ICER Whitepaper on Successfully Navigating the FDA’s Accelerated Approval Pathway

Today, ICER (the Institute for Clinical and Economic Review) announced a new whitepaper on understanding and navigating the FDA’s accelerated approval pathway (AAP). The whitepaper discusses which drugs qualify for AAP, the use of surrogate endpoints, challenges associated with the AAP process, and the outlook on drug policy reforms. One major challenge is the lack of confirmatory studies. The report also notes that the FDA is considering how to utilize RWD (real world data) in its regulatory decisions.

“According to ICER President Dr. Steven Pearson, “Since 1992, the FDA’s Accelerated Approval Program has brought important new treatments to patients faster than would have been possible through the traditional approval process. The successes are many, and the benefits for patients overall have been substantial. But criticisms have emerged as well, including concerns that the FDA has lacked consistency in its application of evidentiary standards, and that the incentives and procedures in place today have proven inadequate to get high quality confirmatory trials completed in a reasonable time frame,” adding, “So today, after nearly three decades of experience, it is a good time to take stock of where the AAP is, and what might be done to strengthen it. No policy reform options come without potential downsides, and the goal of our white paper is to outline the potential benefits and risks of major policy options.” Read the whitepaper here.

(Source: ICER, 4/26/21)