Placeholder Banner

BIO Comments on FDA Draft Guidance on Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model

February 5, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.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.

Download Full Comments Below
BIO Letter Pediatric Rare Diseases A Collaborative Approach For Drug Development Using Gaucher Disease As A Model FDA-2017-N-6476
Read full comment letter below
Discover More
The undersigned food, agriculture, and seafood trade associations respectfully urge the expeditious nomination of the Chief Agricultural Negotiator at the Office of the U.S. Trade Representative. America’s farmers, ranchers, processors, exporters…
The undersigned trade associations and private sector organizations write to express our strong support for the Endless Frontier Act, sponsored by Senate Majority Chuck Schumer (D-NY) and Senator Todd Young (R-IN), which would provide much needed,…
Dr. Michelle McMurry-Heath, along with a group of business and policy leaders, sends an open letter to President Biden urging him "to demonstrate decisive U.S. leadership" to combat COVID-19 in Latin America, Europe, Asia, and Africa…