This paper aims to create a clearer understanding of both the opportunities and challenges inherent in the FDA’s accelerated approval pathway (AAP), and to present an analysis of the potential risks and benefits of a range of reform options that policymakers may consider in efforts to strengthen the AAP moving forward.   

Date of publication: April 2021

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Final Documents

Below you will find the final documents from the policy paper review process:

“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. From various points on the spectrum, concerns are also raised that patients may not be getting fair access to these drugs, while some question whether the high prices for most of the drugs approved through this pathway are merited given the level of uncertainty about their effectiveness at launch. 

“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.”