FDA Sets Priorities For 2016: Quality, Participation, Transparency
By Ed Miseta, Chief Editor, Clinical Leader
As the pharma industry enters 2016, its regulatory agency in the U.S. believes progress has been made on clinical trials. Writing for FDA Voice, Barbara Buch, the Associate Director for Medicine in FDA’s Center for Biologics Evaluation and Research, notes there are few responsibilities the agency takes more seriously 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,” she says. One of her goals is to continue to improve clinical outcomes in 2016.
Last year the FDA took action to support the inclusion of diverse populations in trials. Sex, age, and race/ethnicity are just three demographics the agency took a close look at in regard to trial data. In August, the agency published an Action Plan designed to address three specific priorities: improving the quality of subgroup data collection, eliminating barriers that can result in decreased trial participation rates, and increasing the transparency of that subgroup data. Progress has already been made in each targeted area.
Quality Is Job 1
Perhaps the one area where the most progress has been made is in quality. Via staff training and outreach to stakeholders, FDA has updated and/or finalized guidance on demographic subgroup data. The agency’s Integrated Summary of Effectiveness (ISE) - Guidance for Industry, published in October 2015, is a 20-page document that describes FDA’s thinking regarding information the industry should include in an ISE, so as to provide an analysis with insights beyond those that are observable in individual trials. The guidance contains sections on the analysis of study designs, analysis of effectiveness results, comparison of results in subpopulations, and more.
Guidance on the Evaluation of Sex-Specific Data in Medical Device Clinical Studies, originally issued in August 2014, was updated and made available in conjunction with a training webinar for industry and FDA.
The Office of Minority Health (OMH) developed a plan to support research projects and lead to a better understanding of clinical outcomes in racial/ethnic 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) all modified their clinical review templates. A new CDER review process encourages reviewers to watch for inappropriate inclusion and exclusion criteria, while CDRH and CBER both modified statistical reviewer templates to include analysis of subgroup information. CBER and CDER also incorporated discussions on diverse inclusion and subgroup participation and analysis into pre-application submission meetings.
Also falling under the heading of quality was a MedWatch forms update standardizing the collection of demographic information on possible adverse events that occur after products are made available to the U.S. market. The Office of Women’s Health (OWH) posted a plan for women’s health research and funded two research projects to improve data quality in demographic subgroups and examine sex-specific outcomes with cardiac resynchronization therapy.
Increase Subgroup Participation And Transparency
To increase the amount of diversity that exists in clinical trials, OWH and NIH collaborated on a workshop titled Meet the Faces of Clinical Research: Beyond Inclusion, which features trial participants discussing the importance of patient diversity. FDA is also making demographic information from trials available to consumers through an online webpage, while a Public Meeting and a clinical trials workshop discussed the importance of minority health disparities and assessing safety and efficacy on diverse populations.
The effort to establish greater transparency involved establishing a Language Access Plan Working Group to implement communication strategies to reach under-represented subpopulations, launching a pilot program to make demographic information available to physicians and the public for Biologics License Applications, and modifying templates for certain documents posted to the FDA website for medical device approvals, to make sure demographic information is included.
The effort does not end here. To continue to make the advancements necessary to bring greater patient diversity to trials, all stakeholders will need to be more vigilant moving forward. According to Buch, FDA looks forward to continuing the momentum gained in 2015.