Expedited Programs: BIO Submits Comments to FDA on Expedited Programs for Serious Conditions—Drugs and Biologics

Dear Sir/Madam:
The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the Draft Guidance for Industry on Expedited Programs for Serious Conditions—Drugs and Biologics. BIO fully supported the expansion and modernization of FDA’s Accelerated Approval pathway and the enactment of the new Breakthrough Therapy Designation Process under the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA, P.L. 112-144). BIO believes that these and existing expedited approval pathways will help to foster the development of a new generation of modern, molecularly targeted therapies and provide for earlier patient access to safe and effective new medicines.
BIO represents more than 1,100 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, thereby expanding the boundaries of science to benefit humanity by providing better healthcare, enhanced agriculture, and a cleaner and safer environment.
BIO compliments FDA on a well-crafted Guidance on Expedited Programs for Serious Conditions. The FDA Draft Guidance will be helpful to stakeholders in explaining the procedures, eligibility criteria, and general features under each of the four expedited programs: Accelerated Approval, Breakthrough Therapies, Fast Track, and Priority Review. The Draft Guidance is an encouraging step in FDA’s implementation of these programs under FDASIA. We welcome an ongoing dialogue with FDA to further evaluate the scientific and medical considerations underlying each respective approval pathway and how to encourage the use of these programs for a broad array of serious and life-threatening diseases and conditions.
BIO is pleased to provide several general comments on the issues raised by the Draft Guidance, as well as specific line-by-line comments. In particular, we encourage FDA to:
  • Elaborate upon the enhanced flexibility provided by FDASIA for Accelerated Approval
  • Address unique issues associated with rare diseases under Accelerated Approval
  • Establish a systematic framework and evidentiary criteria for discussing Accelerated Approval and endpoint selection earlier in drug development
  • For Breakthrough Therapies and expedited program products with companion diagnostics, involve Center for Devices and Radiological Health (CDRH) senior staff in cross-disciplinary engagement during drug development
  • Adopt a risk-based, life-cycle approach to review of Chemistry, Manufacturing, and Controls (CMC) data and inspectional activities

BIO appreciates this opportunity to comment on the Draft Guidance for Industry on Expedited Programs for Serious Conditions—Drugs and Biologics. Specific, detailed comments are included in the following chart. We would be pleased to provide further input or clarification of our comments, as needed.