FDA’s Rare Disease Evidence Principles (RDEP): What We Know So Far
Authors:
Karen Sturgeon
Principal Consultant
Nohman Mohammad
Consultant
Introduction
The FDA has announced a new review process for rare diseases based on a molecule’s “plausible mechanism” which would allow the FDA to consider approval on a conditional basis. This initiative is designed to help speed up the approval of drugs to treat rare diseases and provide clear guidance on how evidence of efficacy can be demonstrated with a small patient population, removing the challenge of substantial safety and efficacy requirements.
Eligibility
The FDA’s Centers for Drug Evaluation and Research (CDER) and Biologics Evaluation and Research (CBER) have jointly implemented a new regulatory pathway called the Rare Disease Evidence Principles (RDEP). To be eligible:
The investigational product’s indication must be for an ultra-rare genetic condition affecting fewer than 1000 patients and
Targeted at either replacing or correcting the genetic defect responsible for the disease.