Abstract/Overview

When Medicare conditions coverage of emerging medical technologies on additional evidence development, policymakers must balance the value of the evidence generated against study burden and patient access. These tradeoffs are largely shaped by the specific study parameters CMS deems acceptable. CMS’s recent endorsement of “fit-for-purpose” (FFP) studies — reinforced through updated policies finalized in August 2024 alongside the Transitional Coverage for Emerging Technologies (TCET) pathway — signals a more flexible, intentional approach to evidence generation that recognizes diverse data sources, study designs, and the need for prospective scientific rigor.

In this first part of the article, we propose a structured framework for FFP study design and data source selection within the context of coverage with evidence development (CED). The framework is intended to help resolve uncertainty about novel technologies in the Medicare population while minimizing unnecessary data collection and avoiding undue restrictions on beneficiary access. Click to read the second part of the article, which illustrates how the framework is applied retrospectively to CMS’s use of CED for CAR-T therapies and monoclonal antibodies for Alzheimer’s disease, and recommendations are offered to address operational and policy challenges. Together, these analyses aim to inform CMS’s forthcoming FFP guidance and advance policy discussions on evidence development for emerging technologies.