Abstract/Overview

Medicare has recently endorsed the concept of “fit-for-purpose” (FFP) studies to support ongoing evidence generation as a condition of coverage for selected emerging medical products. In part 1 of this article, we offered a framework to help apply a structured approach to FFP study design and data source selection in the context of coverage with evidence development (CED), which provides for Medicare coverage of selected emerging medical products with the condition that they are furnished in the context of Medicare-approved clinical studies.

In this article – or part 2 — we illustrate the potential impact of our framework by examining how it might have affected two applications of CED by the Centers for Medicare and Medicaid Services (CMS): the proposed use of CED in coverage of CAR-T therapy for oncology, and the use of CED in coverage of monoclonal antibody therapy for Alzheimer’s disease. We also offer suggestions for how Medicare may overcome some operational and policy barriers to the design and implementation of FFP studies.