Patient Registries for Medicare Coverage of New Alzheimer’s Drugs

In July 2023, the Food and Drug Administration (FDA) gave traditional approval to lecanemab (LEQEMBI). Lecanemab, which received accelerated approval earlier in 2023, is a monoclonal antibody that is designed to remove beta-amyloid plaque from the brains of patients with Alzheimer’s disease. Public Citizen’s Health Research Group strongly opposed this decision.[1] We have classified lecanemab as a Do Not Use drug because the clinical benefits are uncertain, and the health risks related to brain swelling and brain bleeding are substantial.[2] Lecanemab is an expensive drug that neither cures nor reverses Alzheimer’s disease.

The Centers for Medicare & Medicaid Services (CMS) is an independent federal agency and has the legal authority to limit coverage of FDA-approved products. The relevant standard is whether a therapy is “reasonable and necessary” for coverage among Medicare beneficiaries.[3] For lecanemab and other similar medications that the FDA may eventually approve, coverage is available for people enrolled in Medicare who have mild cognitive impairment or mild dementia from Alzheimer’s disease, documented evidence of beta-amyloid plaque on the brain and whose physician enrolls them in a qualifying patient registry.[4] Physicians are required to submit specific information to the registry including the patient’s diagnosis and treatment, certain test results, accompanying use of some other medications and evidence of adverse events, such as brain swelling or bleeding. Data are to be submitted at six-month intervals for up to 24 months.[5] CMS has established a national patient registry, which is available without charge, and has also approved other qualifying studies.

The CMS registry was designed to minimize physician burden in providing information and privacy and other potential patient concerns. Importantly, there is no control group of untreated patients. Although the data are unlikely to be useful in generating evidence about lecanemab’s effectiveness, they may have some value for assessing the frequency of adverse events, and how soon they develop. At a minimum, the registries should be publicly accessible, and their data promptly published and available to CMS and qualified researchers for independent analyses.

It is unusual for CMS to even require participation in a patient registry as a condition of Medicare coverage. Nonetheless, the registry approach represents a missed opportunity for the collection of meaningful information about lecanemab and its use. CMS could—and should—have made coverage dependent on patient’s participation in randomized clinical trials, which are the gold standard by which the effectiveness of drugs is evaluated.[6] CMS had required that approach, which is known as coverage with evidence development, when the FDA granted lecanemab accelerated approval, but abandoned it when the drug received full approval.

Although the drug company and industry-supported advocacy groups would have howled if the rules for Medicare coverage of lecanemab had not been eased, weakening the requirements was a mistake. CMS pays for medical products or procedures that are “reasonable and necessary” for Medicare beneficiaries. Registries are not an effective means to identify and remove ineffectual and dangerous drugs from the market.[7] Medicare’s registry approach to data collection for new Alzheimer’s drugs is a missed opportunity for the collection of definitive evidence about whether patients are more likely to have their lives improved or worsened by the medications.
 

References

[1] Steinbrook R. Outrage of the Month: FDA approval of lecanemab for Alzheimer’s disease. Public Citizen’s Health Letter. August 2023. https://www.citizen.org/article/outrage-of-the-month-fda-approval-of-lecanemab-for-alzheimers-disease/ Accessed October 31, 2023.

[2] Lecanemab for Alzheimer’s disease: do not use. Worst Pills, Best Pills News. October 2023.

[3] Ross CJR, Kesselheim AS. Authority of Medicare to limit coverage of FDA-approved products: legal and policy considerations. JAMA Intern Med. 2023;183(9):999-1004.

[4] Centers for Medicare & Medicaid Services. Fact Sheet CMS announces new details of plans to cover new Alzheimer’s drugs. June 22, 2023. https://www.cms.gov/newsroom/fact-sheets/cms-announces-new-details-plan-cover-new-alzheimers-drugs Accessed October 31, 2023.

[5] Odouard IC, Socal MP, Anderson GF. Role of registries in Medicare coverage of new Alzheimer’s drugs. JAMA. 2023;330(14); 1331-1332.

[6] Crosson FX, Redberg RF. Statutory authority if Medicare coverage decisionsCMS is an independent federal agency. JAMA Intern Med. 2023;183(9):910-911.

[7] Ibid.