FDA Introduces Guidance to Expedite Rare Disease Drug Approvals

The FDA shared a draft plan on Wednesday that seeks to guide rare disease drugmakers on how to design their clinical trials in order to potentially speed up drug approvals.

The draft guidance provides recommendations to drug sponsors who are planning clinical trials of cell and gene therapy products intended for use in a disease or condition that affects a small population, the Food and Drug Administration said in a notice in the Federal Register.

“The recommendations are intended for sponsors developing CGTs intended for use in small populations to leverage the use of innovative trial designs to simultaneously expedite ...