Applying Real-World Data and Real-World Evidence for Accelerated Approvals and Coverage Decisions

Executive Summary

Medical products granted accelerated approval by the United States Food and Drug Administration (FDA), and the evidence guiding such approvals, have been subject to controversy in recent years. Meanwhile, there is an ongoing need to deliver rapidly effective products to patients with unmet medical needs while also addressing the need for demonstrable safety and effectiveness in the real world. In parallel, federal agencies are developing guidance on the use of real-world data (RWD) to drive initial and confirmatory evidence generation that supports evaluation of safety and effectiveness. This paper explores the landscape of accelerated approval decisions by FDA, related payer considerations, and three medical product use cases (Elevidys, Vijoice, and Keytruda). In doing so, it offers considerations, based on current events, for RWD submitted as part of a total evidence package to inform both regulators and payers as they evaluate therapies within the FDA accelerated approval pathway. Such considerations warrant a close examination of how RWD sources address shared evidentiary needs among regulatory and payment stakeholders. In this white paper, we specifically examine real-world endpoint selection, generalizability of evidence, approaches for registries and data repositories, point-of-care trials, external control arms, and private payer considerations in the context of leveraging RWD sources.