Dear Sir/Madam:  The Biotechnology Innovation Organization (BIO) thanks to the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments regarding the Draft Guidance, Qualification Process for Drug Development Tools. 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 these diseases, or to prevent them in the first place. BIO commends the Agency for the development of the Draft Guidance on Qualification Process for Drug Development Tools (DDTs) as required by the 21st Century Cures Act. BIO believes that the Draft Guidance provides a useful framework for how new DDTs can be developed and used as well as the process for qualification of such tools, information that will be essential to Sponsors looking to qualify a DDT. BIO has included in this letter several recommendations for FDA’s consideration as the Guidance is finalized including flexibility in timelines, identification of meetings types, detail regarding rescinding or modification of DDTs,  alignment of CDRH regulation, use of external experts, and information on prioritization of review.  

February 11, 2020 Re: Docket No. FDA–2019-D-4751: Food and Drug Administration Reauthorization Act Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments regarding FDA’s Draft Guidance on Implementation of Pediatric Studies of Molecularly Targeted Oncology Drugs. 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 these diseases, or to prevent them in the first place. BIO thanks FDA for the development of the Draft Guidance on FDARA Implementation for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Section 505B of the FD&C Act. It is essential that there is clear and comprehensive guidance for Sponsors on pediatric studies of molecularly targeted oncology drugs so that Sponsors can meet the requirements outlined in statute beginning in August 2020. BIO would like to address some key issues associated with interpretations of the underlying statute that are not entirely clear within the Draft Guidance. Two issues of note relate to the pediatric investigation under section 505B(a)(3), as well as the circumstances surrounding the deferral and/or waiver rules governing applications for drugs/biologics active against molecular targets on the substantial relevance list. We have previously raised these concerns in a letter to FDA’s Office of Chief Counsel and we would like to reiterate our positions in an effort to clarify both issues in any resulting final guidance. Set forth below we elaborate on each of these issues more substantively and provide several…