Earlier this year, the U.S. Food and Drug Administration updated its guidance document on collecting race and ethnicity data in clinical trials. The document updates previous guidance released in 2016 and represents the latest step at the federal level to improve clinical trial diversity and identify what the FDA calls “population-specific signals” in medical products.
The new FDA guidance for clinical trial diversity stems in part from an Office of Management and Budget (OMB) review of its directive on standardized terminology for factors, such as race and ethnicity. Though FDA guidance is not legally binding, it provides trial sponsors with clear recommendations for how to collect race and ethnicity data that will satisfy requirements for diversity plans and regulatory submissions, and what can be used for appropriate product labeling.
Updated FDA guidance: Document diversity in clinical trials in a standard format
In January 2023, OMB began reviewing its 1997 policy directive on how federal agencies collect and use race and ethnicity data, as the nation had seen significant demographic shifts in the subsequent quarter century. FDA had based its 2016 guidance on the “minimum standard categories” as described by OMB and indicated it was in the process of updating its own guidance before OMB announced its review.
As a baseline, the FDA recommends a two-question format for individuals to self-report race and ethnicity in clinical trials. The ethnicity question should come first and offer a minimum of two choices: Hispanic or Latino, or Not Hispanic or Latino. The race question should offer a minimum of five choices – American Indian or Alaska Native, Asian, Black or African American, Native Hawaiian or Other Pacific Islander, and White – and offer the option to select more than one designation.
FDA indicates that more-detailed information than the baseline may be necessary to further define communities of color by geographic region. In documenting ethnicity, for example, trial sponsors may want to offer additional selections as part of the OMB standard for Hispanic or Latino, such as Mexican, Mexican American, or Chicano; Puerto Rican; Cuban, and Other Hispanic or Latino.
In documenting race, trial sponsors may offer varying selections for Asian based on the OMB standard, for example: Asian Indian, Chinese, Filipino, Japanese, Korean, Vietnamese, and Other Asian. Likewise, the OMB standard for Native Hawaiian or Other Pacific Islander may be broken down into Native Hawaiian, Guamanian or Chamorro, Samoan, or Other Pacific Islander.
FDA guidance on uses for data on diversity in clinical trials
FDA, in alignment with OMB’s directive, calls out ethnicity and race as a social construct, not as scientifically defined biological differences. This is an important and overdue distinction. The long-held belief that race is a biological construct has contributed to systemic racism in areas, such as medical research, clinical diagnosis, pain management, and disease treatment.
However, the FDA also indicates intrinsic and extrinsic factors can impact how therapeutics and other treatments work in different population subsets. Intrinsic factors can include differences in genetics, skin pigmentation or pharmacokinetics, which is the body’s reaction to an ingested substance. Extrinsic factors can include environment, diet, culture, and other social determinants of health.
Along these lines, when it comes to reporting diversity in clinical trials, FDA guidance offers three recommendations.
- Individuals should always be given the option to self-report ethnicity and race. Research teams should not report this information on behalf of patients, nor should they rely on electronic health records for this information.
- The term non-white is not acceptable for use in presenting federal government data. In fact, the FDA recommends erring on the side of granularity by reporting ethnicity, single-choice race, and multiple-choice race responses separately. While these differences may be difficult to parse out, trial sponsors will have detailed insight into who was included in a study.
- Trial sponsors should report demographic information in drug and device submissions, in product labels, and in prescribing information – namely, the Clinical Studies and Adverse Reactions sections of the label. Though trial sponsors likely do this already, the FDA now suggests consistency with OMB standards when capturing race and ethnicity data.
The impacts of FDORA
The FDA guidance on clinical trial diversity is the latest step at the federal level in encouraging sponsors to make clinical trials more representative.
Historical efforts began with the NIH Revitalization Act of 1993 and continued with the Diverse and Equitable Participation in Clinical Trials, or DEPICT Act, which encouraged diversity in clinical trials by requiring sponsors to set enrollment targets by demographic subgroups and report on trial participant demographics.
In December of 2022, Congress passed the Food and Drug Omnibus Reform Act (FDORA Act), which incorporated underlying principles of the DEPICT act and includes provisions to encourage greater diversity among clinical trial participants. Notably, it requires sponsors of certain late-stage drug trials and most device trials to submit diversity action plans.
As part of the FDORA Act, trial applicants must submit diversity action plans and enrollment targets by demographic subgroup. It also grants the FDA the authority to mandate post-market studies if diversity enrollment targets are not met. Recently, the FDA released draft guidance on the collection of race and ethnicity data in clinical trials and studies, signaling a concerted effort to standardize data collection practices.
The introduction of FDORA and updated FDA guidance underscores a significant shift in the consequences of limited diversity in clinical trials. In the future, failure to achieve diversity goals may result in more severe financial penalties than previously observed.
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