Finally, there are questions about the product’s cost-effectiveness, given its announced price of $56,000, several-fold higher than the Institute for Clinical and Economic Review’s initial estimate of the upper bound of $8,300 for aducanumab to represent reasonable value for money. Aducanumab’s potential side effects, including brain swelling and bleeding, also elicit questions about its safety. However, the company has seven to nine years to submit results from this confirmatory trial.Īlthough the clinical trials focused on patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease dementia, the FDA granted the product a broad label, raising questions about which patients are most likely to benefit from this therapy. The drug’s manufacturer, Biogen, is required to perform a clinical study to confirm that the drug does in fact slow cognitive decline. The FDA granted aducanumab accelerated approval based on a surrogate endpoint, reduction in amyloid beta plaques (sticky protein build ups that form in the spaces between nerve cells), although the publicly available data linking this surrogate to improvements in cognition are limited. In judging whether and how to cover aducanumab, CMS confronts several issues: One of the therapy’s Phase 3 studies, ENGAGE, failed to meet its primary endpoint of reduced cognitive decline from baseline on the Clinical Dementia Rating-Sum of Boxes the identically designed EMERGE trial showed statistically significant but clinically modest results. While others have written generally about Medicare’s options for aducanumab, including possible action from CMS’s Center for Medicare and Medicaid Innovation, we elaborate on the design of CED studies, drawing on lessons from historical CMS decisions. In the wake of the Food and Drug Administration’s (FDA’s) controversial decision to approve aducanumab for Alzheimer’s disease, how will, and how should, Medicare respond? We propose a coverage with evidence development (CED) option for the Centers for Medicare and Medicaid Services (CMS), and we examine how the agency’s past decisions on high-price technology may offer a guide.

Evidence reviewer for mac