drugs among veterans receiving care from the Veterans
                        Health Administration and similarly found that few patients were




                        26
                          We define use of obesity drugs as having an outpatient prescription filled or refilled for
                        any of the nine obesity drugs.
                        27
                          Not all of the nine obesity drugs were on the market during the entire period from 2012
                        through 2016; two were approved by FDA in 2012 and two were approved in 2014. The
                        MEPS estimates are average annual estimates for the U.S. civilian, noninstitutionalized
                        population.
                        28
                          For these estimates, obesity was identified using a BMI of 30 or higher, calculated from
                        individual’s height and weight as reported by household respondents of MEPS. Because
                        some individuals may self report higher than actual height and lower than actual weight,
                        calculations of BMI from MEPS data may be lower than actual BMI.
                        29
                          The national survey used to obtain these data did not specifically ask about the nine
                        obesity drugs; however, it did ask about prescription medications for weight loss (i.e., diet
                        pills prescribed by a doctor), and the nine obesity drugs are the FDA-approved
                        prescription drugs specifically for weight management.
                        30
                          See appendix IV for a list of selected studies we reviewed pertaining to the use of
                        obesity drugs, as well as studies pertaining to physician considerations about obesity
                        drugs, individuals who used obesity drugs, and insurance coverage of obesity drugs.
                        31
                          C. Hampp, E.M. Kang, and V. Borders-Hemphill, “Use of Prescription Antiobesity Drugs
                        in the U.S.,” Pharmacotherapy, vol. 33 (2013): 1299-1307.




                        Page 11                                                            GAO-19-577 Obesity Drugs
                             prescribed obesity drugs. 32 One of these studies found that about 1
                             percent of the 153,939 veterans who enrolled in the MOVE! Weight
                             Management Program from 2013 through 2016 were prescribed an
                             obesity drug (orlistat, phentermine, phentermine/topiramate, liraglutide, or
                             bupropion/naltrexone) within 1 year of MOVE! initiation. 33


Physicians May Have          According to officials from groups representing physicians and advocacy
Concerns About               groups we interviewed, and seven studies we reviewed, some physicians
                             and other health care providers may not be open to or comfortable with
Prescribing Obesity Drugs;   prescribing obesity drugs. 34 For example, providers may not perceive
Those Who Do Prescribe       obesity drugs to be safe or effective. According to officials from one
These Drugs Consider         advocacy and research group, concerns about the safety of obesity drugs
Multiple Factors             may be related to the adverse consequences associated with past obesity
                             drugs. 35 In addition, one medical association we contacted indicated
                             physicians consider clinical preventive service recommendations from the
                             U.S. Preventive Services Task Force on the use of obesity drugs. 36 The
                             task force recommends that clinicians offer or refer adults with a BMI of

                             32
                               T.P. Semla et al., “Pharmacotherapy for Weight Management in the VHA,” Journal of
                             General Internal Medicine, vol. 32 (2017): 70-73; A.C. Del Re, S.M. Frayne, and A.H.S
                             Harris, “Antiobesity Medication Use Across the Veterans Health Administration: Patient-
                             Level Predictors of Receipt,” Obesity, vol. 22 (2014): 1968-1972; and D.D. Thomas et al.,
                             “Patient Characteristics Associated with Receipt of Prescription Weight-Management
                             Medications Among Veterans Participating in MOVE!” Obesity, vol. 27, no. 7 (2019): 1168-
                             1176.
                             33
                               Thomas et al., “Patient Characteristics Associated with Receipt of Prescription Weight-
                             Management Medications,” pp. 1-9. The MOVE! Weight Management Program is a weight
                             management, health promotion program that encourages healthy eating behavior,
                             increased physical activity, and small weight losses, and is supported by the Veterans
                             Administration’s National Center for Health Promotion and Disease Prevention. MOVE!
                             initiation was defined as having at least one MOVE! visit in the study period without having
                             a MOVE! visit in the previous 6 months.
                             34
                               The studies we reviewed were limited by, for example, small sample sizes and low
                             responses rates; however, they provide information for those physicians or other health
                             care providers who responded to a survey.
                             35
                               Dexfenfluramine and fenfluramine are prescription obesity medications that were
                             withdrawn from the market in 1997 because they were linked to heart valve abnormalities.
                             These medications were used alone or off label in combination with phentermine, which
                             was commonly known as fen-phen.
                             36
                               The U.S. Preventive Services Task Force is an independent, volunteer panel of national
                             experts in disease prevention and evidence-based medicine that makes evidence-based
                             recommendations about clinical preventive services. See U.S. Preventive Services Task
                             Force, Recommendation Statement, pp. 1163–1171.




                             Page 12                                                          GAO-19-577 Obesity Drugs
30 or higher to intensive, multicomponent behavioral interventions.
Further, a systematic review of evidence of the benefits and harms of
behavioral therapy and use of obesity drugs conducted for the task force
found that obesity drugs, but not behavior-based interventions, were
associated with higher rates of harm. 37 The potential for harm (i.e.,
adverse events) may discourage physicians and other health care
providers from prescribing these drugs. In addition, officials we
interviewed and the studies we reviewed noted that a lack of insurance
coverage, high out-of-pocket costs, and the patient’s means to afford
obesity drugs may also discourage physicians from prescribing obesity
drugs.

The officials and studies also noted that physicians might have gaps in
knowledge about obesity drugs. For example, officials from one medical
association noted that lack of education is a barrier to physicians in
prescribing obesity drugs for patients who would be candidates for them,
and officials from another medical association said that many clinicians
are not aware that there are FDA-approved drugs for obesity, and
therefore they do not think about prescribing them. One study we
reviewed found that, of the 111 primary care providers responding to a
survey, most reported limited experience with obesity drugs as a barrier
to prescribing them. 38 While guidelines on the use of obesity drugs
suggest prescribing obesity drugs as an adjunct to other diet and lifestyle
changes, or when other approaches have not resulted in clinically
significant weight loss, physicians and other health care providers may
not understand the recommendations outlined in the guidelines. 39 For
example, one study found that many of the health care providers
responding to a survey reported responses inconsistent with the




37
  See E.S. LeBlanc et al., “Behavioral and Pharmacotherapy Weight Loss Interventions to
Prevent Obesity-Related Morbidity and Mortality in Adults: Updated Evidence Report and
Systematic Review for the U.S. Preventive Services Task Force,” JAMA, vol. 320, no. 11
(2018): 1172-1191.
38
  This study was limited by a small sample size and low response rate, but it does provide
information on the perspectives of the 111 responding primary care providers. See R.
Simon and S.W. Lahiri, “Provider Practice Habits and Barriers to Care in Obesity
Management in a Large Multicenter Health System,” Endocrine Practice, vol. 24, no. 4
(2018): 321-328.
39
  Apovian et al., “Pharmacologic Management of Obesity,” pp. 342-362; and Garvey et al.,
“Comprehensive Clinical Practice Guidelines,” pp. 1-203.




Page 13                                                         GAO-19-577 Obesity Drugs
                           guideline-recommended thresholds to initiate and continue use of obesity
                           drugs. 40

                           Physicians and health care providers who do prescribe obesity drugs take
                           several factors into consideration. Specifically, before prescribing an
                           obesity drug, these providers consider the likely benefits of weight loss,
                           the drug’s possible side effects, the patient’s current health issues and
                           other medications, family medical history, and the cost of the drug,
                           according to the National Institutes of Health. 41 According to officials from
                           an advocacy group, specific considerations include (1) the patient’s other
                           health conditions that may increase the risk from using a particular
                           obesity drug (contraindications); (2) the ability of an obesity drug to treat
                           both the patient’s obesity and other health conditions; (3) the patient’s
                           ability to afford a particular obesity drug, given their insurance coverage
                           and other financial resources; (4) patient preference regarding the dosage
                           and form of the drug; and (5) the average efficacy (weight loss) of an
                           obesity drug. 42 Further, when treating obesity, providers use the least
                           invasive treatments, such as lifestyle-based therapies first, then escalate
                           to obesity drugs if noninvasive treatments prove ineffective, according to
                           officials from the same advocacy group.


Some Limited Data Are      Some limited data are available on individuals who have used obesity
Available on Individuals   drugs, including data on the distribution of BMI, the use of obesity drugs
                           in conjunction with other items or services, whether these individuals
Who Have Used Obesity
                           adhered to using the prescribed obesity drug or maintained their weight
Drugs                      loss over time, and the impact that using obesity drugs has on other
                           medical services directly related to obesity. The following is a summary of
                           available information on specific aspects of individuals who have used
                           obesity drugs.



                           40
                             M. Turner et al., “Current Knowledge of Obesity Treatment Guidelines by Health Care
                           Professionals,” Obesity, vol. 26, no. 4 (2018): 665-671.
                           41
                             National Institutes of Health, Prescription Medications to Treat Overweight and Obesity,
                           accessed May 2, 2019, https://www.niddk.nih.gov/health-information/weight-
                           management/prescription-medications-treat-overweight-obesity.
                           42
                             For example, phentermine is contraindicated for individuals with a history of
                           cardiovascular disease (e.g., coronary artery disease, stroke, arrhythmias, congestive
                           heart failure, and uncontrolled hypertension), and phentermine/topiramate (Qsymia) is
                           contraindicated for people with glaucoma.




                           Page 14                                                         GAO-19-577 Obesity Drugs
Distribution of BMI across individuals who have used obesity drugs.
CDC’s nationally representative estimates for 2013 through 2016 found
that the BMI of adults who reported that they used obesity drugs ranged
from 21 to 64, with a median BMI of 34. However, these data are limited
because they do not indicate how long the individual used the drugs
before their BMI was measured. 43

Use of obesity drugs in conjunction with other items or services.
Two studies we reviewed examined the use of obesity drugs in
conjunction with other items or services. These studies found that
participants who used an obesity drug in conjunction with other services,
such as behavioral counseling, lost more weight than those who did not
take the drug with the other services. For example, in one 2019 study,
participants who received intensive behavioral therapy combined with an
obesity drug, liraglutide, had nearly double the weight loss (an average of
about 12 percent of their body weight) compared to the participants who
received only intensive behavioral therapy (an average of about 6 percent
of their body weight). 44 In addition, the 15 clinical trials for the brand-name
obesity drugs that we reviewed generally found that a significantly higher
percentage of participants who used the obesity drug combined with other
items or services (such as a low-calorie diet or increased physical activity)
achieved 5 percent or more weight loss compared to participants who
used a placebo with the other items or services. One clinical trial that
used an intensive behavior modification program (28 group sessions)
found higher average weight loss (9 percent loss of initial body weight) for
participants who used the obesity drug (bupropion/naltrexone) than for
the placebo group. 45 This clinical trial also found that the placebo group
with the intensive behavior modification had higher weight loss than



43
  These are the BMIs at the time of the physical examination, not necessarily when an
individual started or stopped using the obesity drugs. These estimates are based on data
from 166 NHANES participants who reported that they tried to lose weight and took diet
pills prescribed by a doctor.
44
  The intensive behavioral therapy consisted of 21 visits of counseling. The participants
who received intensive behavioral therapy combined with the obesity drug were divided
into two groups, one group with a meal replacement diet and the other group without this
diet. See T.A. Wadden et al., “Intensive Behavioral Therapy for Obesity Combined with
Liraglutide 3.0 mg: A Randomized Controlled Trial,” Obesity, vol. 27 (2019): 75-86.
45
  T.A. Wadden et al., “Weight Loss With Naltrexone SR/Bupropion SR Combination
Therapy as an adjunct to Behavior Modification: The COR-BMOD Trial,” Obesity, vol. 19,
no. 1 (2011): 110-120.




Page 15                                                         GAO-19-577 Obesity Drugs
placebo groups in the other clinical trials, none of which used intensive
behavioral therapy.

Adherence to using the prescribed obesity drug. FDA’s analysis of
Sentinel System data of obesity drugs dispensed in 2008 through 2017
found that in the majority of patients using obesity drugs, cumulative
treatment duration was 90 days or less. FDA analyzed data for 267,836
new users of obesity drugs and found that about 58 percent of patients
who used any of the obesity drugs did so for 90 days or less; about 31
percent used any of the obesity drugs for 30 or fewer days. 46 The average
duration for the first use of any of the nine obesity drugs was 69 days. 47
(See appendix V for more data from FDA’s analysis.) FDA’s findings are
consistent with the findings of two of the three studies that we reviewed
that measured adherence to using the prescribed obesity drug. 48 These
studies reported that use of obesity drugs dropped significantly after 30
days. For example, one 2018 study that reviewed 1 year of data on
26,522 patients who had new prescription drug claims for one of four
obesity drugs (liraglutide, lorcaserin, bupropion/naltrexone, and
phentermine/topiramate) found that adherence to using any of the four

46
   A new user was defined as having no use of any obesity drug in the previous 183 days.
FDA used a 14-day episode gap to estimate the duration of the first treatment episode (in
days) of prescriptions filled for any of the nine obesity drugs. FDA defined an episode gap
as the maximum allowable number of days between exhaustion of the supply of the
previously dispensed medication and refilling the prescription. According to FDA, the
purpose of the episode gap is to define how multiple dispensings of a medication are
linked to estimate the total number of days patients were taking the medication during one
treatment episode. Further, the episode gap may account for missed doses, late refills,
and other sources of variability in adherence between dispensings. Although these data
are not medication administration data, this analysis assumes that patients who obtained
multiple dispensings took the dispensed doses of their obesity drugs prior to the last
refill. In addition, FDA assessed all dispensed prescriptions to maximize the information
captured. Because prescribers are not required to document the indication, it is possible
that some drug dispensings were for indications that are not labeled, according to FDA.
While some obesity drugs are FDA approved for short-term use and some are approved
for long-term use, FDA’s analysis found that the median duration for the first treatment
episode for individual obesity drugs was 46 days for phentermine/topiramate (Qsymia); 42
days for liraglutide (Saxenda), and 30 days for each of the other obesity drugs.
47
  For individual obesity drugs, average duration for the first treatment ranged from about
47 days for benzphetamine to about 91 days for phentermine/topiramate (Qsymia). For
phentermine, which was prescribed for 198,203 of the 267,836 patients in FDA’s analysis,
and which is approved for short-term use but used off label for longer durations, the
average duration for the first treatment was about 68 days.
48
  The studies refer to this as (1) adherence to taking the obesity drugs, (2) duration of
use, or (3) persistence with taking the drugs.




Page 16                                                           GAO-19-577 Obesity Drugs
obesity drugs dropped markedly during the first month following the initial
claim for the drug. 49 In addition, while the 15 clinical trials we reviewed
were not designed to measure adherence to taking obesity drugs, they
provide some information on whether or not study participants adhered to
using these drugs during the trials. Participant dropout rates for these
clinical trials ranged from 14 percent to 66 percent for the obesity drug
treatment and the placebo groups, which could indicate difficulty in
adherence to the study regimen; however, participants using the placebo
generally had higher dropout rates than those using the obesity drug. The
reasons for discontinuation among study participants in the clinical trials
included side effects, such as headaches and nausea; being unavailable
for follow up; and withdrawal of consent.

Maintaining weight loss over time by individuals who have used
obesity drugs. The recent systematic review conducted for the U.S.
Preventive Services Task Force noted that data on long-term weight loss
with obesity drugs are limited. The review found that individuals using
obesity drugs were more likely to maintain their weight loss over 12 to 36
months compared with placebo, but noted that the evidence was limited
by the small number of trials for each medication, poor follow up with
participants, and limited applicability (given that participants had to meet
narrowly defined inclusion criteria), among other limitations. 50 We also
identified six studies—each of which reviewed one of the FDA-approved
obesity drugs—that examined weight loss maintenance, generally after
about 1 year. For example, a 2018 study for one obesity drug (lorcaserin)
found that while the obesity drug initially improved upon weight loss
achieved with weight loss maintenance counseling, this advantage was


49
  Specifically, the study reviewed 26,522 patients that had new prescription drug claims
for one of the four obesity drugs and found at month 12, about 28 percent of patients were
still taking liraglutide compared to about 7 percent taking lorcaserin, 9 percent taking
bupropion/naltrexone, and 11 percent taking phentermine/topiramate. One limitation noted
in the study was that even though the obesity drugs were prescribed to patients, it does
not necessarily mean that the drugs were used. See R. Ganguly et al., “Persistence of
Newer Anti-Obesity Medications in a Real-World Setting,” Diabetes Research and Clinical
Practice, vol. 143 (2018): 348-356.
50
  The review examined 35 clinical trials that examined the effectiveness of or harms of
medication for weight loss and weight loss maintenance for five obesity drugs: liraglutide,
lorcaserin, bupropion/naltrexone, orlistat (both prescription strength and the over-the-
counter dosage), and phentermine/topiramate. Other limitations included methodological
variability and missing data regarding dispersion. The review noted that strength of
evidence of medication-based weight loss maintenance was insufficient. See LeBlanc et
al., “Behavioral and Pharmacotherapy Weight Loss Interventions,” pp. 1172-1191.




Page 17                                                          GAO-19-577 Obesity Drugs
not maintained at 1 year. 51 That is, after 1 year, there was no significant
difference in weight loss maintenance between the participants treated
with the obesity drug along with counseling, compared to those treated
with placebo along with counseling. Another study that examined clinical
trial data for one obesity drug (bupropion/naltrexone) concluded that
participants who lost at least 5 percent of their body weight after 16
weeks were likely to maintain clinically significant weight loss (of at least 5
percent) after 1 year of treatment with the drug. 52

The impact of using obesity drugs on medical services directly
related to obesity. We did not identify any studies on the impact that the
use of obesity drugs had on the utilization of medical services directly
related to obesity. In terms of studies on the impact on health outcomes,
the systematic review conducted for the U.S. Preventive Services Task
Force concluded that health outcomes data for individuals receiving
treatment with obesity drugs were limited. 53 The review reported that
clinical trials of obesity drugs for weight loss examined few outcomes
beyond quality of life measures, and that none of the drug-based
maintenance trials reported the effects of the obesity drug interventions
on health outcomes. The review noted that the trials included in the
51
  The study examined 137 adults who had lost at least 5 percent of initial weight during a
14-week low-calorie diet program before being put into the obesity drug or placebo
groups. Participants were randomly assigned to the obesity drug (lorcaserin) or a placebo
and were provided 16 group weight loss maintenance counseling sessions over 52 weeks.
See J.S. Tronieri et al., “A Randomized Trial of Lorcaserin and Lifestyle Counseling for
Maintaining Weight Loss Achieved with a Low-Calorie Diet,” Obesity, vol. 26 (2018): 299-
309.
52
  The study examined data for 1,310 participants who took the obesity drug
(bupropion/naltrexone) in four clinical trials designed to evaluate the efficacy and safety of
the drug over a 56-week period. See K. Fujioka et al., “The Relationship between Early
Weight Loss and Weight Loss at 1 Year with Naltrexone ER/Bupropion ER Combination
Therapy,” International Journal of Obesity, vol. 40 (2016): 1369-1375.

In addition, one of the clinical trials examined weight loss maintenance over a 56-week
period and found that more participants taking the obesity drug (liraglutide) with diet and
exercise maintained their initial ≥ 5 percent weight loss than those taking a placebo with
diet and exercise. This trial also noted that further study is needed of the frequency of
medication usage required to facilitate weight loss maintenance. See T.A. Wadden et al.,
“Weight Maintenance and Additional Weight Loss with Liraglutide after Low-Calorie-Diet-
Induced Weight Loss: The SCALE Maintenance Randomized Study,” International Journal
of Obesity, vol. 37 (2013): 1443-1451.
53
  The review examined 10 trials of medications for weight loss that examined health
outcomes and concluded the strength of evidence was low for benefit for health outcomes
from medication-based weight loss. See LeBlanc et al., “Behavioral and Pharmacotherapy
Weight Loss Interventions,” pp. 1172-1191.




Page 18                                                            GAO-19-577 Obesity Drugs
                       review were of highly selected populations with multiple exclusions
                       relevant to health outcomes (e.g., history of serious medical conditions).
                       The review further noted that while it appears that weight loss
                       interventions, including obesity drugs, can reduce diabetes incidence,
                       larger studies with longer-term follow up are required to understand the
                       full benefits of these interventions on health outcomes and whether those
                       effects are long lasting.


                       Health insurance coverage for obesity drugs is limited—that is, not all
Health Insurance       public and private health insurance provided coverage for obesity drugs
Coverage for Obesity   or may have additional requirements to determine these drugs are
                       medically necessary. Medicare Part D plans may opt to cover obesity
Drugs Is Limited and   drugs, and state Medicaid programs or Medicaid managed care plans
Varied across Types    within states may choose either to cover or exclude obesity drugs from
                       coverage. We found that both Medicare Part D and Medicaid reimbursed
of Insurance           for a relatively small number of prescriptions for obesity drugs in 2016
                       and 2017. For private health insurance—which includes employer-
                       sponsored health insurance, individually purchased health plans, and
                       FEHBP plans—we found that coverage varied and, when obesity drugs
                       were covered, the coverage could have additional requirements such as
                       prior authorization or determination that a drug is medically necessary for
                       the patient.

                       Medicare. Under Medicare’s prescription drug benefit, Medicare Part D
                       plans may choose to cover obesity drugs—in these cases, obesity drugs
                       are considered supplemental drugs under an enhanced alternative
                       coverage plan. 54 Medicare beneficiaries who select a Part D plan that
                       offers supplemental benefits, which may include coverage of excluded
                       drugs such as obesity drugs, must pay the full premium cost for those




                       54
                         Drugs for weight loss, even if used for a non-cosmetic purpose, are excluded from the
                       definition of a Part D drug covered under Medicare Part D. See 42 U.S.C. § 1395w-
                       102(e), 42 C.F.R. § 423.100 (2018), and 42 C.F.R. § 423.104(f)(ii)(A) (2018). Medicare
                       Part D prescription drug plans include Medicare Advantage Prescription Drug plans and
                       Medicare standalone prescription drug plans, which are collectively referred to as
                       Medicare Part D plans in our report.




                       Page 19                                                        GAO-19-577 Obesity Drugs
additional benefits (i.e., Medicare does not subsidize them). 55 Medicare
Part D plans can choose whether or not to offer enhanced alternative
coverage, and not all Medicare Part D plans that provide enhanced
alternative coverage cover obesity drugs as supplemental drugs. 56 For
example:

•    Roughly half of the Medicare beneficiaries covered by one large
     insurer’s Medicare Part D plans in one state have coverage for
     obesity drugs as a supplemental drug under enhanced alternative
     coverage, according to officials from that insurer.
•    Officials at another large insurer told us that their Medicare Part D
     plans have historically covered supplemental drugs based on
     consumer demand, and obesity drugs do not typically meet their
     threshold for offering supplemental coverage. The officials noted that
     their plans have limited funds to cover supplemental drugs and that
     consumer demand is typically highest for other types of drugs, such
     as drugs to treat erectile dysfunction.




55
  For example, in 2017, average monthly premiums for beneficiaries in standalone
Medicare Part D plans with enhanced benefits were $54 per month compared with an
average premium of $31 for basic coverage. See Medicare Payment Advisory
Commission, June 2018 Data Book: Health Care Spending and the Medicare Program,
Section 10 (Washington, D.C.: July 2018), and Medicare Payment Advisory Commission,
Report to the Congress: Medicare Payment Policy, Chapter 14 (Washington, D.C.: March
2018).
56
  Once a Medicare Part D plan sponsor offers a plan with basic benefits in a region, it can
also offer up to two plans with additional drug coverage that supplements the standard
benefit, called enhanced plans. As of February 2017, 1,949 Medicare Part D plans
provided enhanced alternative coverage to 18.9 million Medicare beneficiaries, according
to the Medicare Payment Advisory Commission. These numbers include all of the plans
that provided supplemental benefits. Plans may offer supplemental benefits, such as
reductions in cost sharing, without covering any supplemental drugs, and plans choose
which supplemental drugs they cover (so not all plans offering coverage of supplemental
drugs choose to cover obesity drugs). For more information on Medicare Part D plans, see
Medicare Payment Advisory Commission, June 2018 Data Book: Health Care Spending
and the Medicare Program, Section 10 (Washington, D.C.: July 2018), and Medicare
Payment Advisory Commission, Report to the Congress: Medicare Payment Policy,
Chapter 14 (Washington, D.C.: March 2019).




Page 20                                                         GAO-19-577 Obesity Drugs
Enhanced Alternative Coverage and                     Data from CMS on Medicare Part D reimbursement for obesity drugs
Supplemental Drugs under Medicare                     provide some insight on coverage. 57 For example, our analysis found that
Enhanced alternative coverage is                      in 2017, 27 Medicare Part D plans reimbursed for obesity drugs under
alternative prescription drug coverage under
Medicare Part D with value exceeding that of          enhanced alternative coverage for 209 Medicare beneficiaries. 58 (See
Medicare Part D’s defined standard coverage.          table 2 for 2016 and 2017 data.) See appendix VI for more information.
Enhanced alternative coverage may include
basic prescription coverage and supplemental
benefits such as supplemental drugs.                  Table 2: Number of Medicare Part D Plans and Beneficiaries Reimbursed for
Supplemental drugs are drugs—including                Obesity Drugs, 2016 and 2017
drugs for weight loss—that would be covered
Part D drugs but for the fact that they are            Medicare Part D plan reimbursements for obesity drugs as                                     2016       2017
specifically excluded as Part D drugs under            supplemental drugs under enhanced alternative coverage
Medicare Part D’s basic prescription drug
coverage. Medicare Part D plans may offer                                                                                                             32          27
                                                       Number of Part D plans
these excluded drugs, such as obesity drugs,
as a supplemental drug under enhanced                  Number of claims                                                                              419        555
alternative coverage.                                                                                                                                196        209
                                                       Number of beneficiaries
A Medicare Part D plan can choose which
drugs it covers as a supplemental drug under          Source: GAO analysis of Centers for Medicare & Medicaid Services data. | GAO-19-577
enhanced alternative coverage—that is, not
all plans cover the same supplemental drugs
as part of enhanced alternative coverage.
Source: Centers for Medicare & Medicaid Services. |   Medicaid. State Medicaid programs or Medicaid managed care plans
GAO-19-577
                                                      within states may choose either to cover or exclude obesity drugs from
                                                      coverage. 59 Our analysis found that in 2017, Medicaid programs or
                                                      Medicaid managed care plans in 41 states reimbursed pharmacies and
                                                      other providers for at least one claim for an obesity drug, for a total of




                                                      57
                                                        Our analysis was limited to those instances in which a Medicare Part D plan reimbursed
                                                      for an obesity drug as a supplemental drug under enhanced alternative coverage, and the
                                                      actual number of Medicare Part D plans that provided coverage for obesity drugs could be
                                                      higher. For example, some plans may have covered obesity drugs but none of the
                                                      beneficiaries enrolled in these plans filled a prescription and submitted a claim for an
                                                      obesity drug.
                                                      58
                                                        According to CMS, the amount a plan pays for a supplemental drug under enhanced
                                                      alternative coverage is not included as part of the Medicare Part D benefit for payment
                                                      purposes. Additionally, according to the Research Data Assistance Center (a CMS
                                                      contractor that provides assistance to researchers and others regarding CMS data) the
                                                      amount the plan pays for supplemental drugs is recouped by the plan through higher
                                                      premiums for the enhanced benefit.
                                                      59
                                                       State Medicaid programs may exclude drugs when used for anorexia, weight loss, or
                                                      weight gain. See 42 U.S.C. § 1396r–8(d)(2)A).




                                                      Page 21                                                                               GAO-19-577 Obesity Drugs
30,800 prescriptions. 60 (See table 3 for 2016 and 2017 data.) Medicaid
managed care organizations may provide coverage of obesity drugs not
covered by the state plan, according to CMS. See appendix VII for more
information.

Table 3: Numbers of States and Prescriptions with Medicaid Reimbursement for
Obesity Drugs, 2016 and 2017

 Medicaid reimbursements for                                                           2016                 2017
 obesity drugs
 Number of states                                                                        42                    41
 Number of prescriptions                                                              25,312               30,800
Source: GAO analysis of Centers for Medicare & Medicaid Services data. | GAO-19-577




60
  States include the 50 states and the District of Columbia. The amount Medicaid
reimbursed includes both federal and state reimbursement and includes dispensing fees.
There were about 732 million prescriptions reimbursed in Medicaid in fiscal year 2016 and
about 757 million prescriptions reimbursed in fiscal year 2017, according to the Medicaid
and CHIP Payment and Access Commission. See Medicaid and CHIP Payment and
Access Commission, MACStats: Medicaid and CHIP Data Book (Washington, D.C.:
December 2017) and Medicaid and CHIP Payment and Access Commission, MACStats:
Medicaid and CHIP Data Book (Washington, D.C.: December 2018).

This analysis was limited to those instances in which Medicaid’s data indicated that
Medicaid reimbursed for an obesity drug (including instances in which the reimbursement
for the drug was included in a monthly capitated rate under a Medicaid managed care
plan, and therefore showed up as $0 amounts in the CMS data), and the actual number of
states in which Medicaid plans or Medicaid managed care plans provided coverage for
obesity drugs could be higher. For example, a state could have provided coverage for
obesity drugs, but no beneficiaries in that state filled a prescription for an obesity drug. We
did not find comprehensive, reliable data on the number of Medicaid state programs or
Medicaid managed care plans within states that provided coverage for obesity drugs.




Page 22                                                                                   GAO-19-577 Obesity Drugs
Employer-sponsored and individually purchased health plans.
Coverage of the nine obesity drugs varied in employer-sponsored and
individually purchased health plans, according to the insurers and
pharmacy benefit managers we interviewed. 61 For example:

•   Officials from one large insurer told us that coverage of obesity drugs
    is included in plans for about 90 percent of their members; only a
    small percentage of members do not have plans with this coverage.
•   Officials from another large insurer surveyed its health plans in
    different geographic locations and found that, of those that responded,
    four of the six employer-sponsored and three of the six individually
    purchased health plans covered the nine obesity drugs. They said that
    many of the plans that covered obesity drugs in their employer-
    sponsored markets also covered these drugs in their individual
    market.
•   Officials at a large pharmacy benefit manager said employers that
    provide employer-sponsored health insurance can choose to
    customize their formulary and decide whether to include obesity
    drugs. They said their select and premium prescription drug
    formularies include obesity drugs, so companies that decide to offer
    those formularies would cover obesity drugs, but many companies
    choose to customize their formularies and may not include obesity
    drugs.

Even if employer-sponsored and individually purchased health plans offer
coverage of obesity drugs, these plans often put requirements in place to
determine a beneficiary’s eligibility for coverage of obesity drugs,
according to officials from insurers and pharmacy benefit managers we
interviewed. For example, plans may require beneficiaries to obtain prior
authorization, require a determination of medical necessity of the drug for
the patient, and review the drug’s effectiveness prior to making a
coverage decision. For example, an official from one large insurer told us

61
  Employer-sponsored health plans may be self-funded (by setting aside funds to pay for
employee health care) or fully insured (by purchasing coverage from an issuer). Most
small employers purchase fully insured plans, while most large employers self-fund at
least some of their employee health benefits. For more information see GAO, Private
Health Insurance: Enrollment Remains Concentrated among Few Issuers, including in
Exchanges, GAO-19-306 (Washington, D.C.: Mar. 21, 2019). Individual health insurance
plans refer to health insurance that an individual can purchase on their own. Private
insurance plans may be subject to state insurance requirements pertaining to drug
coverage.




Page 23                                                       GAO-19-577 Obesity Drugs
their drug formulary does not include obesity drugs because the clinical
evidence indicates that other therapies are more effective for weight loss.
However, this official also said that some of its plans would cover obesity
drugs as a nonformulary option if a physician or other health care provider
indicates that the obesity drug is medically necessary (e.g., after a patient
has tried other treatment options, such as behavioral therapy). 62

Further, if a patient is offered coverage of an obesity drug but fails to
receive a clinical benefit within a specified time frame, insurers and
pharmacy benefit managers told us the following:

•    A patient and his or her physician may decide together whether the
     patient should continue or discontinue the obesity drug, and plans
     often defer to physicians to determine whether an obesity drug is
     medically necessary for a patient.
•    Some plans may require additional information from a patient’s
     physician every 6 to 12 months for reapproval of coverage of an
     obesity drug, such as reporting outcomes (e.g., weight loss) while
     using the drug.
•    Plans could require prior authorization to continue using an obesity
     drug.
•    An individual may be able to try a different obesity drug covered by
     the formulary.

For the largest employer-sponsored health care program in the United
States—FEHBP, managed by the Office of Personnel Management—we
found that some FEHBP plans offered by large insurers excluded obesity
drugs from coverage. 63 We examined the formularies for 12 plans offered
by three large FEHBP insurers and found that the formularies for two
plans from one insurer indicated some type of coverage of obesity drugs
in 2018. One plan offered coverage for 50 percent of the plan’s allowed
amount for weight management drugs, and the other plan offered
62
  Nonformulary drugs are not covered unless approved by the health plan as a coverage
exception.
63
  Recognizing that many plans exclude such drugs from coverage, the Office of Personnel
Management issued guidance clarifying that exclusion of obesity drugs on the basis that
obesity is a “lifestyle” condition and not medical, or that obesity treatment is “cosmetic” is
not permissible. It also clarified that there is no prohibition on FEHBP plans covering such
drugs, provided that appropriate safeguards are implemented. See Office of Personnel
Management, FEHBP Carrier Letter, No. 2014-04 (March 20, 2014), p. 1.




Page 24                                                           GAO-19-577 Obesity Drugs
coverage of two obesity drugs as tier 2 drugs, which have higher
copayments than tier 1 drugs. 64

For individually purchased health plans offered on health care exchanges,
nine of the 34 states with federally facilitated exchanges had at least one
plan in the silver tier of coverage that included some type of coverage for
obesity drugs in 2018, according to a 2018 study. 65 The study found that
covered obesity drugs were generally the older drugs and that the newer
drugs tended to be covered with higher copayments or more likely to
require prior authorizations than other medications. 66




64
  The two drugs covered as tier 2 drugs were diethylpropion and phentermine. A third
formulary for a plan offered by this insurer indicated that the five brand-name drugs and
Adipex (a brand name for phentermine) are tier 3 drugs, which means that these drugs
are not listed on the drug formulary and are not covered unless approved through an
exception process.
65
  This study examined coverage for eight of nine FDA-approved obesity drugs in plans in
the silver tier of coverage. See G. Gomez and F.C. Stanford, “US Health Policy and
Prescription Drug Coverage of FDA-approved Medications for the Treatment of Obesity,”
International Journal of Obesity, vol. 42 (2018): 495-500.

Federally facilitated exchanges are CMS-operated individual market exchanges. They are
categorized into four “metal” tiers of coverage—bronze, silver, gold and platinum—which
reflect out-of-pocket costs that may be incurred by an enrollee. For more information see
GAO, Health Insurance Exchanges: HHS Should Enhance Its Management of Open
Enrollment Performance, GAO-18-565 (Washington, D.C.: Jul. 24, 2018). Silver plans,
according to 2016 enrollment data, accounted for 71 percent of enrollees using the
federally facilitated exchanges at healthcare.gov.
66
  Lower-tiered drugs tend to be generic and are often included in the plan’s formulary.
Therefore, the copayment is typically lower and often does not require prior authorization.
Higher-tiered drugs tend to be brand name, and they are more likely to have higher
copayments and often require prior authorization and/or quantity limits. See Gomez and
Stanford, “Prescription Drug Coverage of FDA-approved Medications for the Treatment of
Obesity,” pp. 495-500.




Page 25                                                          GAO-19-577 Obesity Drugs
                             Out-of-pocket payments from the patient or patient’s family made up two-
Two-Thirds of Obesity        thirds of the amounts paid for obesity drugs, according to nationally
Drug Payments Were           representative estimates for 2012 through 2016. These amounts could
                             include insurance copayments and deductible amounts, and payments for
Made Out of Pocket;          obesity drugs not covered by insurance. Private health insurance paid
Adults Who Used              about one quarter of the amount paid for obesity drugs, and Medicare and
                             other public health insurance paid the remainder. Average annual medical
Obesity Drugs Had            spending and prescription drug spending were higher for adults who used
Higher Average               any of the nine obesity drugs than for those who did not, according to
                             these estimates. 67 However, the differences in these estimates do not
Estimated Medical            establish any causal relationship between using obesity drugs and having
Spending                     higher average annual medical or prescription drug spending.


Two-Thirds of Obesity        Out-of-pocket payments made up about two-thirds of total amounts paid
Drug Payments Were Paid      for obesity drugs for U.S. adults and private health insurance paid a
                             quarter, according to AHRQ’s nationally representative estimates from
Out of Pocket by Patients;   MEPS data for 2012 through 2016. 68 Medicare, Medicaid, and other
Phentermine Was Most         public health insurance paid the remainder; however, estimates for each
Purchased                    of these sources of payment are imprecise. (See fig. 2.) Similar to studies
                             on the use of obesity drugs, AHRQ’s estimates also found that 80 percent
                             of amounts paid for any of the nine obesity drugs was for one obesity
                             drug, phentermine, which is available as a generic drug.




                             67
                               We define use of obesity drugs as having an outpatient prescription fill or refill for any of
                             the nine obesity drugs.
                             68
                               For this analysis, AHRQ estimated the distribution of payments for obesity drugs from
                             MEPS pooled data for years 2012 through 2016 for adults in the U.S. civilian,
                             noninstitutionalized population. For additional information on this analysis, see appendix
                             VIII.




                             Page 26                                                            GAO-19-577 Obesity Drugs
Figure 2: Estimated Average Annual Estimates of Distribution of Payments for
Obesity Drugs by Insurance Type, 2012–2016




a
 Other payments include payments made by TRICARE, the Veterans Administration, or other federal
government sources, such as the Indian Health Service and military treatment facilities. The relative
standard error is greater than 30 percent for this payment category estimate.
b
    The relative standard error is greater than 30 percent for the Medicare payment estimate.
c
 The relative standard error is greater than 30 percent for the Medicaid payment estimate.
d
 Out-of-pocket payments include payments made by the patient or the patient’s family, including
insurance copayments and deductible amounts, and payments for obesity drugs not covered by
insurance.




We also examined available spending data from CMS on payments for
obesity drugs and found the following:

•       Medicare Part D prescription drug plans spent $19,714 for obesity
        drugs in 2016 and $140,296 in 2017, according to our analysis of
        CMS’s Prescription Drug Event data. 69 These amounts include
        Medicare Part D plan reimbursements for any of the nine obesity
        drugs under enhanced alternative coverage. CMS’s data also showed
        that total beneficiary spending—that is, the total amount Medicare
        beneficiaries paid out of pocket as copayments or deductibles—for
69
     In our analysis, Medicare Part D spending reflects plan payments to pharmacies.




Page 27                                                                    GAO-19-577 Obesity Drugs
     any of these prescriptions totaled $4,048 in 2016 and $5,376 in
     2017. 70 See appendix VI for more information.
•    Total Medicaid state and federal spending—that is, reimbursement
     amounts for the nine obesity drugs—was at least $5,017,424 in 2016
     and $7,453,442 in 2017, according to our analysis of available data
     from CMS’s Medicaid State Drug Utilization data. 71 These amounts do
     not include all Medicaid spending for obesity drugs under Medicaid
     managed care. For example, if a Medicaid program pays a managed
     care organization for drugs as part of their capitated payment for all
     Medicaid services, they are not reimbursed on a per-drug basis, and
     obesity drugs covered by Medicaid in that state would show up as a
     $0 reimbursement amount in CMS’s Medicaid State Drug Utilization
     data. 72 According to CMS data, Medicaid spending for obesity drugs
     was the greatest in California in 2016 and 2017. See appendix VII for
     more information.

In addition, when the number of prescriptions dispensed are counted,
FDA’s estimates from 2017 IQVIATM data—which are projected nationally
from prescriptions dispensed in about 59,900 outpatient retail
pharmacies—found that most prescriptions dispensed for obesity drugs

70
  Beneficiary spending refers to how much a beneficiary’s responsibility is for a given
claim. The amount includes how much a beneficiary pays for a drug that is not reimbursed
by a third party, the amount a patient’s liability is reduced due to other benefits such as the
Veterans Administration and TRICARE, the amount a plan reduced the patient’s liability
due a beneficiary’s low-income cost sharing subsidy amount, and all other qualified third
party payments on behalf of the beneficiary, which are referred to as the Other True Out-
of-Pocket. The low-income cost sharing subsidy amount and Other True Out-of-Pocket
amounts are zero for obesity drugs because they are covered under an enhanced benefit,
not as Part D drugs.

In 2015, total Medicare Part D spending for prescription drugs was about $137 billion—
this represents payments from all payers including beneficiaries (cost sharing), and
excluding rebates and discounts from pharmacies and manufacturers that are not
reflected in prices at the pharmacies. See Medicare Payment Advisory Commission, June
2018 Data Book: Health Care Spending and the Medicare Program (Washington, D.C.:
July 2018), 173.
71
  State and federal reimbursement amounts refer to gross spending or expenditures prior
to the application of manufacturer rebates. Total Medicaid state and federal
reimbursement for all prescription drugs in fiscal year 2016 was about $60.8 billion and
about $64 billion in fiscal year 2017.
72
  We found 776 and 144 prescriptions for obesity drugs in CMS’s Medicaid State Drug
Utilization data in 2016 and 2017, respectively, that showed $0 reimbursement; according
to CMS officials, obesity drugs covered as part of capitated payments under Medicaid
managed care plans can be reported with zero dollar amounts in the CMS data.




Page 28                                                            GAO-19-577 Obesity Drugs
                          were paid for by private insurance. FDA’s analysis found that almost 64
                          percent of prescriptions dispensed for any of the nine obesity drugs was
                          paid for by private health insurance, and 35 percent of prescriptions
                          dispensed was paid for by cash (i.e., out-of-pocket) payments paid for by
                          the patient or their family in 2017. 73 The remaining 1 percent of
                          prescriptions dispensed for obesity drugs was paid for by Medicare Part D
                          and Medicaid at an estimated 0.9 percent and 0.1 percent, respectively.


Adults Age 18 to 64 Who   For all U.S. adults age 18 to 64, the estimated average annual medical
Used Obesity Drugs Had    and prescription drug spending per adult was higher for those who used
                          an obesity drug than for those who did not use an obesity drug. 74
Higher Average Medical
                          Specifically, the estimated average annual medical expenditures were
and Prescription Drug     $7,575 per adult who used an obesity drug and $4,302 for those who did
Spending Than Those       not, according to AHRQ’s nationally representative estimates from MEPS
Who Did Not               data for 2012 through 2016. Further, the estimated average annual
                          prescription drug expenditures per adult were $2,198 for those who used
                          an obesity drug and $1,111 for those who did not. However, these data
                          do not necessarily indicate that use of obesity drugs leads to higher
                          average annual medical and prescription drug spending.

                          For U.S. adults with obesity, there was not a significant difference
                          between the estimated average annual medical and prescription drug
                          expenditures per adult for those who used an obesity drug and those who
                          did not use an obesity drug. 75 This may be due to the small sample size
                          of 279 adults with obesity who used an obesity drug in the MEPS data.
                          Appendix VIII provides more information on AHRQ’s estimated
                          expenditures for obesity drugs and other medical and prescription drug
                          spending. We did not identify any studies other than AHRQ’s estimates


                          73
                            Cash represents a prescription transaction when a patient pays out of pocket due to
                          reasons such as the drug is not covered by insurance, the patient does not have
                          insurance, or the patient did not present their prescription card; however, the reasons for
                          all cash transactions are not available in the data, according to FDA.
                          74
                            These differences were significant at the 95 percent confidence level. AHRQ’s analysis
                          of MEPS data did not report estimates for adults age 65 and older who used obesity drugs
                          due to inadequate precision for estimates resulting from an insufficient sample size.
                          75
                            For these estimates, obesity was defined as a BMI of 30 or higher. BMI is calculated
                          based on individuals’ height and weight as reported by household respondents in MEPS.
                          As a result, because some individuals may self report higher than actual height and lower
                          than actual weight, calculations of BMI from MEPS data may be lower than actual BMI.




                          Page 29                                                          GAO-19-577 Obesity Drugs
                  from MEPS data that specifically addressed the medical spending for
                  adults who used obesity drugs compared to those who did not.


                  We provided a draft of this report to HHS for review and comment. HHS
Agency Comments   provided technical comments, which we incorporated as appropriate.



                  We are sending copies of this report to the Secretary of Health and
                  Human Services, appropriate congressional committees, and other
                  interested parties. In addition, the report will be available at no charge on
                  the GAO Web site at http://www.gao.gov.

                  If you or your staff have any questions about this report, please contact
                  me at (202) 512-7114 or dickenj@gao.gov. Contact points for our Offices
                  of Congressional Relations and Public Affairs are on the last page of this
                  report. GAO staff who made major contributions to this report are listed in
                  appendix IX.




                  John E. Dicken
                  Director, Health Care




                  Page 30                                                GAO-19-577 Obesity Drugs
List of Committees

The Honorable Charles E. Grassley
Chairman
The Honorable Ron Wyden
Ranking Member
Committee on Finance
United States Senate

The Honorable Lamar Alexander
Chairman
The Honorable Patty Murray
Ranking Member
Committee on Health, Education, Labor, and Pensions
United States Senate

The Honorable Frank Pallone, Jr.
Chairman
The Honorable Greg Walden
Republican Leader
Committee on Energy and Commerce
House of Representatives

The Honorable Richard Neal
Chairman
The Honorable Kevin Brady
Ranking Member
Committee on Ways and Means
House of Representatives




Page 31                                          GAO-19-577 Obesity Drugs
Appendix I: Objectives, Scope, and
                        Appendix I: Objectives, Scope, and
                        Methodology



Methodology

                        The Bipartisan Budget Act of 2018 included a provision for GAO to review
                        the prevalence of obesity and the use of obesity drugs in the Medicare
                        and non-Medicare populations, including spending for and coverage of
                        these drugs. 1 We examined (1) the prevalence of obesity among adults in
                        the United States; (2) what is known about the use of obesity drugs and
                        the individuals who use them; (3) what is known about health insurance
                        coverage of obesity drugs; and (4) what is known about spending on
                        obesity drugs and about medical spending for adults who used obesity
                        drugs compared to those who did not. 2

                        To address our reporting objectives, we examined estimates from federal
                        agencies within the Department of Health and Human Services (HHS),
                        including the Centers for Disease Control and Prevention’s (CDC)
                        estimates from the National Health and Nutrition Examination Survey
                        (NHANES), the Agency for Health Care Research and Quality’s (AHRQ)
                        estimates from the Medical Expenditure Panel Survey (MEPS), and the
                        Food and Drug Administration’s (FDA) estimates from IQVIA and the
                        Sentinel System. We also analyzed Medicare Part D Prescription Drug
                        Event data and Medicaid State Drug Utilization data from the Centers for
                        Medicare & Medicaid Services (CMS). For each data source, we
                        examined the latest available data at the time of our review. In addition,
                        we conducted a literature review; interviewed officials and reviewed
                        documents from stakeholder organizations, federal agencies, insurers,
                        and others; and examined relevant laws and regulations.


National Health and     We examined CDC’s nationally representative estimates from NHANES
Nutrition Examination   of the prevalence of obesity among U.S. adults and use of obesity drugs.
                        NHANES is a cross-sectional survey designed to monitor the health and
Survey
                        nutritional status of the civilian, noninstitutionalized U.S. population. The
                        survey consists of interviews conducted in participants’ homes and
                        standardized physical examinations, including measured height and
                        weight, conducted in mobile examination centers. CDC analyzed data
                        from two 2-year cycles of NHANES (2013 through 2014 and 2015 through
                        2016) for the prevalence of obesity [defined as a body mass index (BMI)

                        1
                        Pub. L. No. 115-123, div. E, tit. III, § 50352, 132 Stat. 64, 212.
                        2
                         For our review, we included the following nine FDA-approved prescription drugs for
                        weight loss: benzphetamine, diethylpropion, phendimetrazine, phentermine,
                        bupropion/naltrexone (Contrave), liraglutide (Saxenda), lorcaserin (Belviq), orlistat
                        (Xenical), and phentermine/topiramate (Qsymia). See appendix II for additional
                        information on the obesity drugs included in our review.




                        Page 32                                                              GAO-19-577 Obesity Drugs
                            Appendix I: Objectives, Scope, and
                            Methodology




                            of 30 or higher] for all adults by age (18 and older, 18 through 64, and 65
                            and older), health insurance coverage, and class of obesity. 3 The
                            insurance categories were mutually exclusive: (1) Medicare, which
                            includes all adults who reported having Medicare, regardless of whether
                            they reporting having another type of health insurance (e.g., private
                            health insurance) in addition to Medicare; (2) private health insurance
                            (excluding individuals with Medicare); (3) Medicaid/public health
                            insurance (excluding Medicare); and (4) uninsured. 4 We also examined
                            CDC’s estimates from NHANES on the prevalence of overweight (defined
                            as a BMI of 25 to <30) among U.S. adults. In addition, we examined
                            CDC’s estimates from NHANES for 2013 through 2016 on adults who
                            took prescription medications for weight loss. 5 NHANES asks participants
                            if they tried to lose weight, and, for those who did, if they took diet pills
                            prescribed by a doctor. CDC’s estimates included the lower and upper
                            bounds of the 95 percent confidence intervals (the interval that would
                            contain the actual population value for 95 percent of the samples
                            NHANES could have drawn).


Medical Expenditure Panel   We examined AHRQ’s nationally representative estimates from MEPS
Survey                      data on the use of and payment sources for obesity drugs. 6 MEPS
                            collects nationally representative data on health care use, expenditures,
                            sources of payment, and insurance coverage for the U.S. civilian,
                            noninstitutionalized population. For this analysis, AHRQ estimated the
                            distribution of payments for obesity drugs using MEPS pooled data for



                            3
                             These estimates are based on an unweighted sample of 11,375 adults and an estimated
                            population of 240.5 million adults. BMI was calculated from measured weight and
                            measured height (weight in kilograms divided by height in meters squared) from the
                            NHANES physical examination. Pregnant women are excluded from this analysis.
                            4
                             The private health insurance category excluded coverage by a single service plan, such
                            as a dental or a vision plan, or a Medigap (Medicare supplement insurance) plan. The
                            Medicaid/public insurance category excludes Medicare and includes Medicaid, military
                            healthcare, Indian Health Service, state-sponsored health plans, and other government
                            programs. According to CDC’s estimates from NHANES data, 46.4 percent of adults with
                            public insurance only had Medicaid. The estimates presented here are internal to CDC’s
                            analysis of NHANES data.
                            5
                             These estimates are based on data from 166 NHANES sample members, who reported
                            that they tried to lose weight and took diet pills prescribed by a doctor.
                            6
                             We define use of obesity drugs as having an outpatient prescription filled or refilled for
                            any of the nine obesity drugs.




                            Page 33                                                            GAO-19-577 Obesity Drugs
        Appendix I: Objectives, Scope, and
        Methodology




        years 2012 through 2016. 7 We also examined AHRQ’s estimates from
        MEPS of annual expenditures for medical care and all prescription
        drugs—for those individuals who used obesity drugs and those who did
        not—and annual expenditures for obesity drugs. 8 AHRQ’s estimates
        included the lower and upper bounds of the 95 percent confidence
        intervals.


IQVIA   We examined FDA’s nationally projected data on the prescriptions
        dispensed for obesity drugs from outpatient retail pharmacies using 2017
        IQVIA™ National Prescription Audit Extended Insights and IQVIA™ Total
        Patient Tracker. 9 IQVIA™ is proprietary data that includes data for
        prescriptions dispensed at approximately 59,900 U.S. outpatient retail
        pharmacies. FDA analyzed IQVIA data and provided aggregated results
        for the nationally estimated number of prescriptions dispensed for the
        nine obesity drugs from U.S. outpatient retail pharmacies, by payment
        method. These patterns may not apply to other settings of care (e.g.,
        mail-order or specialty pharmacies or clinics). In addition, the analysis
        captures data when a prescription was dispensed; it does not indicate
        that the patient took the obesity drug, and it does not indicate if the drug
        was prescribed off label for something other than weight loss.




        7
         Because two of the drugs—liraglutide (Saxenda) and bupropion/naltrexone (Contrave)—
        were approved for the U.S. market in 2014, not all of the obesity drugs were available in
        all of the years included in this analysis. AHRQ’s estimates are based on an unweighted
        sample of 397 MEPS sample members and an estimated average annual population of
        approximately 940,000 adults who were reported to have used obesity drugs. This
        analysis excludes adults age 18 and older with missing BMI data, and women who
        reported that they were pregnant during the reference period of the interview in which they
        reported height and weight.
        8
         Prescription drug expenditures in 2012 to 2015 were inflated to 2016 dollars using the
        Consumer Price Index for Prescription Drugs, available from the Bureau of Labor
        Statistics. Medical expenditures in 2012 to 2015 were inflated to 2016 dollars using the
        Personal Health Care Expenditure Index available from CMS. Prescription drug
        expenditure and medical expenditure estimates are based on an unweighted sample of
        118,615 adults and an estimated average annual population of 233,060,000 adults. BMIs
        calculated in MEPS are based on height and weight as reported by household
        respondents; as a result, fewer individuals are identified as having obesity (with a BMI of
        30 or higher) in MEPS data. AHRQ provided the upper and lower bounds of the 95
        percent confidence intervals that account for the complex survey design of MEPS.
        9
          This analysis included prescriptions dispensed for the obesity drugs; it does not include a
        link between the prescription and the patient’s diagnosis.




        Page 34                                                           GAO-19-577 Obesity Drugs
                          Appendix I: Objectives, Scope, and
                          Methodology




Sentinel System           We examined FDA’s national estimates of prescriptions for obesity drugs
                          dispensed by outpatient pharmacies for new users of obesity drugs (by
                          number of days supplied and by age and gender of patient) from the
                          agency’s Sentinel System. 10 FDA’s Sentinel System uses prescription
                          drug dispensing data from populations with federal or commercial
                          insurance to characterize drug utilization of a large U.S. population with
                          private and public health insurance. FDA examined drug dispensing data
                          from January 1, 2008, through December 31, 2017, from 17 of 18
                          Sentinel data partners, including Medicare, which contributed fee-for-
                          service enrollee data. FDA analyzed dispensings for 267,836 new users
                          of the nine prescription obesity drugs. 11 FDA estimated the duration of the
                          first treatment episode (in days) for patients’ prescription dispensings for
                          any of the nine obesity drugs using a 14-day episode gap—that is, if there
                          were more than 14 days between exhausting the previous dispensing’s
                          days supplied for that prescription and refilling the prescription, then FDA
                          counted it as a new treatment episode. FDA estimated cumulative
                          treatment duration by summing days’ supply of all dispensings of an
                          obesity drug during a patient’s presence in the database, without regard
                          to time between dispensings.


Medicare Part D           For information on the number of claims for obesity drugs that were
Prescription Drug Event   reimbursed, the number of plans that provided reimbursement, and the
                          amount reimbursed for obesity drugs under the Medicare prescription
Data
                          drug program known as Medicare Part D, we analyzed Medicare




                          10
                            Dispensing is the act of delivering a prescription drug to a patient or an intermediary who
                          is responsible for administering the drug. A new user was defined as having no use of any
                          obesity drug in the previous 183 days.
                          11
                            FDA’s analysis included dispensings of Alli, a lower dose of orlistat that is an over-the-
                          counter weight-loss medication that does not require a prescription.

                          Because two of the obesity drugs—lorcaserin (Belviq) and phentermine/topiramate
                          (Qsymia) were approved for the U.S market in 2012, and two of the drugs—liraglutide
                          (Saxenda) and bupropion/naltrexone (Contrave) were approved for the U.S. market in
                          2014, not all of the drugs were available in all of the years included in FDA’s analysis of
                          Sentinel System data. Although these data are not medication administration data, this
                          analysis assumes that patients who obtained multiple dispensings took the dispensed
                          doses of their obesity drugs prior to the last refill. Because prescribers are not required to
                          document the indication, it is possible that some drug dispensings were for indications that
                          are not labeled, according to FDA.




                          Page 35                                                           GAO-19-577 Obesity Drugs
Appendix I: Objectives, Scope, and
Methodology




Prescription Drug Event data from CMS for 2016 and 2017. 12 We
analyzed Medicare Part D plan reimbursements (payments to
pharmacies) and beneficiary spending (the total amount Medicare
beneficiaries paid out of pocket as copayments or deductibles) for the
nine obesity drugs for claims that CMS’s data coded as reimbursed as a
supplemental drug under enhanced alternative coverage. 13 We excluded
1,787 claims in 2016 and 1,775 claims in 2017 for one obesity drug,
orlistat (Xenical), that were listed in CMS’s data as covered under
Medicare Part D (and were not coded as a supplemental drug under
enhanced alternative coverage). According to CMS officials, orlistat has
off-label indications including diabetes and hyperlipidemia, and when
orlistat is used for these indications the drug would be covered under
Medicare Part D, and the Medicare Part D plan is responsible for
ensuring it is dispensed appropriately per Medicare Part D policy. We
also excluded 25 claims in 2016 and 26 claims in 2017 for prescription
obesity drugs listed as over-the-counter in the prescription drug event
data because, according to CMS, these appear to be outliers. Because
our analysis was limited to those instances in which a Medicare Part D
plan reimbursed for an obesity drug as a supplemental drug under
enhanced alternative coverage, the number of Medicare Part D plans that
provided coverage for obesity drugs could be higher. For example, some
plans may have covered obesity drugs, but none of the beneficiaries
enrolled in these plans filled a prescription for such a drug.



12
  Medicare Part D provides a voluntary outpatient prescription drug benefit for Medicare
beneficiaries. In 2018, there were about 44 million enrollees covered by a Medicare Part D
plan. CMS’s Prescription Drug Event data contains cost and payment data submitted by
plans to CMS for covered Part D drugs to enable CMS to make payments to plans and
administer the Part D benefit. Our analysis was limited to those instances in which a
Medicare Part D plan reimbursed for an obesity drug as a supplemental drug under
enhanced alternative coverage, and the actual number of Medicare Part D plans that
provided coverage for obesity drugs could be higher. We did not find comprehensive data
on the number of Medicare Part D plans that provided coverage or the number of
beneficiaries with coverage for obesity drugs.
13
  Beneficiary spending refers to how much a beneficiary’s responsibility is for a given
claim. The amount includes how much a beneficiary pays for a drug that is not reimbursed
by a third party, the amount a patient’s liability is reduced due to other benefits such as the
Veterans Administration and TRICARE, the amount a plan reduced the patient’s liability
due a beneficiary’s low-income cost sharing subsidy amount and all other qualified third-
party payments on behalf of the beneficiary, which are referred to as the Other True Out-
of-Pocket. The low-income cost sharing subsidy amount and Other True Out-of-Pocket
amounts are zero for obesity drugs because they are covered under an enhanced benefit,
not as Part D drugs.




Page 36                                                            GAO-19-577 Obesity Drugs
                               Appendix I: Objectives, Scope, and
                               Methodology




Medicaid State Drug            For information on obesity drugs reimbursed by state Medicaid programs
Utilization Data               or Medicaid managed care programs within those states, we analyzed
                               CMS’s Medicaid State Drug Utilization data for 2016 and 2017. 14 We
                               analyzed the data to estimate the number of prescriptions reimbursed and
                               total Medicaid state and federal spending—that is, the Medicaid amount
                               reimbursed (state and federal reimbursement, including dispensing
                               fees)—for the nine obesity drugs. 15 These amounts do not include all
                               Medicaid spending for obesity drugs because managed care
                               organizations can be paid for the drugs as part of their capitated payment
                               for all Medicaid services, they are not reimbursed on a per-drug basis,
                               and their payments are not recorded in CMS’s Medicaid State Drug
                               Utilization data. Because our analysis was limited to those instances in
                               which Medicaid reimbursed for an obesity drug, the number of states in
                               which state Medicaid programs or Medicaid managed care plans provided
                               coverage for obesity drugs could be higher. For example, a state could
                               have provided coverage for obesity drugs, but no beneficiaries in that
                               state filled a prescription for an obesity drug.


Interviews with Officials in   We obtained information and reviewed studies from officials from eight
Stakeholder                    stakeholder organizations (representing medical associations and
                               advocacy groups for obesity research and treatment) on the use of
Organizations, Federal
                               obesity drugs and guidelines for using obesity drugs and to obtain their
Agencies, Insurers, and        perspectives on what physicians and other health care providers take into
Others                         consideration when prescribing these drugs, among other things. 16 These
                               stakeholders were selected because of their medical or scientific
                               expertise, relevant publications, or familiarity with the treatment of obesity
                               and obesity drugs.

                               14
                                As of March 2019, there were 65.9 million enrollees covered by Medicaid.
                               15
                                 The Medicaid State Drug Utilization data does not include information on the indication
                               or indications for which these drugs were prescribed. Therefore, we cannot determine
                               whether these obesity drugs were prescribed off label. A 2018 study compared 2015
                               Medicaid State Drug Utilization data and publicly available 2016-2017 Medicaid program
                               documents, and found that more states reimbursed for obesity drugs than indicated
                               coverage in the program documents. See N. Jannah, J. Hild, C. Gallagher, and W. Dietz,
                               “Coverage for Obesity Prevention and Treatment Services: Analysis of Medicaid and State
                               Employee Health Insurance Programs,” Obesity, vol. 26 (2018): 1834-1840.
                               16
                                 The stakeholder organizations we contacted were the American Academy of Family
                               Physicians, American Association of Clinical Endocrinologists, American College of
                               Cardiology, American Heart Association, Obesity Action Coalition, Obesity Medicine
                               Association, The Obesity Society, and the Strategies to Overcome and Prevent (STOP)
                               Obesity Alliance.




                               Page 37                                                        GAO-19-577 Obesity Drugs
                    Appendix I: Objectives, Scope, and
                    Methodology




                    We also reviewed data and documents and interviewed officials from
                    HHS agencies: CDC, FDA, AHRQ, CMS, and the National Institutes of
                    Health. In addition, we reviewed guidance documents and obtained
                    information from the Office of Personnel Management, which administers
                    the Federal Employees Health Benefits program (FEHBP). FEHBP is the
                    largest employer-sponsored health insurance program in the United
                    States, providing health insurance coverage to about 8 million federal
                    employees, retirees, and their dependents in 2016 through contracts with
                    private health insurance plans.

                    We obtained information about the health insurance coverage of obesity
                    drugs from officials from the three largest pharmacy benefit managers,
                    four large insurers, and two organizations knowledgeable about
                    prescription drug benefits for employer-sponsored health plans. 17 We also
                    reviewed drug formularies for selected private health insurance plans,
                    including FEHBP plans, to determine if any of the nine obesity drugs were
                    included. 18


Literature Review   We conducted a literature review of relevant peer-reviewed studies
                    published from January 2012 through January 2019. We identified studies
                    through a search of bibliographic databases, including ProQuest, Scopus,
                    MEDLINE, and International Pharmaceutical Abstracts, using terms such
                    as “obesity,” “weight loss,” and “prescriptions.” Of the 765 citations we
                    identified, we reviewed 220 full studies, which we examined for
                    information related to the use of obesity drugs and individuals who use
                    17
                      Pharmacy benefit managers administer prescription drug programs for health plans,
                    including commercial health plans and Medicare Part D plans. The pharmacy benefit
                    managers that we contacted were Express Scripts, Optum RX, and CVS Caremark, all of
                    which comprise about 70 percent of the pharmacy benefit manager market. We selected
                    the four large insurers—Blue Cross Blue Shield, United Healthcare, Kaiser Permanente,
                    and Aetna—based on the criteria of the insurers covering more than 1 million lives and
                    operating in multiple states. The other two organizations we contacted were Willis Towers
                    Watson, an employer benefit consultant, and the National Business Group on Health, an
                    organization that represents over 430 large employers.
                    18
                      A formulary is a list of prescription drugs covered by a prescription drug plan or another
                    insurance plan offering prescription drug benefits. A plan may cover drugs that are not
                    included on the formulary if, for example, a patient has previously used other prescription
                    drugs or therapies and the health care provider determines the prescription drug not
                    included in the formulary is medically necessary for the patient. We reviewed formularies
                    from the three largest FEHBP plans, including the Blue Cross Blue Shield’s Basic and
                    Standard formularies, Kaiser Permanente’s eight regional formularies, and Government
                    Employees Health Association preferred and specialty drug lists. See GAO-18-52 for more
                    information on FEHBP plans.




                    Page 38                                                          GAO-19-577 Obesity Drugs
Appendix I: Objectives, Scope, and
Methodology




them, coverage of obesity drugs, and spending for obesity drugs for
individuals who used them compared to those who did not. We
determined 19 studies were relevant to the use of obesity drugs and 1
study was relevant to coverage of obesity drugs. Our literature review
focused on studies with a U.S.-based, adult population (age 18 and
older); we excluded studies related to childhood obesity and studies on
animals. We also examined available information on the clinical trials
conducted prior to FDA approval of the prescription obesity drugs for the
U.S. market, including 64 studies from our literature review that
summarized one or more of the clinical trials. We also identified 17
additional studies in our literature review that provided relevant
background information.

Additionally, we reviewed five studies provided by stakeholder
organizations (in addition to the studies we had identified in our literature
review) that we determined were relevant to our research objectives, as
well as guidelines for the use of obesity drugs in obesity treatment. 19

To determine the reliability of the data we used for all four objectives—
CDC’s estimates from NHANES, AHRQ’s estimates from MEPS, FDA’s
data from IQVIA and the Sentinel System, and CMS’s Medicare Part D
Prescription Drug Event data and Medicaid State Drug Utilization data—
we reviewed documentation on data collection processes and discussed
limitations of the data with the relevant federal agency officials. In
addition, we conducted data reliability checks on the data, when
appropriate. We determined the data used in this report were sufficiently
reliable for our purposes.

We conducted this performance audit from April 2018 to August 2019 in
accordance with generally accepted government auditing standards.
Those standards require that we plan and perform the audit to obtain
sufficient, appropriate evidence to provide a reasonable basis for our

19
  These guidelines include: (1) American College of Cardiology and American Heart
Association Task Force on Practice Guidelines and The Obesity Society, “A Guideline for
the Management of Overweight and Obesity in Adults,” (2013); (2) C.M. Apovian et al.,
“Pharmacological Management of Obesity: An Endocrine Society Clinical Practice
Guideline,” Journal of Clinical Endocrinology and Metabolism, vol. 100, no. 2, (2015): 342-
362; (3) W.T. Garvey et al., “American Association of Clinical Endocrinologists and
American College of Endocrinology Comprehensive Clinical Practice Guidelines for
Medical Care of Patients with Obesity,” Endocrine Practice, vol. 22, supp. 3, (2016): 1-
203; and (4) Obesity Medicine Association, Obesity Algorithm 2017-2018, (Denver, CO:
June 2018).




Page 39                                                         GAO-19-577 Obesity Drugs
Appendix I: Objectives, Scope, and
Methodology




findings and conclusions based on our audit objectives. We believe that
the evidence obtained provides a reasonable basis for our findings and
conclusions based on our audit objectives.




Page 40                                             GAO-19-577 Obesity Drugs
Appendix II: List of FDA-Approved
                                                               Appendix II: List of FDA-Approved Prescription
                                                               Obesity Drugs



Prescription Obesity Drugs

                                                               Table 4 provides additional information on the nine prescription drugs
                                                               approved by the Food and Drug Administration (FDA) to treat obesity that
                                                               we included in our review.

Table 4: FDA-Approved Prescription Obesity Drugs, as of June 2019

 Generic name                                        Brand name(s)                       Year approved           Approved use           Approved for
                                                                                                                                        adults/children
                                                                                                            a                 b
 Phentermine                                         Adipex-P and                                    1959        Short-term             Age 16 and older
                                                     Lomaira
                                                           c
 Diethylpropion                                      n/a                                             1959        Short-term             Age 16 and older
                                                           c
 Benzphetamine                                       n/a                                             1960        Short-term             Age 12 and older
                                                           c
 Phendimetrazine                                     n/a                                             1961        Short-term             Age 17 and older
 Orlistat                                            Xenical                                         1999        Long-term              Age 12 and older
 Lorcaserin                                          Belviq                                          2012        Long-term              Adults (18 and older)
 Phentermine + Topiramate                            Qsymia                                          2012        Long-term              Adults (18 and older)
 Liraglutide                                         Saxenda                                         2014        Long-term              Adults (18 and older)
 Bupropion + Naltrexone                              Contrave                                        2014        Long-term              Adults (18 and older)
Source: Food and Drug Administration (FDA). | GAO-19-577

                                                               Notes: Two additional prescription drugs approved by FDA for weight loss—methamphetamine and
                                                               amphetamine—were not recommended for use by any medical society guidelines for the treatment of
                                                               obesity. Methamphetamine is a controlled substance with high abuse potential and is thus rarely used
                                                               for obesity treatment, according to an official from the National Institutes of Health’s Office of Obesity
                                                               Research. In addition, amphetamines are not used in the treatment of obesity, according to officials
                                                               from a scientific membership organization focused on obesity issues and officials from medical
                                                               associations with clinician members who treat obesity. We therefore excluded methamphetamine and
                                                               amphetamine from the list of prescription obesity drugs. Additionally, we excluded Victoza, which has
                                                               the same active ingredient as Saxenda (liraglutide), because it is indicated for diabetes. We also
                                                               excluded Alli, a lower dose of orlistat, because it is an over-the-counter drug that does not require a
                                                               prescription.
                                                               a
                                                               Phentermine was initially approved by FDA in 1959; however, it has since been approved and
                                                               marketed under different brand names in different dosage levels.
                                                               b
                                                                   Short-term use is generally considered to be use of the drug for about 12 weeks or less.
                                                               c
                                                                According to FDA, these drugs are not marketed under a brand name, they are only available as a
                                                               generic drug—that is, a drug that is created to be the same as an already marketed brand-name drug
                                                               in dosage form, safety, strength, route of administration, quality, performance characteristics, and
                                                               intended use.




                                                               Page 41                                                                    GAO-19-577 Obesity Drugs
Appendix III: Prevalence of Obesity and
              Appendix III: Prevalence of Obesity and
              Overweight among U.S. Adults



Overweight among U.S. Adults

              This appendix presents national estimates of the prevalence of obesity
              among U.S. adults age 18 and older, based on the Centers for Disease
              Control and Prevention’s (CDC) estimates from the National Health and
              Nutrition Examination Survey (NHANES) for 2013 through 2016. 1 It
              presents the estimates and the ranges for the 95 percent confidence
              intervals for prevalence of obesity by age and class of obesity (see table
              5), and by insurance coverage and class of obesity (see table 6). 2 It also
              presents national estimates of the prevalence of overweight (defined as a
              body mass index of 25 to <30) among U.S. adults, by age and insurance
              coverage (see table 7).




              1
               Data from two 2-year cycles of NHANES (2013 through 2014 and 2015 through 2016)
              were used for this analysis. NHANES is a cross-sectional survey designed to monitor the
              health and nutritional status of the civilian, noninstitutionalized U.S. population. The survey
              consists of interviews conducted in participants’ homes and standardized physical
              examinations, including measured height and weight, in mobile examination centers.
              2
               The 95 percent confidence interval is the interval that would contain the actual population
              value for 95 percent of the samples NHANES could have drawn. As a result, we are 95
              percent confident that each of the confidence intervals based on NHANES includes the
              true values in the population.




              Page 42                                                            GAO-19-577 Obesity Drugs
                                                                Appendix III: Prevalence of Obesity and
                                                                Overweight among U.S. Adults




Table 5: Prevalence of Obesity among U.S. Adults, 2013–2016, by Age and Class of Obesity

(percentage)

                                                                          Estimate                                       95 percent confidence interval
                                                                                                                           Lower bound                                 Upper bound
 All U.S. adults                                                                 38.4                                                    36.5                                   40.2
                                       a
 Class 1 (BMI of 30 to <35)                                                      21.0                                                    19.8                                   22.2
 Class 2 (BMI of 35 to <40)                                                        9.8                                                     9.0                                  10.6
 Class 3 (BMI ≥40)                                                                 7.6                                                     6.7                                   8.5
 Age 18–64                                                                       38.5                                                    36.3                                   40.8
 Class 1 (BMI of 30 to <35)                                                      20.4                                                    18.9                                   21.9
 Class 2 (BMI of 35 to <40)                                                        9.7                                                     8.8                                  10.7
 Class 3 (BMI ≥40)                                                                 8.4                                                     7.4                                   9.6
 Age 65 and older                                                                37.6                                                    34.7                                   40.5
 Class 1 (BMI of 30 to <35)                                                      23.6                                                    21.2                                   26.1
 Class 2 (BMI of 35 to <40)                                                      10.0                                                      8.3                                  11.9
 Class 3 (BMI ≥40)                                                                 4.0                                                     2.8                                   5.6
Source: Centers for Disease Control and Prevention’s estimates from the National Health and Nutrition Examination Survey (NHANES), 2013–2016. | GAO-19-577

                                                                Notes: Adults are defined as age 18 and older. Pregnant women are excluded from this analysis.
                                                                These estimates are based on an unweighted sample of 11,375 adults and an estimated population
                                                                of 240.5 million adults.
                                                                a
                                                                 Body mass index (BMI) is calculated from measured weight and measured height (weight in
                                                                kilograms divided by height in meters squared) from the NHANES physical examination.




Table 6: Prevalence of Obesity among U.S. Adults, 2013–2016, by Insurance Coverage and Class of Obesity

(percentage)

                                                                          Estimate                                       95 percent confidence interval
                                                                                                                           Lower bound                                 Upper bound
                             a
 Medicare coverage                                                               39.5                                                    36.5                                   42.5
                                       b
 Class 1 (BMI of 30 to <35)                                                      23.9                                                    21.3                                   26.6
 Class 2 (BMI of 35 to <40)                                                        9.7                                                     8.2                                  11.4
 Class 3 (BMI ≥40)                                                                 5.8                                                     4.4                                   7.6
                         c
 Private insurance                                                               37.0                                                    34.5                                   39.7
 Class 1 (BMI of 30 to <35)                                                      20.3                                                    18.3                                   22.5
 Class 2 (BMI of 35 to <40)                                                        9.4                                                     8.2                                  10.7
 Class 3 (BMI ≥40)                                                                 7.3                                                     6.4                                   8.3
 Medicaid and other public health                                                42.1                                                    36.8                                   45.7
                                d
 insurance (excluding Medicare)




                                                                Page 43                                                                                      GAO-19-577 Obesity Drugs
                                                               Appendix III: Prevalence of Obesity and
                                                               Overweight among U.S. Adults




                                                                          Estimate                                       95 percent confidence interval
                                                                                                                           Lower bound                              Upper bound
 Class 1 (BMI of 30 to <35)                                                      20.5                                                    17.8                                23.5
 Class 2 (BMI of 35 to <40)                                                      10.8                                                     9.1                                12.7
 Class 3 (BMI ≥40)                                                               10.8                                                     8.5                                13.4
 Uninsured                                                                       38.0                                                    34.5                                41.7
 Class 1 (BMI of 30 to <35)                                                      19.3                                                    16.9                                21.9
 Class 2 (BMI of 35 to <40)                                                      10.3                                                     8.6                                12.1
 Class 3 (BMI ≥40)                                                                 8.4                                                    6.6                                10.6
Source: Centers for Disease Control and Prevention’s (CDC) estimates from the National Health and Nutrition Examination Survey (NHANES), 2013–2016. | GAO-19-577

                                                               Notes: All of the health insurance coverage categories are mutually exclusive. Adults are defined as
                                                               age 18 and older. Pregnant women are excluded from this analysis.
                                                               These estimates are based on an unweighted sample of 11,375 adults and an estimated population
                                                               of 240.5 million adults.
                                                               a
                                                                Medicare estimates include all adults who reported having Medicare, including adults who also
                                                               reported having private health insurance or other public health insurance; if they reported having
                                                               Medicare, they were counted in the Medicare category and not in the private insurance or other public
                                                               insurance category. An estimated 87 percent of adults covered by Medicare were 65 and older, while
                                                               an estimated 13 percent were age 18 through 64.
                                                               b
                                                                Body mass index (BMI) is calculated from measured weight and measured height (weight in
                                                               kilograms divided by height in meters squared) from the NHANES physical examination.
                                                               c
                                                                Adults who reported having private health insurance and another type of insurance, such as
                                                               Medicaid, were counted in the private insurance category except if they had Medicare—in which case
                                                               they were counted in the Medicare category. This excluded coverage by a single service plan, such
                                                               as a dental or a vision plan, or a Medigap (Medicare supplement insurance) plan.
                                                               d
                                                                Medicaid accounted for about 46.4 percent of adults in the Medicaid/other public insurance category
                                                               in the NHANES analysis, according to CDC. The category also includes military healthcare, Indian
                                                               Health Service, state-sponsored health plans, and other government programs. This excludes
                                                               Medicare coverage.




                                                               Page 44                                                                                    GAO-19-577 Obesity Drugs
                                                               Appendix III: Prevalence of Obesity and
                                                               Overweight among U.S. Adults




Table 7: Prevalence of Overweight among U.S. Adults, 2013–2016, by Age and Insurance Coverage

(percentage)

                                                                          Estimate                                       95 percent confidence interval
                                                                                                                           Lower bound                              Upper bound
 All U.S. adults                                                                 32.0                                                   31.0                                 33.0
 Age 18–64                                                                       30.8                                                   29.4                                 32.2
 Age 65 and older                                                                36.9                                                   34.6                                 39.4
                             a
 Medicare coverage                                                               35.8                                                   33.6                                 38.0
                         b
 Private insurance                                                               31.5                                                   29.8                                 33.1
 Medicaid and other public health                                                26.7                                                   24.4                                 29.1
                                c
 insurance (excluding Medicare)
 Uninsured                                                                       32.9                                                   29.4                                 36.6
Source: Centers for Disease Control and Prevention’s (CDC) estimates from the National Health and Nutrition Examination Survey (NHANES), 2013–2016. | GAO-19-577

                                                               Notes: Overweight is defined as a body mass index (BMI) of 25 to <30. BMI is calculated from
                                                               measured weight and measured height (weight in kilograms divided by height in meters squared)
                                                               from the NHANES physical examination.
                                                               All of the health insurance coverage categories are mutually exclusive.
                                                               Adults are defined as age 18 and older. Pregnant women are excluded from this analysis.
                                                               These estimates are based on an unweighted sample of 11,375 adults and an estimated population
                                                               of 240.5 million adults.
                                                               a
                                                                Medicare estimates include all adults who reported having Medicare, including adults who also
                                                               reported having private health insurance or other public health insurance; if they reported having
                                                               Medicare, they were counted in the Medicare category and not in the private insurance or other public
                                                               insurance category. An estimated 87 percent of adults covered by Medicare were 65 and older, while
                                                               an estimated 13 percent were age 18 through 64.
                                                               b
                                                                Adults who reported having private health insurance and another type of insurance, such as
                                                               Medicaid, were counted in the private insurance category except if they had Medicare—in which case
                                                               they were counted in the Medicare category. This excluded coverage by a single service plan, such
                                                               as a dental or a vision plan, or a Medigap (Medicare supplement insurance) plan.
                                                               c
                                                                Medicaid accounted for about 46.4 percent of adults in the Medicaid/other public insurance category
                                                               in the NHANES analysis, according to CDC. The category also includes military healthcare, Indian
                                                               Health Service, state-sponsored health plans, and other government programs. This excludes
                                                               Medicare coverage.




                                                               Page 45                                                                                    GAO-19-577 Obesity Drugs
Appendix IV: List of Selected Studies
                                             Appendix IV: List of Selected Studies
                                             Reviewed



Reviewed

                                             Table 8 is a list of selected studies, categorized by specific topic area,
                                             that we reviewed that pertain to our research objectives, including
                                             information related to the use of obesity drugs and individuals who use
                                             them, physician considerations in prescribing obesity drugs, and health
                                             insurance coverage of obesity drugs. We identified these studies either
                                             through our literature review of peer-reviewed studies published from
                                             January 2012 through January 2019 or from one of the stakeholder
                                             organizations we contacted.

Table 8: List of Selected Studies Pertaining to Our Research Objectives, by Topic Area

Topic area                  Study citation
Use of obesity drugs        A.C. Del Re, S.M. Frayne, and A.H.S Harris, “Antiobesity Medication Use Across the Veterans Health
among U.S. adults           Administration: Patient-Level Predictors of Receipt,” Obesity, vol. 22 (2014): 1968-1972.
                            C. Hampp, E.M. Kang, and V. Borders-Hemphill, “Use of Prescription Antiobesity Drugs in the U.S.,”
                                                                          a
                            Pharmacotherapy, vol. 33 (2013): 1299-1307.
                            T.P. Semla, C. Ruser, C.B. Good, S.Z. Yanovski, D. Ames, L.A. Copeland, C. Billington, U.I. Ferguson,
                            L.J. Aronne, T.A. Wadden, W.T. Garvey, C.M. Apovian, and D. Atkins, “Pharmacotherapy for Weight
                            Management in the VHA,” Journal of General Internal Medicine, vol. 32 (2017): 70-73.
                            C.E. Thomas, E.A. Mauer, A.P. Shukla, S. Rathi, L.J. Aronne. “Low Adoption of Weight Loss Medications:
                            A Comparison of Prescribing Patterns of Anti-Obesity Pharmacotherapy and SGLT2s.” Obesity, vol. 24
                            (2016): 1955-1961.
                            D.D. Thomas, M.E. Waring, O. Ameli, J.I. Reisman, and V.G. Vimalananda, “Patient Characteristics
                            Associated with Receipt of Prescription Weight-Management Medications Among Veterans Participating
                            in MOVE!” Obesity, vol. 27, no. 7 (2019): 1168-1176.
                            S. Zhang, S. Manne, J. Lin, and J. Yang, “Characteristics of Patients Potentially Eligible for
                            Pharmacotherapy for Weight Loss in Primary Care Practice in the United States,” Obesity Science &
                            Practice, vol. 2, no. 2 (2016): 104-144.
Physician considerations    T.A. Glauser, N. Roepke, B. Stevenin, A.M. Dubois, and S.M. Ahn, “Physician Knowledge About and
and attitudes in            Perceptions of Obesity Management,” Obesity Research & Clinical Practice, vol. 9 (2015): 573-583.
prescribing obesity drugs   B. Granara and J. Laurent, “Provider Attitudes and Practice Patterns of Obesity Management with
                            Pharmacotherapy,” Journal of the American Association of Nurse Practitioners, (2017): 543-550.
                            S. Iwamoto, D. Saxon, A. Tsai, E. Leister, R. Speer, H. Heyn, E. Kealey, E. Juarez-Colunga, K. Gudzune,
                            S. Bleich, J. Clark, and D. Bessesen, “Effects of Education and Experience on Primary Care Providers’
                            Perspectives of Obesity Treatments during a Pragmatic Trial,” Obesity, vol. 26 (2018): 1532-1538.
                            L.M. Kaplan, A. Golden, K. Jinnett, R.L. Kolotkin, T.K. Kyle, M. Look, J. Nadglowski, P.M. O’Neil, T. Parry,
                            K.J. Tomaszewski, B. Stevenin, S.K. Lilleøre, and N.V. Dhurandhar, “Perceptions of Barriers to Effective
                            Obesity Care: Results from the National ACTION Study,” Obesity, vol. 26, no. 1 (2018): 61-69.
                            C. Petrin, S. Kahan, M. Turner, C. Gallagher, and W.H. Dietz, “Current Practices of Obesity
                            Pharmacotherapy, Bariatric Surgery Referral and Coding for Counselling by Healthcare Professionals,”
                            Obesity Science & Practice, vol. 2, no. 3 (2016).
                            R. Simon and S.W. Lahiri, “Provider Practice Habits and Barriers to Care in Obesity Management in a
                            Large Multicenter Health System,” Endocrine Practice, vol. 24, no. 4 (2018): 321-328.
                            M. Turner, N. Jannah, S. Kahan, C. Gallagher, and W. Dietz, “Current Knowledge of Obesity Treatment
                            Guidelines by Health Care Professionals,” Obesity, vol. 26, no. 4 (2018): 665-671.




                                             Page 46                                                         GAO-19-577 Obesity Drugs
                                             Appendix IV: List of Selected Studies
                                             Reviewed




Topic area                  Study citation
Use of obesity drugs in     A.G. Tsai, E. Raube, J. Conrad, D.H. Bessesen, and J.M. Rozwadowski, “A Pilot Randomized Trial
conjunction with other      Comparing a Commercial Weight Loss Program with a Clinic-Based Intervention for Weight Loss,”
items or services (e.g.,    Journal of Primary Care & Community Health, vol. 3, no. 4 (2012): 251–255.
behavioral counseling)      T.A. Wadden, O.A. Walsh, R.I. Berkowitz, A.M. Chao, N. Alamuddin, K. Gruber, S. Leonard, K. Mugler, Z.
                            Bakizada, and J. Shaw Tronieri, “Intensive Behavioral Therapy for Obesity Combined with Liraglutide 3.0
                            mg: A Randomized Controlled Trial,” Obesity, vol. 27 (2019): 75-86.
Adherence to using the      R. Ganguly, Y. Tian, S.X. Kong, M. Hersloev, T. Hobbs, B.G. Smolarz, A. Ramasamy, C.L. Haase, and
prescribed obesity drug     W. Weng, “Persistence of Newer Anti-Obesity Medications in a Real-World Setting,” Diabetes Research
                            and Clinical Practice, vol. 143 (2018): 348-356.
                            T. R. Grabarczyk, “Observational Comparative Effectiveness of Pharmaceutical Treatments for Obesity
                            within the Veterans Health Administration,” Pharmacotherapy, vol. 38, no. 1 (2018): 19-28.
                            C. Hampp, E.M. Kang, and V. Borders-Hemphill, “Use of Prescription Antiobesity Drugs in the U.S.,”
                            Pharmacotherapy, vol. 33 (2013): 1299-1307.
Maintaining weight loss     E.A. Bohula, S.D. Wiviott, D.K. McGuire, S.E. Inzucchi, J. Kuder, K.A. Im, C.L. Fanola, A. Qamar, C.
over time by individuals    Brown, A. Budaj, A. Garcia‑Castillo, M. Gupta, L.A. Leiter, N.J. Weissman, H.D. White, T. Patel, B.
who have used obesity       Francis, W. Miao, C. Perdomo, S. Dhadda, M.P. Bonaca, C.T. Ruff, A.C. Keech, S.R. Smith, M.S.
drugs                       Sabatine, and B.M. Scirica, “Cardiovascular Safety of Lorcaserin in Overweight or Obese Patients,” The
                                                                                                   b
                            New England Journal of Medicine, vol. 379, no. 12 (2018): 1107-1117.
                            K. Fujioka, P.M. O’Neil, M. Davies, F. Greenway, D.C.W. Lau, B. Claudius, T.V. Skjøth, C.B. Jensen, and
                            J.P.H. Wilding, “Early Weight Loss with Liraglutide 3.0 mg Predicts 1-Year Weight Loss and is Associated
                            with Improvements in Clinical Markers,” Obesity, vol. 24, no. 11 (2016): 2278-2288.
                            K. Fujioka, R. Plodkowski, P.M. O’Neil, K. Gilder, B. Walsh, and F.L. Greenway, “The Relationship
                            between Early Weight Loss and Weight Loss at 1 Year with Naltrexone ER/Bupropion ER Combination
                            Therapy,” International Journal of Obesity, vol. 40 (2016): 1369-1375.
                            E.S. LeBlanc, C.D. Patnode, E.M. Webber, N. Redmond, M. Rushkin, and E.A. O’Connor, “Behavioral
                            and Pharmacotherapy Weight Loss Interventions to Prevent Obesity-Related Morbidity and Mortality in
                            Adults: Updated Evidence Report and Systematic Review for the U.S. Preventive Services Task Force,”
                            JAMA, vol. 320, no. 11 (2018): 1172-1191.
                            K.H. Lewis, H. Fischer, J. Ard, L. Barton, D.H. Bessesen, M.F. Daley, J. Desai, S. Fitzpatrick, M. Horberg,
                            C. Koebnick, C. Oshiro, A. Yamamoto, D.R. Young, and D.E. Arterburn, “Safety and Effectiveness of
                            Longer-Term Phentermine Use: Clinical Outcomes from an Electronic Health Record Cohort,” Obesity,
                            vol. 27, no. 4 (2019): 591-602.
                            S.R. Smith, P.M. O’Neil, A. Astrup, N. Finer, M. Sanchez-Kam, K. Fraher, R. Fain, and W.R. Shanahan,
                            “Early Weight Loss While on Lorcaserin, Diet and Exercise as a Predictor of Week 52 Weight-Loss
                            Outcomes,” Obesity, vol. 22, no. 10 (2014): 2137–2146.
                            J.S. Tronieri, T.A. Wadden, R.I. Berkowitz, A.M. Chao, R.L. Pearl, N. Alamuddin, S.M. Leonard, R.
                            Carvajal, Z.M. Bakizada, E. Pinkasavage, K.A. Gruber, O.A. Walsh, and N. Alfaris, “A Randomized Trial
                            of Lorcaserin and Lifestyle Counseling for Maintaining Weight Loss Achieved with a Low-Calorie Diet,”
                            Obesity, vol. 26 (2018): 299-309.
Health insurance coverage G. Gomez and F.C. Stanford, “US Health Policy and Prescription Drug Coverage of FDA-approved
of obesity drugs          Medications for the Treatment of Obesity,” International Journal of Obesity, vol. 42 (2018): 495-500.
Source: GAO. | GAO-19-577
                                             a
                                              This study is also listed in the adherence to using the prescribed obesity drug section because it also
                                             examines duration of use for select obesity drugs, in addition to the use of obesity drugs.
                                             b
                                              While this study was conducted in eight countries and is not an entirely U.S.-based study, we
                                             included it because of the large number of participants (12,000 patients with obesity or who were
                                             overweight), the length of the study (median follow-up period of 3.3 years), and because it was a
                                             recent study (2018). Further, although the primary outcome of this study was to assess the




                                             Page 47                                                                  GAO-19-577 Obesity Drugs
Appendix IV: List of Selected Studies
Reviewed




cardiovascular safety of lorcaserin, the study also assessed the effect of lorcaserin on weight through
40 months of follow up, which we determined was relevant to maintaining weight loss over time.




Page 48                                                                  GAO-19-577 Obesity Drugs
Appendix V: Estimates of New Adult Users of
              Appendix V: Estimates of New Adult Users of
              Obesity Drugs, 2008-2017



Obesity Drugs, 2008-2017

              This appendix presents estimates of prescriptions dispensed for new
              adult users of obesity drugs by duration of use and by age and gender,
              using data from the Food and Drug Administration’s (FDA) Sentinel
              System from 2008 through 2017. 1 Of the 267,836 new users of obesity
              drugs included in this analysis, the first treatment episode did not exceed
              30 days in about 54 percent of patients and exceeded 90 days in about
              22 percent of patients. Cumulatively, about 42 percent of patients who
              used any of the obesity drugs did so for more than 90 days across
              treatment episodes. (See table 9.) Overall, most new users of obesity
              drugs were female (82.2 percent) and under age 65 (91.7 percent). (See
              table 10.) Phentermine and bupropion/naltrexone (Contrave) were the
              most commonly used obesity drugs in FDA’s Sentinel System analysis.




              1
                Dispensing is the act of delivering a prescription drug to a patient or an intermediary who
              is responsible for administering the drug. FDA’s Sentinel System uses prescription drug
              dispensing data to characterize drug utilization on a large U.S. population with private and
              public health insurance. A new user was defined as having no use of any obesity drug in
              the previous 183 days. FDA examined drug dispensing data for new users of obesity
              drugs from January 1, 2008, through December 31, 2017, from 17 of 18 Sentinel data
              partners, including Medicare, which contributed fee-for-service enrollee data. FDA
              analyzed dispensings of the nine prescription obesity drugs: benzphetamine,
              diethylpropion, phendimetrazine, phentermine, bupropion/naltrexone (Contrave),
              liraglutide (Saxenda), lorcaserin (Belviq), orlistat (Xenical), and phentermine/topiramate
              (Qsymia). In addition, FDA’s analysis included dispensings of Alli, a lower dose of orlistat
              that is an over-the-counter weight-loss medication that does not require a prescription.
              Because two of the drugs—lorcaserin (Belviq) and phentermine/topiramate (Qsymia) were
              approved for the U.S market in 2012, and two of the drugs—liraglutide (Saxenda) and
              bupropion/naltrexone (Contrave)—were approved for the U.S. market in 2014, not all of
              the drugs were available in all of the years included in FDA’s analysis. The Sentinel
              System only contains data from populations with federal or commercial insurance.




              Page 49                                                           GAO-19-577 Obesity Drugs
Appendix V: Estimates of New Adult Users of
Obesity Drugs, 2008-2017




Table 9: Duration of Use for New Adult Users of Obesity Drugs, 2008-2017

    Duration of Number of new adult users (percent)                            Total     Median                Average
    use         1-30     31-60   61-90      91 or                              number duration                 duration
                days     days    days       more                               of new    (days)                (days)
                                            days                               users
                                                                               (percent)
    First             144,563         26,939        38,551         57,783         267,836               30.0        69.1
    treatment
                        (53.9)         (10.1)        (14.4)         (21.6)         (100.0)
    episode
             a
    duration
    Cumulative          83,994        37,772        32,706        113,364         267,836               72.0       135.1
    treatment
             b           (31.4)        (14.1)        (12.2)         (42.3)         (100.0)
    duration
Source: Food and Drug Administration’s (FDA) estimates from FDA’s Sentinel System, 2008-2017. | GAO-19-577
Notes: A new user was defined as having no use of any obesity drug in the previous 183 days. In
addition to new users of the nine prescription obesity drugs, FDA’s analysis included 125 new users
of Alli, a lower dose of orlistat that is an over-the-counter weight-loss medication that does not require
a prescription. Because four of the drugs—lorcaserin (Belviq), phentermine/topiramate (Qsymia),
liraglutide (Saxenda), and bupropion/naltrexone (Contrave)—were approved for the U.S. market in
2012 or later, not all of the drugs were available in all of the years included in this analysis.
a
 FDA estimated the duration of the first treatment episode (in days) for patients’ prescription
dispensings for any of the nine obesity drugs using a 14-day episode gap—that is, if there were more
than 14 days between exhausting the previous dispensing’s days supplied for that prescription and
refilling the prescription, then FDA counted it as a new treatment episode.
b
 FDA estimated cumulative treatment duration by summing days’ supply of all dispensings of an
obesity drug within each patient’s presence in the database, without regard to time between
dispensings. This measure combined drug exposure from multiple treatment episodes, if present.



Table 10: New Adult Users of Obesity Drugs by Age and Gender, 2008-2017

                                                                   Number of new adult users (percent)
    Age
          18-44                                                    132,317 (49.4)
          45-64                                                    113,325 (42.3)
          65 and older                                             22,194 (8.3)
    All Ages                                                       267,836 (100)
    Gender
          Female                                                   220,273 (82.2)
          Male                                                     47,557 (17.8)
Source: Food and Drug Administration’s (FDA) estimates from FDA’s Sentinel System, 2008-2017. | GAO-19-577

Notes: A new user was defined as having no use of any obesity drug in the previous 183 days. In
addition to new users of the nine prescription obesity drugs, FDA’s analysis included 125 new users
of Alli, a lower dose of orlistat that is an over-the-counter weight-loss medication that does not require
a prescription. Because four of the drugs—lorcaserin (Belviq), phentermine/topiramate (Qsymia),
liraglutide (Saxenda), and bupropion/naltrexone (Contrave)—were approved for the U.S. market in
2012 or later, not all of the drugs were available in all of the years included in this analysis.




Page 50                                                                                  GAO-19-577 Obesity Drugs
Appendix VI: Reimbursement for Obesity
             Appendix VI: Reimbursement for Obesity
             Drugs in Medicare Part D Enhanced Alternative
             Coverage, 2016 and 2017


Drugs in Medicare Part D Enhanced
Alternative Coverage, 2016 and 2017
             This appendix presents information on Medicare Part D plan
             reimbursement for obesity drugs under enhanced alternative coverage
             from our analysis of Centers for Medicare & Medicaid Services’ (CMS)
             Prescription Drug Event data. 1 Medicare Part D plans can choose
             whether or not to offer enhanced alternative coverage, and not all
             Medicare Part D plans that provide enhanced alternative coverage cover
             obesity drugs as supplemental drugs. 2 As of February 2017, 1,949
             Medicare Part D plans provided enhanced alternative coverage to 18.9
             million Medicare beneficiaries, according to the Medicare Payment
             Advisory Commission. Additionally, in 2015, total Medicare Part D
             spending for prescription drugs was about $137 billion—this represents
             payments from all payers including beneficiaries (cost sharing), and
             excluding rebates and discounts from pharmacies and manufacturers that
             are not reflected in prices at the pharmacies. 3

             Tables 11 and 12 show the number of claims reimbursed, the number of
             plans that provided reimbursement, and the amount reimbursed for




             1
              Enhanced alternative coverage is alternative prescription drug coverage under Medicare
             Part D with value exceeding that of Medicare Part D’s defined standard coverage.
             Enhanced alternative coverage may include basic prescription coverage and
             supplemental benefits such as supplemental drugs. Medicare beneficiaries who select a
             Part D plan that offers supplemental benefits, which may include coverage of obesity
             drugs, must pay the full premium cost for those additional benefits (i.e., Medicare does not
             subsidize them). For example, in 2017, average monthly premiums for beneficiaries in
             standalone Medicare Part D plans with enhanced benefits were $54 per month compared
             with an average premium of $31 for basic coverage. See Medicare Payment Advisory
             Commission, June 2018 Data Book: Health Care Spending and the Medicare Program,
             Section 10 (Washington, D.C.: July 2018) and Medicare Payment Advisory Commission,
             Report to the Congress: Medicare Payment Policy, Chapter 14 (Washington, D.C.: March
             2018).
             2
              Medicare Payment Advisory Commission, June 2018 Data Book: Health Care Spending
             and the Medicare Program, Section 10 (Washington, D.C.: July 2018), and Medicare
             Payment Advisory Commission, Report to the Congress: Medicare Payment Policy,
             Chapter 14 (Washington, D.C.: March 2019).
             3
              Medicare Payment Advisory Commission, June 2018 Data Book: Health Care Spending
             and the Medicare Program (Washington, D.C.: July 2018), 173.




             Page 51                                                          GAO-19-577 Obesity Drugs
                                                               Appendix VI: Reimbursement for Obesity
                                                               Drugs in Medicare Part D Enhanced Alternative
                                                               Coverage, 2016 and 2017




                                                               obesity drugs under Medicare Part D enhanced alternative coverage for
                                                               2016 and 2017, respectively. 4

Table 11: Medicare Part D Number of Claims, Unique Plans, Unique Beneficiaries, Plan Spending, and Beneficiary Liability for
the Nine Obesity Drugs, 2016

 Obesity drug                        Number of claims                 Number of plans                Number of            Plan spending Beneficiary liability
                                                                                                   beneficiaries                (dollars)          (dollars)
 Phentermine                                              344                               24                170                       714                    2,648
 Any obesity drug                                         419                               32                196                   19,714                     4,048
 (covered as a
                    a
 supplemental drug)
Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

                                                               Notes: We excluded 1,787 claims for one obesity drug (orlistat) that were listed in CMS’s data as
                                                               covered under Medicare Part D. According to CMS officials, orlistat has off-label indications, including
                                                               diabetes and hyperlipidemia, and it is covered under Medicare Part D when it is prescribed for these
                                                               indications. We also excluded 25 claims for obesity drugs listed as over-the-counter drugs in CMS’s
                                                               data.
                                                               a
                                                                Any obesity drug includes aggregated data for all nine obesity drugs.




                                                               4
                                                                We analyzed CMS’s Prescription Drug Event data for 2016 and 2017 for nine prescription
                                                               obesity drugs: benzphetamine, diethylpropion, phendimetrazine, phentermine,
                                                               bupropion/naltrexone (Contrave), liraglutide (Saxenda), lorcaserin (Belviq), orlistat
                                                               (Xenical), and phentermine/topiramate (Qsymia). We limited our analysis to claims coded
                                                               as supplemental drugs covered under enhanced alternative coverage and excluded
                                                               claims for one obesity drug (orlistat) that were listed in CMS’s data as covered (and not
                                                               enhanced) drugs under Medicare Part D. According to CMS officials, orlistat has off-label
                                                               indications, including for diabetes and hyperlipidemia, and it is covered under Medicare
                                                               Part D when it is prescribed for these indications. We also excluded claims for obesity
                                                               drugs listed as over-the-counter drugs in CMS’s data.




                                                               Page 52                                                                  GAO-19-577 Obesity Drugs
                                                               Appendix VI: Reimbursement for Obesity
                                                               Drugs in Medicare Part D Enhanced Alternative
                                                               Coverage, 2016 and 2017




Table 12: Medicare Part D Number of Claims, Unique Plans, Unique Beneficiaries, Plan Spending, and Beneficiary Liability for
the Nine Obesity Drugs, 2017

 Obesity drug                        Number of claims                 Number of plans                Number of            Plan spending Beneficiary liability
                                                                                                   beneficiaries                (dollars)          (dollars)
 Bupropion +                                              282                               ≤10               110                   76,209                     2,919
 Naltrexone
 Lorcaserin                                               157                               ≤10                 60                  45,865                     2,114
 Phentermine                                                84                               11                 32                       676                     135
 Any obesity drug                                         555                                27               209                  140,296                     5,376
 (covered as a
                    a
 supplemental drug)
Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

                                                               Notes: We excluded 1,775 claims for one obesity drug (orlistat) that were listed in CMS’s data as
                                                               covered under Medicare Part D. According to CMS officials, orlistat has off-label indications including
                                                               for diabetes and hyperlipidemia, and it is covered under Medicare Part D when it is prescribed for
                                                               these indications. We also excluded 26 claims for obesity drugs listed as over-the-counter drugs in
                                                               CMS’s data.
                                                               a
                                                                 Any obesity drug includes aggregated data for all nine obesity drugs.




                                                               Page 53                                                                   GAO-19-577 Obesity Drugs
Appendix VII: Reimbursement for Obesity
              Appendix VII: Reimbursement for Obesity
              Drugs in Medicaid, 2016 and 2017



Drugs in Medicaid, 2016 and 2017

              This appendix presents information on Medicaid reimbursements for
              obesity drugs under state Medicaid programs or Medicaid managed care
              programs within those states from our analysis of Centers for Medicare &
              Medicaid Services’ (CMS) Medicaid State Drug Utilization data. State
              Medicaid programs or Medicaid managed care programs reimbursed for
              at least one obesity drug prescription in 42 states in 2016 and 41 states in
              2017. 1 The amount that Medicaid reimbursed and the total number of
              prescriptions for obesity drugs reimbursed by Medicaid in 2016 and 2017
              are shown by state (tables 13 and 14), and by obesity drug (tables 15 and
              16). 2 Over half of the prescriptions for obesity drugs reimbursed under
              Medicaid in 2016 and 2017 were for the generic obesity drug,
              phentermine.

              Table 13: Medicaid Amount Reimbursed and Number of Prescriptions for Obesity
              Drugs in Each State, 2016

              State                                    Medicaid amount        Number of prescriptions
                                                    reimbursed (dollars)
              Alabama                                                 509                            ≤10
              Arizona                                              92,012                            383
              California                                        1,438,603                          8,346
              Colorado                                               1,084                           ≤10
              Connecticut                                         214,849                            426
              District of Columbia                                 21,658                            190
              Delaware                                               7,396                            25
              Florida                                              13,630                            120
              Georgia                                                9,114                            15
              Hawaii                                              377,311                            370
              Iowa                                                 34,524                            149
              Idaho                                                10,707                             21
              Illinois                                               4,505                            26


              1
               The Medicaid State Drug Utilization data do not include information on the indication or
              indications for which these drugs were prescribed. Therefore, we cannot determine
              whether these obesity drugs were prescribed off label for an indication other than obesity.
              2
               The Medicaid amount reimbursed includes state and federal reimbursement and
              dispensing fees. These amounts do not include all Medicaid spending for obesity drugs
              under Medicaid managed care—because managed care organizations can be paid for the
              drugs as part of their capitated payment for all Medicaid services, they are not reimbursed
              on a per-drug basis and their payment amounts are not recorded in CMS’s Medicaid State
              Drug Utilization data.




              Page 54                                                          GAO-19-577 Obesity Drugs
Appendix VII: Reimbursement for Obesity
Drugs in Medicaid, 2016 and 2017




 State                                                  Medicaid amount                     Number of prescriptions
                                                     reimbursed (dollars)
 Indiana                                                                   2,207                                ≤10
 Kansas                                                                 544,255                               1,092
 Kentucky                                                               342,704                                 742
 Louisiana                                                                82,352                                146
 Massachusetts                                                            77,312                              3,198
 Maryland                                                                 79,232                                154
 Michigan                                                               144,506                                 844
 Minnesota                                                                42,427                                 93
 Missouri                                                                 15,539                                 84
 Mississippi                                                                  130                               ≤10
 Montana                                                                   1,095                                ≤10
 North Carolina                                                                 19                              ≤10
 North Dakota                                                              2,432                                 35
 New Hampshire                                                            10,945                                 65
 New Jersey                                                                4,374                                 18
 New Mexico                                                                1,098                                ≤10
 Nevada                                                                    7,787                                 79
 New York                                                                 29,359                              1,351
 Ohio                                                                      2,158                                 13
 Oregon                                                                    6,406                                 24
 Pennsylvania                                                             26,117                                121
 Rhode Island                                                              7,417                                117
 South Carolina                                                           22,036                                168
 Tennessee                                                                16,236                                 32
 Texas                                                                  152,087                                 296
 Virginia                                                               115,677                                 704
 Washington                                                                9,226                                 51
 Wisconsin                                                           1,040,564                                5,789
 West Virginia                                                             5,826                                 11
Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

Notes: The table shows the obesity drugs for which the state Medicaid Program or Medicaid
managed care program paid a portion or all of a claim. The Medicaid amount reimbursed includes
state and federal reimbursement and dispensing fees. These amounts do not include all Medicaid
spending for obesity drugs under Medicaid managed care—because managed care organizations
can be paid for the drugs as part of their capitated payment for all Medicaid services, they are not
reimbursed on a per-drug basis, and their payment amounts are not recorded as amounts reimbursed
in CMS’s Medicaid State Drug Utilization data. The number of prescriptions reimbursed includes 776
prescriptions for obesity drugs that showed zero dollar amounts for Medicaid reimbursement in
CMS’s Medicaid State Drug Utilization data.




Page 55                                                                                     GAO-19-577 Obesity Drugs
Appendix VII: Reimbursement for Obesity
Drugs in Medicaid, 2016 and 2017




Table 14: Medicaid Amount Reimbursed and Total Number of Prescriptions for
Obesity Drugs in Each State, 2017

State                                   Medicaid amount     Number of prescriptions
                                     reimbursed (dollars)
Alabama                                            6,080                         25
Arizona                                           96,919                        379
California                                     2,846,888                     11,437
Colorado                                             815                        ≤10
Connecticut                                      260,890                        484
District of Columbia                              33,728                        154
Delaware                                              12                         90
Florida                                            3,489                         78
Georgia                                            1,623                         31
Hawaii                                            27,099                        492
Idaho                                              9,245                         16
Illinois                                           2,625                         14
Indiana                                            1,056                         15
Kansas                                         1,200,333                      2,086
Kentucky                                         751,439                      1,233
Louisiana                                         71,627                        122
Massachusetts                                     94,737                      3,961
Maryland                                          49,391                         99
Michigan                                          93,505                        496
Minnesota                                         12,307                         28
Missouri                                           2,444                         56
Mississippi                                        3,346                        ≤10
North Carolina                                     2,004                        ≤10
North Dakota                                       2,248                         82
Nebraska                                             546                        ≤10
New Hampshire                                     15,270                         75
New Jersey                                           116                        ≤10
New Mexico                                         2,462                         20
Nevada                                             8,570                         78
New York                                           2,752                         22
Ohio                                               5,796                         14
Oregon                                            25,140                         39
Pennsylvania                                      33,359                         88
Rhode Island                                      24,196                      1,388




Page 56                                                     GAO-19-577 Obesity Drugs
                                                               Appendix VII: Reimbursement for Obesity
                                                               Drugs in Medicaid, 2016 and 2017




                                                                State                                                  Medicaid amount                     Number of prescriptions
                                                                                                                    reimbursed (dollars)
                                                                South Carolina                                                           24,461                                180
                                                                Tennessee                                                                21,012                                 29
                                                                Texas                                                                  149,520                                 281
                                                                Virginia                                                                 70,657                                473
                                                                Washington                                                                8,715                                 69
                                                                Wisconsin                                                           1,484,032                                6,633
                                                                West Virginia                                                             2,984                                ≤10
                                                               Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

                                                               Notes: The table shows the obesity drugs for which the state Medicaid Program or Medicaid
                                                               managed care program paid a portion or all of a claim. The Medicaid amount reimbursed includes
                                                               state and federal reimbursement and dispensing fees. These amounts do not include all Medicaid
                                                               spending for obesity drugs under Medicaid managed care—because managed care organizations
                                                               can be paid for the drugs as part of their capitated payment for all Medicaid services, they are not
                                                               reimbursed on a per-drug basis, and their payment amounts are not recorded as amounts reimbursed
                                                               in CMS’s Medicaid State Drug Utilization data. The number of prescriptions reimbursed includes 144
                                                               prescriptions for obesity drugs that showed zero dollar amounts for Medicaid reimbursement in
                                                               CMS’s Medicaid State Drug Utilization data.




Table 15: Medicaid Amount Reimbursed and Total Number of Prescriptions for Each of the Nine Obesity Drugs, 2016

 Obesity drug                                                       Medicaid amount reimbursed (dollars)                                                   Number of prescriptions
 Phentermine                                                                                                      421,084                                                   13,274
 Diethylpropion                                                                                                    31,735                                                     203
 Benzphetamine                                                                                                          907                                                     53
 Phendimetrazine                                                                                                     8,861                                                    238
 Orlistat                                                                                                      1,240,859                                                     2,403
 Lorcaserin                                                                                                       623,974                                                    2,942
 Phentermine + Topiramate                                                                                         342,891                                                    1,910
 Liraglutide                                                                                                   1,941,148                                                     2,052
 Bupropion + Naltrexone                                                                                           406,144                                                    2,237
 All obesity drugs                                                                                             5,017,424                                                    25,312
Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

                                                               Notes: The table shows the obesity drugs for which the state Medicaid Program or Medicaid
                                                               managed care program paid a portion or all of a claim. The Medicaid amount reimbursed includes
                                                               state and federal reimbursement and dispensing fees. These amounts do not include all Medicaid
                                                               spending for obesity drugs under Medicaid managed care—because managed care organizations
                                                               can be paid for the drugs as part of their capitated payment for all Medicaid services, they are not
                                                               reimbursed on a per-drug basis, and their payment amounts are not recorded as amounts reimbursed
                                                               in CMS’s Medicaid State Drug Utilization data. The number of prescriptions reimbursed includes 776
                                                               prescriptions for obesity drugs that showed zero dollar amounts for Medicaid reimbursement in
                                                               CMS’s Medicaid State Drug Utilization data.




                                                               Page 57                                                                                     GAO-19-577 Obesity Drugs
                                                               Appendix VII: Reimbursement for Obesity
                                                               Drugs in Medicaid, 2016 and 2017




Table 16: Medicaid Amount Reimbursed and Total Number of Prescriptions for Each of the Nine Obesity Drugs, 2017

 Obesity drug                                                       Medicaid amount reimbursed (dollars)                             Number of prescriptions
 Phentermine                                                                                           130,329                                            15,526
 Diethylpropion                                                                                           5,919                                               260
 Benzphetamine                                                                                            1,612                                                44
 Phendimetrazine                                                                                          2,095                                               125
 Orlistat                                                                                            1,195,505                                              2,214
 Lorcaserin                                                                                            629,405                                              2,695
 Phentermine + Topiramate                                                                              382,877                                              2,147
 Liraglutide                                                                                         4,534,941                                              4,858
 Bupropion + Naltrexone                                                                                570,759                                              2,931
 All obesity drugs                                                                                   7,453,442                                            30,800
Source: GAO analysis of Centers for Medicare & Medicaid Services (CMS) data. | GAO-19-577

                                                               Notes: The table shows the obesity drugs for which the state Medicaid Program or Medicaid
                                                               managed care program paid a portion or all of a claim. The Medicaid amount reimbursed includes
                                                               state and federal reimbursement and dispensing fees. These amounts do not include all Medicaid
                                                               spending for obesity drugs under Medicaid managed care—because managed care organizations
                                                               can be paid for the drugs as part of their capitated payment for all Medicaid services, they are not
                                                               reimbursed on a per-drug basis, and their payment amounts are not recorded as amounts reimbursed
                                                               in CMS’s Medicaid State Drug Utilization data. The number of prescriptions reimbursed includes 144
                                                               prescriptions for obesity drugs that showed zero dollar amounts for Medicaid reimbursement in
                                                               CMS’s Medicaid State Drug Utilization data.




                                                               Page 58                                                               GAO-19-577 Obesity Drugs
Appendix VIII: Estimates of Medical and
              Appendix VIII: Estimates of Medical and
              Prescription Drug Expenditures for Adults
              Who Used and Did Not Use Obesity Drugs


Prescription Drug Expenditures for Adults
Who Used and Did Not Use Obesity Drugs
              This appendix presents nationally representative estimates of U.S. adults’
              average annual expenditures (spending) for medical care, all prescription
              drugs, and for obesity drugs from the Agency for Healthcare Research
              and Quality (AHRQ) based on data from the Medical Expenditure Panel
              Survey (MEPS) for 2012 through 2016. Table 17 shows the estimated
              average annual expenditures for all prescription drugs and table 18
              shows the estimated average annual medical expenditures, including
              prescription drugs, per adult who used and per adult who did not use any
              obesity drugs. 1 For adults age 18 to 64, the differences in the estimated
              average annual expenditures for all medical care and for all prescriptions
              drugs per adult who used and who did not use any of the nine obesity
              drugs in our review were statistically significant. 2 However, the
              differences in these estimates do not indicate that there was a causal
              relationship between using obesity drugs and having higher average
              annual medical or prescription drug expenditures. Table 19 shows the
              estimated average annual expenditures per adult for obesity drugs.




              1
               MEPS collects nationally representative data on health care use, expenditures, sources
              of payment, and insurance coverage for the U.S. civilian, noninstitutionalized population.
              For this analysis, AHRQ pooled MEPS data from 2012 through 2016 for individuals,
              including individuals who used any of nine obesity drugs: benzphetamine, diethylpropion,
              phendimetrazine, phentermine, bupropion/naltrexone (Contrave), liraglutide (Saxenda),
              lorcaserin (Belviq), orlistat (Xenical), and phentermine/topiramate (Qsymia). Because two
              of the drugs—Saxenda and Contrave—were approved for the U.S. market in 2014, not all
              of the drugs were available in all of the years included in this analysis. We define use of
              obesity drugs as having an outpatient prescription fill or refill for any of the nine obesity
              drugs.
              2
               We compared the 95 percent confidence intervals of these estimates to determine
              whether any differences we found are statistically significant. This is the interval that would
              contain the actual population value for 95 percent of the samples MEPS could have
              drawn. Statistical significance indicates that the difference between the estimates is
              unlikely due to chance alone. Confidence intervals account for the complex survey design
              of the MEPS.




              Page 59                                                            GAO-19-577 Obesity Drugs
                                                              Appendix VIII: Estimates of Medical and
                                                              Prescription Drug Expenditures for Adults
                                                              Who Used and Did Not Use Obesity Drugs




Table 17: Estimates of Average Annual Expenditures per Adult for All Prescription Drugs, 2012–2016

(dollars)

                                                                      Estimate                                         95 percent confidence interval
                                                                                                                        Lower bound                          Upper bound
 All U.S. adults                                                           1,419                                                    1,358                            1,481
        BMI ≥ 30                                                           1,999                                                    1,849                            2,149
        BMI < 30                                                           1,163                                                    1,109                            1,217
        Age 18–64                                                          1,117                                                    1,047                            1,186
        Age 65 and older                                                   2,665                                                    2,533                            2,797
                                a
 Used any obesity drug                                                     2,305                                                    1,519                            3,091
        BMI ≥ 30                                                           2,357                                                    1,415                            3,300
        BMI < 30                                                           2,179                                                       992                           3,366
        Age 18–64                                                          2,198                                                    1,390                            3,006
                                                                                    b                                                        b                              b
        Age 65 and older                                                      n/a                                                      n/a                            n/a
 Did not use any obesity drug                                              1,416                                                    1,355                            1,477
        BMI ≥ 30                                                           1,995                                                    1,846                            2,144
        BMI < 30                                                           1,161                                                    1,107                            1,215
        Age 18–64                                                          1,111                                                    1,043                            1,180
        Age 65 and older                                                   2,663                                                    2,531                            2,795
Source: Agency for Health Care Research and Quality’s (AHRQ) estimates from the Medical Expenditure Panel Survey (MEPS), 2012–2016. | GAO-19-577

                                                              Notes: Body mass index (BMI) is calculated from individuals’ height and weight as reported by
                                                              household respondents in MEPS. As a result, because some individuals may self report higher than
                                                              actual height and lower than actual weight, calculations of BMI from MEPS data may be lower than
                                                              actual BMI. These estimates are from MEPS pooled data for years 2012 through 2016 and exclude
                                                              adults age 18 and older with missing BMI data and women who reported that they were pregnant
                                                              during the reference period of the interview in which they reported height and weight.
                                                              Prescription drug expenditures in 2012 to 2015 were inflated to 2016 dollars using the Consumer
                                                              Price Index for Prescription Drugs, available from the Bureau of Labor Statistics. Confidence intervals
                                                              account for the complex survey design of the MEPS. Estimates are based on an unweighted sample
                                                              of 118,615 adults and an estimated average annual population of 233,060,000 adults.
                                                              a
                                                               Estimates are based on 397 MEPS sample members who were reported to have used any of the
                                                              nine obesity drugs in the 2012 through 2016 MEPS. AHRQ estimates that there are an average of
                                                              940,000 adults per year who reported using obesity drugs.
                                                              b
                                                               Estimates for individuals age 65 and older who used obesity drugs are not reported due to
                                                              inadequate precision for estimates resulting from an insufficient sample size.




                                                              Page 60                                                                              GAO-19-577 Obesity Drugs
                                                              Appendix VIII: Estimates of Medical and
                                                              Prescription Drug Expenditures for Adults
                                                              Who Used and Did Not Use Obesity Drugs




Table 18: Estimates of Average Annual Medical Expenditures per Adult, 2012 – 2016

(dollars)

                                                                      Estimate                                         95 percent confidence interval
                                                                                                                        Lower bound                          Upper bound
 All U.S. adults                                                           5,493                                                    5,338                            5,647
        BMI ≥ 30                                                           6,666                                                    6,370                            6,962
        BMI < 30                                                           4,973                                                    4,814                            5,131
        Age 18–64                                                          4,317                                                    4,161                            4,473
        Age 65 and older                                                  10,326                                                    9,951                           10,701
                                a
 Used any obesity drug                                                     7,932                                                    5,603                           10,261
        BMI ≥ 30                                                           8,386                                                    5,234                           11,539
        BMI < 30                                                           6,837                                                    4,897                            8,778
        Age 18–64                                                          7,575                                                    5,139                           10,010
                                                                                    b                                                        b                              b
        Age 65 and older                                                      n/a                                                      n/a                            n/a
 Did not use any obesity drug                                              5,483                                                    5,330                            5,636
        BMI ≥ 30                                                           6,650                                                    6,358                            6,941
        BMI < 30                                                           4,969                                                    4,811                            5,128
        Age 18–64                                                          4,302                                                    4,147                            4,456
        Age 65 and older                                                  10,322                                                    9,947                           10,696
Source: Agency for Health Care Research and Quality’s (AHRQ) estimates from the Medical Expenditure Panel Survey (MEPS), 2012–2016. | GAO-19-577

                                                              Notes: Estimates include expenditures for prescription drugs. Body mass index (BMI) is calculated
                                                              from individuals’ height and weight as reported by household respondents in MEPS. As a result,
                                                              because some individuals may self report higher than actual height and lower than actual weight,
                                                              calculations of BMI from MEPS data may be lower than actual BMI. These estimates are from MEPS
                                                              pooled data for years 2012 through 2016 and exclude adults age 18 and older with missing BMI data
                                                              and women who reported that they were pregnant during the reference period of the interview in
                                                              which they reported height and weight.
                                                              Medical expenditures in 2012 to 2015 were inflated to 2016 dollars using the Personal Health Care
                                                              Expenditure Index available from the Centers for Medicare & Medicaid Services. Confidence intervals
                                                              account for the complex survey design of the MEPS. Estimates are based on an unweighted sample
                                                              of 118,615 adults and an estimated average annual population of 233,060,000 adults.
                                                              a
                                                               Estimates are based on 397 MEPS sample members who were reported to have used any of the
                                                              nine obesity drugs in the 2012 through 2016 MEPS. AHRQ estimates that there are an average of
                                                              about 940,000 adults per year who reported using obesity drugs.
                                                              b
                                                               Estimates for individuals age 65 years and older who used obesity drugs are not reported due to
                                                              inadequate precision for estimates resulting from an insufficient sample size.




                                                              Page 61                                                                              GAO-19-577 Obesity Drugs
                                                              Appendix VIII: Estimates of Medical and
                                                              Prescription Drug Expenditures for Adults
                                                              Who Used and Did Not Use Obesity Drugs




Table 19: Estimates of Average Annual and Median Expenditures per Adult for All Obesity Drugs Purchased, 2012–2016

(dollars)

                             Estimated                 95 percent confidence interval                         Estimated                 95 percent confidence interval
                               average                                                                          median
                                                        Lower bound                 Upper bound                                           Lower bound        Upper bound
                                (mean)
 All U.S. adults                       174                            141                         207                     87                        74                   103
 BMI ≥ 30                              193                            149                         237                     94                        71                   118
 BMI < 30                              129                              93                        165                     81                        59                    98
 Age 18–64                             169                            137                         202                     87                        72                   103
                                            a                               a                           a                     a                          a                     a
 Age 65 and                           n/a                             n/a                         n/a                   n/a                        n/a                   n/a
 older
Source: Agency for Health Care Research and Quality’s (AHRQ) estimates from the Medical Expenditure Panel Survey (MEPS), 2012–2016. | GAO-19-577

                                                              Notes: Estimates are based on 397 MEPS sample members who were reported to have used any of
                                                              the nine obesity drugs in the 2012 through 2016 MEPS. AHRQ estimates that there are an average of
                                                              about 940,000 adults per year who reported using obesity drugs. Body mass index (BMI) is calculated
                                                              from individuals’ height and weight as reported by household respondents in MEPS. As a result,
                                                              because some individuals may self report higher than actual height and lower than actual weight,
                                                              calculations of BMI from MEPS data may be lower than actual BMI. These estimates are from MEPS
                                                              pooled data for years 2012 through 2016 and exclude adults age 18 and older with missing BMI data
                                                              and women who reported that they were pregnant during the reference period of the interview in
                                                              which they reported height and weight.
                                                              Expenditures in 2012 to 2015 were inflated to 2016 dollars using the Consumer Price Index for
                                                              Prescription Drugs from the Bureau of Labor Statistics. Confidence intervals account for the complex
                                                              survey design of the MEPS.
                                                              a
                                                               Estimates for individuals age 65 years and older who used obesity drugs are not reported due to
                                                              inadequate precision for estimates resulting from an insufficient sample size.




                                                              Page 62                                                                              GAO-19-577 Obesity Drugs
Appendix IX: GAO Contact and Staff
                  Appendix IX: GAO Contact and Staff
                  Acknowledgments



Acknowledgments

                  John E. Dicken (202) 512-7114 or dickenj@gao.gov
GAO Contact
                  In addition to the contact above, Kim Yamane (Assistant Director), Lisa A.
Staff             Lusk (Analyst-in-Charge), George Bogart, Zhi Boon, Kaitlin Dunn, Laurie
Acknowledgments   Pachter, and Merrile Sing made key contributions to this report. Also
                  contributing to this report were Alexander Cattran, Leia Dickerson, Diona
                  Martyn, Christina Ritchie, and Ethiene Salgado-Rodriguez.




(102781)
                  Page 63                                             GAO-19-577 Obesity Drugs
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