United States: Anti-Obesity Medications As Medicare Part D Drugs: Legal And Health Policy Rationales

This white paper was commissioned by Pfizer Inc.

Executive Summary

Obesity is a complex, multifactorial disease that has serious health consequences, affects millions of Americans and drives hundreds of billions of dollars in annual health care spending. Scientific understanding of the nature of obesity has evolved over the course of the past several decades. It is now clinically recognized that obesity is a disease, not simply a cosmetic concern or issue of personal behavior. We now have a much greater understanding of obesity's various metabolic implications and direct links to cardiovascular disease, type 2 diabetes, liver disease and certain types of cancer, among other diseases. Multiple prescription medications have been developed, approved and recognized as safe and effective for long-term use in treating obesity. However, the Centers for Medicare & Medicaid Services (CMS) has historically prohibited coverage of these drugs under Medicare Part D, citing a decades-old federal statute excluding "agents when used for anorexia, weight loss, or weight gain" (the Statutory Exclusion). A new generation of drugs, commonly referred to as "GLP-1 agonists" (GLP-1s) have demonstrated significant efficacy in treating the disease of obesity and its well-known comorbidities. These are not the "weight loss drugs" of the past; instead, this class of drugs should be referred to as "anti-obesity medications" (AOMs). This paper summarizes the legal and policy rationales for CMS to alter its interpretation and concludes that the Statutory Exclusion does not prevent Part D coverage of AOMs. Put simply, obesity treatment, including use of AOMs, is not treatment solely for weight loss and the Statutory Exclusion need not prevent Part D coverage of AOMs on the basis of their weight loss properties. By both recognizing obesity as a complex chronic disease and describing AOMs as agents targeting obesity, rather than describing each solely in terms of effect on weight, CMS could interpret the Statutory Exclusion to reflect modern clinical understanding and not to preclude Part D coverage of AOMs. Doing so would align CMS policy with advances in pharmacology, clinical treatment guidelines and the Biden Administration's policy priorities.

Revising CMS's Interpretation to Characterize Obesity More Accurately as a Disease Would Provide CMS With Flexibility to Cover AOMs Under Part D

CMS characterizes obesity predominantly in terms of an individual's weight and maintains that drugs for obesity treatment are not covered by Medicare because they remain "agents...used for weight loss" subject to the Statutory Exclusion.¹ In contrast, various federal agencies, including the U.S. Centers for Disease Control and Prevention (CDC), in addition to leading professional medical societies, agree that obesity should be treated as an independent disease state, which is characterized by adipose tissue buildup and other metabolic implications (see Figure 1).² In conflating all drugs for obesity treatment with drugs for "weight loss," CMS disregards the metabolic implications of obesity and the ability of AOMs to treat obesity, unnecessarily rendering AOMs as non-covered under Part D. In addition, to reflect the current scientific and medical consensus around obesity as an independent disease state, the U.S. Food and Drug Administration (FDA) should update its governing 2007 guidance document on obesity drug development,³ which uses outdated and stigmatizing language focusing on "weight management," even though the guidance's conceptual and scientific principles reflect the need to assess obesity and all of its metabolic implications and comorbidities. In July 2023, FDA announced its intention to update this guidance.⁴ In recognition of the roadblock to addressing obesity posed by CMS's interpretation, Congress has repeatedly expressed support for reinterpreting the Statutory Exclusion not to preclude Part D coverage of AOMs, thereby improving access to obesity care.

Figure 1: Federal Agencies Indicating a Modern Understanding of Obesity

Changing CMS's Interpretation of the Statutory Exclusion Would Be Consistent With CMS's Precedents

CMS has previously modified its interpretation of the Statutory Exclusion to permit coverage of Serostim, a drug used to treat wasting/cachexia resulting from acquired immunodeficiency syndrome (AIDS) by increasing the patient's weight but has, to date, declined to interpret the Statutory Exclusion to permit coverage of AOMs under analogous circumstances. Serostim is indicated to treat HIV patients with wasting or cachexia—a contributing factor in the death rate of patients with AIDS—"to increase lean body mass and body weight, and improve physical endurance."⁵ CMS initially considered Serostim as subject to the Statutory Exclusion, only later declaring that drugs used to treat AIDS wasting/cachexia were not considered agents used for weight gain.⁶ Consistent application of the Serostim coverage precedent would enable Part D coverage of AOMs by recognizing that these drugs treat the life-threatening disease of obesity, both by producing significant weight loss and with top-line results demonstrating reduction in risk of major adverse cardiovascular events.⁷ Especially with recently announced evidence of cardiovascular benefit, CMS can directly apply the same rationale for reinterpretation of coverage for anti-obesity medications that it applied to Serostim.

Significant Improvements in Safety and Effectiveness of New Generation of AOMs Distinguish Them From Previous Weight Loss Drugs

A new class of medicines to treat obesity, GLP-1s, has emerged that improves upon the previous generation of weight loss therapies, many of which were developed prior to 1990 and had been withdrawn due to safety concerns. In addition to producing significant weight loss, these new GLP-1s have been demonstrated to result in improvements in metabolic processes affected by obesity and demonstrable reduction in risk associated with other linked diseases, including diabetes and heart disease.⁸ FDA Commissioner Robert Califf has characterized this class of medicines as "the beginning of a revolution in the way that we control weight, not just with the pills, but because we'll understand the biological mechanisms better."⁹ Coverage of and reimbursement for this new generation of AOMs prescribed for medically necessary treatment can help patients achieve better health outcomes and produce savings to health care systems. In light of evidence supporting AOMs' clinical impact and detailing barriers to accessing obesity treatment, there is little clinical basis to single out this class of drugs for exclusion from the Part D program.

CMS's Formal Recognition of Obesity as a Disease and Its Coverage of AOMs Would Allow Medicare Beneficiaries to Access Clinical Guidelines-Recommended Treatment

Clinical guidelines recommend the use of AOMs adjunctively to lifestyle, behavioral and surgical obesity interventions, depending on a patient's personalized obesity treatment plan.¹⁰ Incorporation of AOMs into clinical practice is attributable, at least in part, to research demonstrating that adding AOMs to such interventions produces greater weight loss and maintenance of weight loss than non-pharmacologic interventions alone.¹¹ FDA's guidance for drug developers recognizes the complexity of obesity as chronic and relapsing and urges manufacturers to study drugs to treat it not only for their impact on reduction in weight but also on other metabolic parameters, such as blood pressure and lipid levels.¹² In recognition of such advances, Congress has urged CMS to clarify that the Statutory Exclusion does not include AOMs approved by FDA.¹³ However, AOMs remain non-covered, and the scope of lifestyle, behavioral and surgical obesity interventions covered by Medicare remains narrow; Medicare does not cover these treatment modalities unless beneficiaries satisfy various preconditions, and contrary to clinical guidelines, Medicare does not allow for the use of AOMs to supplement covered interventions.¹⁴ This has led to substantially limited uptake of such treatment modalities and indicates the need for expanded access to them and to AOMs as additional covered obesity treatment options.¹⁵ The exclusion of AOMs from Part D supplants the judgment of medical professionals, stripping providers of their ability to prescribe appropriate treatments consistent with clinical guidelines.

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Footnotes

1. Social Security Act § 1927(d)(2)(A), 42 U.S.C. § 1396r-8(d)(2)(A); Social Security Act § 1860D-2(e)(2)(A), 42 U.S.C. § 1395w-102(e)(2).

2. Centers for Disease Control and Prevention (CDC), Overweight & Obesity (2022), https://www.cdc.gov/obesity/Index.html; Garvey, W.T., Hurley, D.L., and Mechanick, J.I., Adiposity-Based Chronic Disease as a New Diagnostic Term: The American Association of Clinical Endocrinologists and American College of Endocrinology Position Statement, 23(3) Endocrine Pract. 372 (Dec. 14, 2017), https://www.endocrinepractice.org/article/S1530-891X(20)35834-1/fulltext; American Medical Association (AMA), Recognition of Obesity as a Disease H-440.842 (2013), https://policysearch.ama-assn.org/policyfinder/detail/obesity?uri=%2FAMADoc%2FHOD.xml-0-3858.xml.

3. Food and Drug Administration (FDA), Guidance for Industry Developing Products for Weight Management, Draft Guidance, Revision 1 (Feb. 2007), https://www.fda.gov/media/71252/download.

4. Food and Drug Administration (FDA), CDER Guidance Agenda New & Revised Draft Guidance Documents Planned for Publication in Calendar Year 2023 (July 2023), https://www.fda.gov/media/134778/download.

5. Food and Drug Administration (FDA)-approved Serostim label (online at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/020604s078lbl.pdf).

6. See National Library of Medicine (NIH), After Protests, HCFA Requires Coverage of Anti-Wasting Drug, 14(8) AIDS Policy & Law 11 (1999), https://pubmed.ncbi.nlm.nih.gov/11366530/; National Library of Medicine (NIH), HCFA Reverses Ruling on AIDS Wasting Treatment, 14(6) AIDS Alert 69 (June 1999), https://pubmed.ncbi.nlm.nih.gov/11366412/; Centers for Medicare & Medicaid Services (CMS), Medicaid Drug Rebate Program Release #88 (Mar. 5, 1999), https://www.medicaid.gov/medicaid-chip-program-information/by-topics/prescription-drugs/downloads/rx-releases/state-releases/state-rel-088.pdf.

7. Novo Nordisk, Company Announcement: Semaglutide 2.4 mg reduces the risk of major adverse cardiovascular events by 20% in adults with overweight or obesity in the SELECT trial (Aug. 8, 2023), https://www.novonordisk.com/news-and-media/news-and-ir-materials/news-details.html?id=166301#:~:text=Novo%20Nordisk%20A%2FS%3A%20Semaglutide,the%20SELECT%20cardiovascular%20outcomes%20trial.

8. Wilding, John, D.M., Once-Weekly Semaglutide in Adults with Overweight or Obesity, 384 New. Eng. J. Med. 989 (2021), https://www.nejm.org/doi/pdf/10.1056/NEJMoa2032183?articleTools=true; Garvey, W.T., Frias, J.P., et al. Tirzepatide once weekly for the treatment of obesity in people with type 2 diabetes (SURMOUNT-2): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial, Lancet (Aug. 19, 2023), https://pubmed.ncbi.nlm.nih.gov/37385275/; Jastreboff, A.M., PhD, Kaplan, L.M., PhD, et al. Triple-Hormone-Receptor Agonist Retatrutide for Obesity-A Phase 2 Trial, New Eng. J. Med. (2023), https://www.nejm.org/doi/full/10.1056/NEJMoa2301972; Lilly Investors, News Release: Lilly's phase 2 retatrutide results published in The New England Journal of Medicine show the investigational molecule achieved up to 17.5% mean weight reduction at 24 weeks in adults with obesity and overweight, (Jun. 26, 2023), https://investor.lilly.com/news-releases/news-release-details/lillys-phase-2-retatrutide-results-published-new-england-journal; Vosoughi, K., Salman, R.R., et al. Effects of GLP-1 agonists on proportion of weight loss in obesity with or without diabetes; Systematic review and meta-analysis, 35 Obesity Med. (2022), https://www.sciencedirect.com/science/article/abs/pii/S2451847622000689; Drucker, Daniel, GLP-1 Physiology Informs the Pharmacology of Obesity, Molecular Metabolism (Mar. 2022), https://www.sciencedirect.com/science/article/pii/S2212877821001988#sec7; Mihaela-Simona Popoviciu, et al., Emerging Role of GLP-1 Agonists in Obesity: A Comprehensive Review of Randomised Controlled Trials, 24(13) Inter. J. of Molecular Sci. 10449 (June, 2021), https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10341852; Gao, Zueqin, et al. Efficacy and safety of semaglutide on weight loss in obese or overweight patients without diabetes: A systematic review and meta-analysis of randomized controlled trials, 13 Frontiers Pharmacology (Sept. 14, 2022), https://www.frontiersin.org/articles/10.3389/fphar.2022.935823/full; Bergmann, N.C., MD, et al. Semaglutide for the treatment of overweight and obesity: A review, 25 Diabetes Obesity & Metabolism (2022), https://dom-pubs.pericles-prod.literatumonline.com/doi/10.1111/dom.14863; Hong, M., Lin, Y.H., et al. Efficacy and safety of GLP-1 receptor agonists versus SGLT-2 inhibitors in overweight/obese patients with or without diabetes mellitus: a systematic review and network meta-analysis. BMJ Open (2023), https://bmjopen.bmj.com/content/13/3/e061807; Vosoughi, K. et al. Association of glucagon-like peptide 1 analogs and agonists administered for obesity with weight loss and adverse events: a systematic review and network meta-analysis, 42 EClinicalMed. (2021), https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(21)00494-6/fulltext; Liu, Yupeng et al., The Weight-loss Effect of GLP-1RAs Glucagon-Like Peptide-1 Receptor Agonists in Non-diabetic Individuals with Overweight or Obesity: A Systematic Review with Meta-Analysis and Trial Sequential Analysis of Randomized Controlled Trials, 118 Am. J. Clinical Nutrition (2023), https://ajcn.nutrition.org/article/S0002-9165(23)46846-3/fulltext; Deng, Y., et al. Effect of semaglutide and liraglutide in individuals with obesity or overweight without diabetes: a systematic review. Therapeutic Advances in Chronic Disease (2022), https://journals.sagepub.com/doi/full/10.1177/20406223221108064; Paul Ryan, P.M., MB, BCh, BAO, PhD et al. Safety and Efficacy of Glucagon-Like Peptide-1 Receptor Agonists in Children and Adolescents with Obesity: A Meta-Analysis, 236 J. Pediatrics (2021), https://www.jpeds.com/article/S0022-3476(21)00432-7/fulltext.

9. Interview with Dr. Robert Califf, FDA Comm. (Apr. 6, 2023) (transcript on file, https://chcradio.s3.us-east-2.amazonaws.com/transcripts/Episode667Califf.pdf).

10. Bays, H.E., Burridge, K., et al., Anti-Obesity Medications and Investigational Agents: An Obesity Medicine Association (OMA) Clinical Practice Statement (CPS) 2022, 2 Obesity Pillars 10001.8 (2022), https://www.sciencedirect.com/science/article/pii/S2667368122000092; Grunvald, E., Hernaez, R., et al., AGA Clinical Practice Guideline on Pharmacological Interventions for Adults with Obesity, 163 Gastroenterology 1198 (2022), https://www.gastrojournal.org/article/S0016-5085(22)01026-5/fulltext; Adamo, K., Alberga, A., et al., Obesity in Adults: A Clinical Practice Guideline, 192(31) CMAJ E875–E891 (Aug. 4, 2020), https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7828878/; McKinney, L., and Skolnick, N., Diagnosis and Management of Obesity, Am. Acad. Fam. Physicians (2013), https://www.aafp.org/dam/AAFP/documents/patient_care/fitness/obesity-diagnosis-mono.pdf; Hampl, Sarah, E., et al., Clinical Practice Guideline for the Evaluation and Treatment of Children and Adolescents With Obesity. Pediatrics. (Feb 1. 2023), https://pubmed.ncbi.nlm.nih.gov/36622115/; Cornier, Marc-Andre, MD, A Review of Current Guidelines for the Treatment of Obesity, 28 Am. J. Managed Care (2022), https://www.ajmc.com/view/review-of-current-guidelines-for-the-treatment-of-obesity; VA and DoD, VA/DoD Clinical Practice Guideline for the Management of Adult Overweight and Obesity – Version 3 (2020), https://www.healthquality.va.gov/guidelines/CD/ obesity/VADoDObesityCPGFinal5087242020.pdf.

11. See Am. Acad. Clin. Endocrinology (AACE), How Do We Treat Obesity?, Sec. 3.2, https://pro.aace.com/disease-state-resources/nutrition-and-obesity/depth-information/section-3-how-do-we-treat-obesity (accessed Aug. 19, 2022); Mayo Clinic, Prescription Weight-Loss Drugs: Examine the Pros and Cons of Medications to Treat Obesity, https://www.mayoclinic.org/healthy-lifestyle/weight-loss/in-depth/weight-loss-drugs/art-20044832 (last updated Nov. 4, 2020); For example, one prominent study focusing on the Part B covered benefit for IBT found that "[i]n individuals with obesity, liraglutide-IBT was superior to placebo-IBT in reducing baseline body weight and producing a clinically meaningful ≥ 5% weight loss at week 56." Auerbach, P., Lund, M.T., et al., Liraglutide 3.0 mg and Intensive Behavioral Therapy (IBT) for Obesity in Primary Care: The SCALE IBT Randomized Controlled Trial, 28 Obesity 529–536 (2020), https://onlinelibrary.wiley.com/doi/full/10.1002/oby.22726.

12. Food and Drug Administration (FDA), Guidance for Industry Developing Products for Weight Management, Draft Guidance, Revision 1 (Feb. 2007), https://www.fda.gov/media/71252/download.

13. See H.R. Rep. No. 117–403, at 187 (2022), https://www.congress.gov/117/crpt/hrpt403/CRPT-117hrpt403.pdf; Sen. Bill Cassidy and Sen. Thomas R. Carper, Letter to Administrator Seema Verma (July 1, 2020), https://asmbs.org/app/uploads/2020/07/Sens-Cassidy-and-Carper-Letter-to-CMS-on-Obesity.pdf (accessed Aug. 19, 2022).

14. See, e.g., Centers for Medicare & Medicaid Services (CMS), National Coverage Determination, Pub. No. 100-3, Manual Sec. 100.1, Bariatric Surgery for Treatment of Co-Morbid Conditions Related to Morbid Obesity, https://www.cms.gov/medicare-coverage-database/view/ncd.aspx?ncdid=57&ncdver=4& (effective Sept. 24, 2013); Centers for Medicare & Medicaid Services (CMS), National Coverage Determination, Pub. No. 100-3, Manual Sec. 210.12, Intensive Behavioral Therapy for Obesity, (effective Nov. 29, 2011). https://www.cms.gov/medicare-coverage-database/view/ncd.aspx?NCDId=353 (effective Nov. 29, 2011).

15. See Alfaris, N., Aronne, L.J., et al., The Utility of Weight Loss Medications After Bariatric Surgery for Weight Regain or Inadequate Weight Loss: A Multi-Center Study, 13(3) Surgery for Obesity and Related Diseases (Mar. 2017), https://pubmed.ncbi.nlm.nih.gov/27986587/; Aminian, A., Brethauer, S.A., et al., Efficacy of Adjuvant Weight Loss Medication after Bariatric Surgery, 14(1), Surgery for Obesity and Related Diseases (Jan. 2018), https://pubmed.ncbi.nlm.nih.gov/29287757/; Anderson, W.A., Apovian, C.M., et al., The Mitigating Effect of Phentermine and Topiramate on Weight Regain After Roux-en-Y Gastric Bypass Surgery, 28 Obesity 1023–1030 (May 22, 2020), https://onlinelibrary.wiley.com/doi/10.1002/oby.22786; Hoagland, G.W., Lovegrove, M., et al., Expanding Access to Obesity Treatments for Older Adults, Bipartisan Policy Center (Feb. 2022), https://bipartisanpolicy.org/wp-content/uploads/2022/02/BPC_ExpandingAccessToObesityFinal.pdf (estimating $248 billion in annual medical expenditures related to obesity in 2020); Batsis, J.A., Bynum, J.P.W., Uptake of the Centers for Medicare and Medicaid Obesity Benefit: 2012–2013 24(9) Obesity 1983–88 (Sept. 24, 2016), https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5003721/ ("While there has been a small, steady rise in the use of the CMS Medicare Obesity benefit, utilization and uptake across the United States remains very low, and is not correlated with high obesity rates.").

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