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BIO Comments on FDA Draft Guidance Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings

December 17, 2019

Re: Docket No. FDA–2018-D-3268: FDA Draft Guidance, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings.

Dear Sir/Madam:                                                                      

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the Draft Guidance for industry entitled Rare Diseases and Early Drug Development and the Role of Pre-IND Meetings.

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 applauds the FDA’s initiative to continue to advance drug development and review for therapies to treat rare diseases by releasing the Draft Guidance on Rare Diseases: Early Drug Development and the Role of the Pre-IND Meetings. It is estimated that there are 7,000 identified rare diseases, and only 5% of those rare diseases have an approved therapy[1], underscoring the need for new therapies. The Draft Guidance provides important information to drug developers regarding Pre-IND meetings to support better the consistency, transparency, efficiency of drug development, and the review of rare disease therapies. BIO has included comments below as line edits for the FDA’s review as the Draft Guidance is finalized.

BIO appreciates the Agency’s consideration of our comments regarding FDA’s Draft Guidance, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings. We would be pleased to provide further input or clarification of our comments, as needed.

 

[1] National Institutes of Health, FAQs About Rare Diseases