BIO Submits Comments Re: FDA Rare Diseases Guidance

 

Dear Sir/Madam:

 

The Biotechnology Industry Organization (BIO) appreciates the opportunity to provide feedback in response to the Food and Drug Administration’s (FDA’s) Draft Guidance for Industry, entitled “Rare Diseases: Common Issues in Drug Development” (Draft Guidance).

 

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.

 

 

BIO shares FDA’s goal of improving the efficiency and success of development programs that target the treatment or prevention of a rare disease. We agree that the issues encountered in this space “are frequently more difficult to address in the context of a rare disease,” and thus, BIO appreciates the Agency’s attention—in the form of issuing this Draft Guidance—to drug development challenges manufacturers face.2 While we agree that therapies to treat rare diseases should be held to the same stringent regulatory approval standard imposed for therapies to treat more common conditions, BIO echoes the Agency’s recognition that regulatory flexibility is all the more crucial to “creat[ing] successful drug development programs that address the particular challenges posed by each disease.”3 Additionally, in the context of the Draft Guidance, BIO would like to reiterate that regulatory flexibility is necessary, but not entirely sufficient, to improve the efficiency and success of rare disease development programs. This flexibility must be provided uniformly throughout the Agency’s review divisions to support rare disease drug development across therapeutic areas.