Clinical Trials: BIO Comments on FDA Draft Guidance Adaptive Designs for Clinical Trials of Drugs and Biologics

November 28, 2018

Re: Docket No. FDA-2018-D-3124: Adaptive Designs for Clinical Trials of Drugs and Biologics; Draft Guidance for Industry

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Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments to the Draft Guidance on Adaptive Designs for Clinical Trials of Drugs and Biologics.

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.

This important Draft Guidance was well written and provides important information to allow companies to use adaptive trial designs. Of note, the draft guidance suggests in several places that applicability of trial adaptations that could allow earlier stopping for efficacy may be limited by the need to collect adequate safety data. The Draft Guidance would benefit from additional discussion as to the feasibility to trigger early submission based on reaching the primary efficacy endpoint, while also having additional enrolment and/or follow up to generate the requisite amount of safety data. We have also provided additional specific, detailed comments to improve the clarity of the Draft Guidance in the following chart.

BIO appreciates this opportunity to submit comments on the Draft Guidance on Adaptive Designs for Clinical Trials of Drugs and Biologics. We would be pleased to provide further input or clarification of our comments, as needed.