FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials

Earlier this month the U.S. Food and Drug Administration issued a draft guidance intended to help cut the cost and development time of rare disease therapies. The guidance focused on the potential to use multi-arm, multi-company trials to reduce the total number of patients needed to evaluate experimental therapeutics targeting the same indication. We spoke to James Valentine, associate with the law firm Hyman, Phelps & McNamara, about the draft guidance, how broadly applicable it might be, and what it says about the direction of the FDA in terms of addressing issues of concern to the rare disease community.