Medicare Coverage With Evidence Development for Drugs Approved for Alzheimer’s Disease: Which Study Designs and Data Sources Would Best Answer Medicare’s Questions?


Moderator: Sean Tunis, MD, MSc, Center for the Evaluation of Value and Risk in Health, Tufts University, Boston, MA, USA
Panelists: Maria Glymour, ScD, MS, Dept of Epidemiology and Biostatistics, UCSF, San Francisco, CA, USA; Nirosha Mahendraratnam Lederer, PhD, MSPH, Aetion, Inc, Washington, DC, USA; Emily Zeitler, MD, MHS, Dartmouth-Hitchcock Medical Center, Lebanon , NH, USA


Several disease modifying therapies for Alzheimer’s Disease are likely to be approved by the FDA within the next 1-3 years, showing direct evidence of cognitive benefits.

The Medicare program has indicated that these drugs will only be covered when patients participate in clinical studies designed to evaluate real world benefits and risks of these drugs.

Panelists will debate three different approaches to generate this evidence: pragmatic clinical trials, clinical registries, and secondary analysis of routinely collected healthcare data


In 2021, the FDA approved the first disease-modifying therapy for Alzheimer’s Disease (AD) using the accelerated approval pathway, based on the surrogate marker beta-amyloid. In 2022, CMS issued a national coverage decision that limited coverage of these FDA-approved drugs to patients enrolled in randomized controlled trials, citing the lack of direct evidence of cognitive benefit. CMS stated that future drugs approved with direct evidence of cognitive benefits would be covered under a coverage with evidence development (CED) policy.

Medicare’s CED policy requires that patients receiving these therapies are included in “prospective comparative studies” as a condition of payment, allowing some flexibility in study design and data source. The participants in this issues panel will debate the pros and cons of three different approaches to evidence generation for CED studies of drugs for AD.

The moderator will provide a 10 minute summary of the CMS policy, followed by the 3 panelists describing their recommended evidence generation approach (12 minutes each). The remainder of the time will be allocated to moderated dialogue with the panelists and audience members.

This issues panel will appeal to those interested in understanding different approaches to studying the real world benefits and risks of novel therapies.




Health Policy & Regulatory