Medical product sponsors should focus on the overarching goal of enrolling diverse patient populations in clinical trials, rather than getting bogged down in differing terminology around race and ethnicity, according to Lola Fashoyin-Aje, MD, of the US Food and Drug Administration’s Oncology Center of Excellence.
“The big picture here is that we want to enroll a population that reflects the diversity of the population that will use the drug once it’s approved,” Fashoyin-Aje said at a Convergence 2022 session on strategies to enhance diversity in clinical trial populations. “Race and ethnicity is just one factor among many other factors to be considered in enrolling that diverse population.”
In April 2022, FDA issued draft guidance on developing a Race and Ethnicity Diversity Plan to enroll adequate numbers of participants in clinical trials from underrepresented racial and ethnic populations in the United States. However, in public comments, industry has called for information on how the diversity plans can be applied to global clinical trials as well as greater detail on the type of data that should be used to assess adequate enrollment of underrepresented populations.
During the session, one attendee asked for clarification from FDA about how sponsors running global trials should account for the different ways that patients self-report their race and ethnicity, especially when it is at odds with the goals of the sponsor’s diversity plan.
Fashoyin-Aje said the best practice for collecting data on race and ethnicity is by self-report and that variations by country are expected. Sponsors should do their due diligence in enrolling a diverse population and collecting race, ethnicity and ancestry information at a granular level. “This is not a new problem,” she said.
“We have not been focusing on this issue in this comprehensive a fashion to really implement sustainable, action-driven strategies to enroll a diverse population,” Fashoyin-Aje said. “There’s a lot of learning to be done here, on both sides. The regulators don’t have all the answers; the sponsors don’t have all the answers.”
Diversity plan guidance
In the draft guidance, FDA recommends that sponsors submit a diversity plan for clinical studies that are intended to support a market submission for drugs, biologics and medical devices.
Diversity plans related to drug applications should be submitted “as soon as practicable” but not later than the time sponsors seek advice on a pivotal trial, typically after phase 2, said CDR Mathilda Fienkeng, PharmD, director of the Division of Medical Policy Development at FDA’s Center for Drug Evaluation and Research. For devices, the diversity plan should be submitted as part of the investigational plan included in the investigational device exemption (IDE) application. The diversity plan should also be part of the marketing application and include a description of the successes and challenges with implementing the plan, she said.
The content of the diversity plan should include an overview of the disease or condition, enrollment goals that are based on the epidemiology of the disease and a priori information or data on outcomes, as well as specifics on the actions the sponsor will take to enroll and retain participants, such as community engagement or decreasing the operational burden of the trial.
“Discuss any changes to your diversity plan if enrollment goals fall below the expectations that you set at the beginning,” Fienkeng said.
Industry questions
Monique Carter, a senior director at Pfizer in Global Regulatory Affairs, Global Product Development in Internal Medicine, presented information on Pfizer’s diversity plan template, which was in use at the company before the FDA’s draft guidance was released. The diversity plan template includes an overview of the disease or condition, the clinical development plan, enrollment goals for racial/ethnic subgroups, and specific plans for how to enroll a diverse population.
Carter also detailed some areas where industry still has questions about the implementation of diversity plans. For instance, should enrollment goals include individual study or overall program level goals? Similarly, what are the data sources that the FDA would accept for goal establishment? Carter also said industry is seeking information on the consequences of falling short on enrollment goals set out in the diversity plan. “We put the goals out there and we tried our best to meet them, but what happens if we fail?” she asked.
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