According to a recent FierceBiotech article, the FDA's Center for Biologics Evaluation and Research director, Peter Marks, revealed that the agency is considering ways to expand accelerated approvals for drugs targeting rare diseases. Marks stated that the FDA now has a better understanding of the data needed to grant accelerated approvals, allowing more drugs to potentially receive quicker authorization. Accelerated approvals rely on surrogate endpoints or biomarkers, which can predict clinical benefit more easily than traditional clinical endpoints.
Marks emphasized that the FDA doesn’t require 100% certainty between accelerated approval endpoints and clinical endpoints, as long as there is compelling scientific evidence supporting the drug's potential efficacy. Patient advocates and drug developers have been pushing for the broader use of accelerated approval, especially with the rise of gene therapies, and Marks' comments indicate a positive step towards making this a reality for rare disease patients.