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RMAT: BIO Submits Comments on FDA Draft Guidance Expedited Programs for Regenerative Medicine Therapies for Serious Conditions

Re: Docket No. FDA-2017-D-6159: Expedited Programs for Regenerative Medicine Therapies for Serious Conditions

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments to the Draft Guidance on Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.

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BIO commends FDA on this Draft Guidance which provides important information on the expedited programs available to sponsors of regenerative medicine therapies for serious conditions.

General Comments

  • BIO recommends FDA provide further guidance to sponsors on which designation, RMAT or Breakthrough Therapy, is more appropriate for different types of programs.
  • The Agency should consider including an explicit statement similarly to the FDA’s FAQ on Breakthrough Therapies[1] clarifying that a Sponsor can submit a request for RMAT designation for multiple indications of the same product. 
  • BIO believes strong communication between Sponsors and the Agency are critical in successful and efficient regulatory review process. To that end BIO recommends adding an opportunity for preliminary RMAT Designation Request Advice, similar to the Preliminary Breakthrough Therapy Designation Request Advice.
  • BIO also recommends the Agency provide further clarity on how accelerated approval and post approval requirements may be used for RMAT designated products. Specifically, sponsors should be encouraged to seek stage-appropriate advice through meetings with FDA on topics such as:
  • FDA’s interpretation of whether a surrogate endpoint is reasonably likely to predict long-term clinical benefits.
  • Early discussion of FDA’s scientific concerns that could eventually lead to Post-Marketing Requirements.
  • BIO suggest that Section V, Interactions Between Sponsors and CBER Review Staff, highlight the continued collaboration between CBER Review Staff and other FDA staff with expertise on specific topics, such as endpoints, companion diagnostics, and clinical aspects of disease.

BIO appreciates this opportunity to submit comments on the Draft Guidance on Expedited Programs for Regenerative Medicine Therapies for Serious Conditions. We provide additional specific, detailed comments to improve the clarity of the Draft Guidance in the following chart. We would be pleased to provide further input or clarification of our comments, as needed.