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Regulatory Focus — Focus FDA misses congressional deadline to update clinical trial diversity guidance

Regulatory Focus — Focus FDA misses congressional deadline to update clinical trial diversity guidance

The US Food and Drug Administration (FDA) is several weeks late in issuing guidance on how and when drug and device companies must submit diversity action plans for clinical trial research.

The Food and Drug Omnibus Reform Act (FDORA), passed by Congress in late December 2022, requires drug and device manufacturers to submit diversity action plans to the FDA outlining how they plan to enroll a diverse group of trial participants for phase 3 and other pivotal trials. The enrollment goals must be disaggregated by age group, sex, and racial and ethnic demographic characteristics of clinically relevant study populations and may include geographic location and socioeconomic status. The law requires FDA to issue or update guidance on the content of the diversity plans within one year.

Speaking at a meeting on clinical trial diversity convened by Friends of Cancer Research, Rep. Anna Eshoo (D-CA), who sponsored the clinical trial diversity provisions included in FDORA, noted that the deadline to issue guidance had past several weeks ago.

“You can be sure I’ll stay on this,” she said. “I will hold the FDA, certainly, accountable to ensure it holds up its end of the law because patients and their families should not have to wait any longer to be part of life-changing clinical trials.”

Lola Fashoyin-Aje, of FDA’s Center for Biologics Evaluation Research (CBER), said the agency is “working very hard” to wrap up work on the draft guidance on diversity action plans. She noted that public comments on earlier iterations of the guidance have been helpful in considering “areas where we may need to provide more information about our thinking.” Additionally, a workshop on trial diversity convened by FDA in November 2023 has also been helpful as officials work on the guidance document, she said.

Fashoyin-Aje said she could not provide details on the guidance document since it is “still pending” but noted that the legislation is in alignment with the agency’s initial draft guidance on diversity plans. “I’m not sure that we would expect a different approach there,” she said.

Speakers at the Friends of Cancer Research meeting said there were some areas where they would like to have greater clarity from FDA on diversity plans. Henry Bennett, of Novartis, said sponsors running global trials need more information about how patients from other countries could help to meet certain race/ethnicity enrollment goals. For example, he wondered whether a patient from South Africa could be used to supplement enrollment goals for Black patients.

“These are some of the things that we also need to find more clarity [on] as we build our plans. It is important to us that we can balance the needs for global development of our drugs,” Bennett said.

Data challenges

In a discussion document issued ahead of the meeting, Friends of Cancer Research outlined several data-related challenges to setting enrollment goals for underrepresented groups as part of diversity action plans. For instance, there is not a standardized source for data on patient demographics and the various disparate sources can be incomplete and difficult to combine. Additionally, there is not sufficient biomarker data by demographic group, social determinants of health (SDoH) variables are not routinely collected, there are inconsistent definitions of race and ethnicity, and there is not robust data on populations outside the US.

Friends of Cancer Research proposed two strategies for addressing the data challenges. The first calls for the creation of a central repository for biomarker data in the US and Canada. The repository would include nationally representative data for multiple cancer types and include race and ethnicity data, modeled on what the BROAD Institute currently has for prostate cancer. The second proposal is to bring groups together to consolidate and harmonize curated data sources. The group noted that standards would be needed to accomplish this goal.

Cleo Ryals, of Flatiron Health, said it will take collaboration from across industry to develop the methodology to leverage different data sources. “I know we all have our own sets of interests and priorities, and even competition is at play here, but it’s really going to be important for all of us to come together…to develop a framework with methodological guidance on how to leverage data from multiple data sources,” she said.

Xoli Belgrave, of Parexel, agreed, saying that companies will need to “show and tell” about their experiences to create best practices that can be used by trial sponsors.

Operational approaches

The Friends of Cancer Research discussion document also outlines approaches that trial sponsors can use to achieve the enrollment goals set in diversity action plans, including building trust and partnerships in diverse communities, lowering barriers to enrollment such as restrictive eligibility criteria, engaging in intentional site selection, and implementing real-time tracking of enrollment progress.

Kristi Mitchell, of Atlas Clarity LLC & Health Equity Outcomes (HEO), said the importance of trusted messengers cannot be overstated. She pointed to the success of working with faith-based leaders and community health workers in combating vaccine hesitancy during the COVID-19 pandemic as a model for engaging communities on clinical trial enrollment.

Edward Kim, of City of Hope Orange County, said oncology studies are overcomplicated and the eligibility criteria for most studies could be simplified to allow for greater and more diverse participation. “The more eligibility criteria you tack on to a study, the less number of people can be included in that study. That inherently is discrimination,” he said.

Friends of Cancer Research discussion document 

Friends of Cancer Research meeting