FDA: Don’t rush publishing your diversity guidance plan. Take your time and do it right

When Congress passed the Food and Drug Omnibus Reform Act (FDORA) in December 2022, it was hailed as a landmark step toward codifying diversity and inclusion in clinical trials. Under FDORA, sponsors of Phase 3 and other pivotal trials are required to submit a diversity action plan to the Food and Drug Administration along with their study protocol. The FDA’s goal is to ensure that enrollment goals include clinically relevant study populations. The FDA is also required to publish guidance on diversity action plans. But nearly half a year after a Dec. 29, 2023, deadline for the agency to circulate a draft, the biopharma industry is still waiting for it. If this means the FDA is trying to get this right, we think the delay is OK.

Editor’s note: The FDA published its guidance not long after this essay was published.

Considering the long-standing underrepresentation of many populations in clinical research, the pressure to incorporate them into clinical research is evident. A study published in The Lancet in April 2022 highlighted the representation of five racial and ethnic groups — white, Hispanic/Latino, Black, Asian, and American Indian — in U.S. clinical trials over a 20-year period. Only 47% of the trials that tracked this data reported they included patients from all five groups. Twenty-one percent reported having zero Black enrollees, and 10% had all white participants.

Likewise, a study published in JAMA Oncology in March found that 94% of all U.S. cancer trials were located in relatively affluent areas with a higher proportion of white residents than the national average. Because residents near these sites are less diverse, biases were implicitly introduced by conducting trials at these locations.

An individual’s race or ethnicity can influence their response to medicines or how their disease progresses. The anticoagulant warfarin, for example, is metabolized differently in people of Asian descent. Tacrine is less effective in individuals carrying a particular gene variant that is more common in African Americans or African populations than Asian populations.

Although much of the discussion around diversity centers on race and ethnicity, it is also essential to include individuals across ages, sexual orientations, gender identities, geographic locations, socioeconomic statuses, and disabilities.

Comprehensive diversity guidance will improve clinical trial results, yield a better understanding of treatment effects, and increase trust and participation among underrepresented groups.

It just takes time.

The complexities of developing diversity action plan guidance

Ensuring diversity and inclusion in clinical trials is a complex undertaking, and the FDA must get it right from the outset. It must balance the need for rapid implementation that advances patient outcomes with the need for effective guidance.

One of the FDA’s primary challenges is how to categorize and define various ethnic groups. It may be a national organization, but it has an international reach. Clinical trials are increasingly global — it’s tough to run a Phase 3 trial based in just one country — and it’s also tough to define race and ethnicity across countries and regions. Could someone living in Brazil who has African heritage be considered Black or African American? How do you classify someone who is multiracial? There also can be a lack of standardized patient demographic data — or, in places like Germany, no data on race or ethnicity at all. Collecting data across multiple health care systems, regions, and languages is an important step toward improving health outcomes.

Moreover, the FDA’s guidance must be translatable across therapeutic areas, even those where the science may not be as advanced or the number of clinical trials may be limited. Because of this, it’s unlikely the FDA will release a one-size-fits-all template as it needs the flexibility to accommodate the unique challenges and opportunities associated with various diseases or conditions.

Preparing guidance is also complicated by the assumption that all members of traditionally underrepresented communities want to participate in research, which may not necessarily be the case. Building trust and encouraging participation requires overcoming years of skepticism due to historical neglect and a lack of understanding of clinical trials. Even so, some members of these groups are interested in participating in research, underscoring the need for a diversity action plan that addresses these complexities.

While the FDA’s diligence in developing guidance on trial diversity can strengthen trust and collaboration among regulators, the pharmaceutical industry, and potential participants, there’s also a financial rationale for it. Companies can face the harsh reality that a non-representative trial may waste years’ worth of time and resources. Given that the estimated average investment needed to bring a new medicine to market is $1.6 billion, diversity is a business issue, not just a scientific one.

A call for collaboration and commitment

While stakeholders in the biopharmaceutical industry, life sciences professionals, and regulators worldwide await the FDA’s guidance, they must do their parts to promote clinical trial diversity. Collaboration and commitment from all parties involved are necessary to ensure progress.

The biopharmaceutical industry must actively engage with patient communities, particularly those from underrepresented groups, to build trust and encourage participation in clinical trials. Life sciences professionals can advocate for inclusive research practices. Researchers and sponsors should incorporate diverse populations in their trial designs and recruitment strategies even without FDA guidance.

Regulators in other countries should consider the FDA’s efforts and implement similar measures to promote inclusion within their jurisdictions. International cooperation and the harmonization of diversity guidance can maximize clinical research outcomes.

The need for continued collaboration, with or without the government’s guidance, is more pressing than ever. By working together and remaining committed to increasing diversity in clinical trials, life sciences stakeholders can ensure new medical products benefit all patients, regardless of their background or demographic characteristics.

Tamei Elliott is a regulatory affairs professional and the associate director of scientific programs for DIA, a global life science membership association that drives collaboration in drug, device, and diagnostics development. Maria Vassileva is DIA’s global head of science and regulatory strategy.