RDCC Supports Policy Proposals That Recognize the Potential of Rare Disease Therapies and Reinforce the Role of the Accelerated Approval Pathway

FDA’s accelerated approval pathway recognizes that a “one-size-fits-all” traditional model cannot always work – especially for serious and unique rare diseases with very small population sizes that progress slowly and variably from patient to patient. Therapies approved through the accelerated approval pathway are subject to the same stringent, evidence-based clinical review and approval standards as the traditional FDA approval process, as well as to post-approval confirmatory trials to verify clinical benefit.

While less than 10% of all accelerated approvals in its 30-year history have been used for nononcology rare diseases, today’s increased understanding of rare disease and advances in targeted drug development are making it possible to realize the promise of accelerated approval for rare diseases where current treatments don’t yet exist.

As policymakers consider policy proposals that impact the trajectory of rare disease treatment development, we advocate for an approach that considers the unique circumstances of rare diseases and caution against punitive measures that could chill future investment and development of rare disease treatments.

Click here to download this overview on the benefits of the accelerated approval pathway for rare diseases.