By: Barbara D. Buch, M.D.
There are few responsibilities at FDA more important than reviewing the design and outcomes of clinical trials. Understanding the science behind the trials — and the individuals included in them — helps us to ensure that the medical products we approve are safe and effective.
Last year, FDA took important steps to support the inclusion of diverse populations in clinical trials. Following Congress’s directive in Section 907 of the Food and Drug Administration Safety and Innovation Act, FDA is looking more closely at the sex, age, and race/ethnicity data that are collected in clinical trials.
In August, FDA published an Action Plan designed to address three specific priorities: improving the quality and comprehensiveness of demographic subgroup data collection, reporting and analysis; identifying and eliminating barriers for increased participation in clinical trials; and improving the transparency of subgroup data.
We’ve come far in achieving this plan. As we begin 2016, I want to outline our progress in preparation for the next important milestone: a public meeting on this topic on February 29.
Priority 1 – Quality
- FDA updated and/or finalized relevant guidance on demographic subgroup data, as illustrated by these two examples of FDA staff training and/or outreach to external stakeholders:
- Integrated Summary of Effectiveness: Guidance for Industry
- Evaluation of Sex-Specific Data in Medical Device Clinical Studies, which was made available in conjunction with a training webinar for industry and FDA.
- The Office of Minority Health (OMH) developed a plan that supports specific research projects and leads to better understanding of medical product clinical outcomes in racial/ethnic demographic subgroups.
- The Center for Devices and Radiological Health (CDRH), the Center for Drug Evaluation and Research (CDER), and the Center for Biologics Evaluation and Research (CBER) modified their clinical review templates:
- CDER developed a review process that encourages reviewers to watch for inappropriate clinical trial exclusion and inclusion criteria; accompanying training emphasizes the need to include broad population diversity in clinical trials.
- CDRH and CBER modified statistical reviewer templates to include analysis of demographic subgroup information.
- CBER and CDER incorporated discussions on diverse inclusion and subgroup participation and analysis into pre-application submission meetings with industry.
- FDA updated its MedWatch forms to standardize collection of demographic information on possible adverse events that occur after medical products are broadly available on the U.S. market.
- And a few days ago, the Office of Women’s Health (OWH) posted their Research Roadmap and its strategic plan for women’s health research. OWH also funded two research projects:
- Methods to improve data quality in demographic subgroups
- Examination of sex-specific outcomes with cardiac resynchronization therapy.
Priority 2 – Participation
- OWH collaborated with NIH on a workshop, Meet the Faces of Clinical Research: Beyond Inclusion, which featured clinical trial participants and researchers discussing the importance of diversity in clinical trials.
- FDA is making demographic information from clinical trials more easily available to consumers through its easy-to-read online Drug Trials Snapshots webpage and a corresponding article for consumers.
- The Office of Minority Health and the Institute of Medicine convened a Public Meeting to discuss minority health disparities and clinically meaningful differences.
- FDA and The Johns Hopkins University co-sponsored a clinical trials workshop, Assessing Safety and Efficacy for a Diverse Population.
Priority 3 — Transparency
- FDA established a Language Access Plan Working Group designed to implement communication strategies sensitive to the needs of under-represented subpopulations, focusing on language access and health literacy.
- CBER launched a transparency pilot program to make demographic information available to physicians and the public for original Biologics License Applications.
- CDRH modified templates for certain documents that are posted to the FDA website upon approval of certain medical devices to ensure that demographic information is consistently included.
We’ve certainly made progress, and will continue the forward momentum in the years to come. And we will need the continued investment of our stakeholders and partners.
We look forward to continuing this important and productive conversation with you next month at the public meeting.
Barbara D. Buch, M.D., is the Chair of the 907 Steering committee and the Associate Director for Medicine in FDA’s Center for Biologics Evaluation and Research