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BIO Submits Comments on FDA Draft Guidance, Rare Diseases: Common Issues in Drug Development

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the Draft Guidance on Rare Diseases: Common Issues in Drug Development. 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’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of diseases, and to prevent diseases in the first place. BIO commends the FDA for its efforts to update the 2015 Draft Guidance on Rare Diseases: Common Issues in Drug Development. The updated Draft Guidance serves as an important communication tool between the FDA and Sponsors on issues pertaining to rare disease drug development. Such guidance ensures that Sponsors have appropriate information for developing new therapies for rare disease patients, especially given that many rare diseases still do not have an FDA approved treatment. In the following pages of this letter, BIO has included general comments as well as line edits that we believe will make the Draft Guidance more useful for rare disease drug developers.

April 1, 2019 


Dockets Management Staff (HFA-305) 
Food and Drug Administration  
5630 Fishers Lane, Rm. 1061  
Rockville, MD 20852

 
Re: Docket No. FDA–2015-D-2818: FDA Draft Guidance, Rare Diseases: Common Issues in Drug Development. 


Dear Sir/Madam: 


The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the Draft Guidance on Rare Diseases: Common Issues in Drug Development. 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’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of diseases, and to prevent diseases in the first place. 
BIO commends the FDA for its efforts to update the 2015 Draft Guidance on Rare Diseases: Common Issues in Drug Development. The updated Draft Guidance serves as an important communication tool between the FDA and Sponsors on issues pertaining to rare disease drug development. Such guidance ensures that Sponsors have appropriate information for developing new therapies for rare disease patients, especially given that many rare diseases still do not have an FDA approved treatment. In the following pages of this letter, BIO has included general comments as well as line edits that we believe will make the Draft Guidance more useful for rare disease drug developers. 


Request for Additional Detail Pertaining to Drug Development Tools and Innovative Approaches and Trial Designs: 
While BIO appreciates the FDA’s work to update this Draft Guidance, we note that the Draft Guidance lacks detail pertaining to the use of new drug development tools that have been prioritized by the Agency in recent years, including, real-world evidence, innovative clinical trial designs, and development and use of biomarkers, among others. Additionally, there are several elements of drug development described within the Draft Guidance for which the Agency does not seem to provide specificity regarding rare diseases. 


For example, the sections of the Draft Guidance focused on historical controls appears contrary to the FDA’s overall sentiment outlined in the FDA’s Framework on Real-World Evidence1 where the Agency appears to be open to exploring the use of historical controls. Generally, the draft guidance implies that the FDA has taken the following positions...