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Clinical Trials: BIO Comments on FDA Draft Guidance Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the Draft Guidance titled Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs.

August 6, 2019

Re: Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the Draft Guidance titled Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs.

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BIO applauds the FDA on the development of this important Draft Guidance and welcomes the recommendations to broaden eligibility criteria and increase enrollment of underrepresented populations in clinical trials. BIO believes that improving diversity of clinical trial populations is an important issue and agree with the Agency’s view that adopting more inclusive enrollment practices will open clinical trials to a diverse participant population reflective of the population that will use the drug.

While we understand the FDA’s goal of improving representativeness of clinical trial populations, we believe that policy development on this topic requires a more comprehensive approach. For example, broadening eligibility criteria, even if scientifically and clinically appropriate, may have profound impact on drug development feasibility and timeliness and, ultimately, patient access to needed treatment options. When the broadening of eligibility criteria is considered, the urgency of addressing an unmet need, the perspectives of patients and caregivers, and the health of the drug development ecosystem in the therapeutic area should be thoroughly considered. BIO encourages the FDA to present this drug development challenge and trade-off less simplistically in the introduction section of the Draft Guidance, particularly when robust and effective mechanisms, for example, use of real-world evidence, are emerging as ways to complement or fill in gaps in knowledge achieved through traditional clinical trials.

BIO also suggest FDA more clearly define what is mean by population diversity and more clearly delineate how their recommendations apply to different patient groups and the prevalence of disease. In addition, we particularly appreciate the Agency's recognition of the need to exclude patients with potentially negative benefit/risk and complex cases that, due to confounding, make it difficult to assess the safety profile of the product. In order to increase further patient representativeness, BIO suggests that the FDA consider allowing the enrollment of younger children without providing a requirement for a specific number to be enrolled, then pediatric trials could be more inclusive and provide additional safety information for use in children.

Lastly, BIO would like to take this opportunity to thank the FDA Office of Minority Health and Health Equity (OMHHE) for continuously being open to collaborate with Sponsors. This collaborative spirit and willingness to engage in dialogue with a diverse group of stakeholders is a promising and commendable approach that FDA is taking to tackling this challenging issue. BIO would welcome the opportunity to collaborate with OMHHE on ways of partnering on enhancing clinical trial diversity.

BIO appreciates this opportunity to submit comments on the Draft Guidance titled Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs. 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.