BIO Comments on FDA Draft Guidance on Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.
BIO appreciates the FDA’s intended goal with the guidance – to reduce the total number of children needed for enrollment in clinical studies – and applauds the agency’s willingness to accept innovative trial designs to limit the number of patients in the control arm and thereby improve trial efficiency.
However, BIO has concerns with the guidance, particularly with the FDA’s suggestion for the use of multi-arm, multi-company clinical studies, which have inherent limitations and challenges. In the comments, BIO details these concerns and provides suggestions for clarifying and improving the guidance.