FDA recommends sponsors plan to include race, ethnicity in clinical trial design

On 13 April 2022, the FDA published a draft guidance titled, “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials”.  In it the Agency recommends sponsors submit a “Race and Ethnicity Diversity Plan” whenever submitting an application for an investigational new drug (IND), biologics license application (BLA) or an investigational device exemption (IDE).  Regulators note they will consider the plan “an important part” of the product’s development program.

“The U.S. population has become increasingly diverse, and ensuring meaningful representation of racial and ethnic minorities in clinical trials for regulated medical products is fundamental to public health,” said FDA Commissioner Robert Califf.  “Going forward, achieving greater diversity will be a key focus throughout the FDA to facilitate the development of better treatments and better ways to fight diseases that often disproportionately impact diverse communities.”

The Commissioner also noted that the guidance is meant to support the Biden administration’s Cancer Moonshot project, part of which aims to address inequities in cancer care, as well as bring new cancer diagnostics and treatments to market faster.  The FDA said that sponsors can discuss their strategy for enrolling a diverse cohort for their trials at any given time during the product development process, though the guidance lays out a number of timeframes that are more ideal for reviewers.

“For drugs, sponsors should submit the plan to the relevant IND application as soon as practicable during drug development but no later than when a sponsor is seeking feedback regarding the applicable pivotal trial(s) for the drug (often at the [end of phase 2] meeting),” said the guidance.  “The plan can be submitted to the IND as part of a milestone meeting package, or on its own.”

For devices, sponsors should submit their plan as part of the IDE application and use the Q-submission process to get feedback on their enrollment strategy, according to the FDA.

FDA emphasized that the guidance is not meant to address all issues in clinical development such as study design, trial endpoints or the necessary data package to support a marketing application.  For answers to those issues, the agency refers sponsors to other previously published guidances.

“FDA guidance documents define a diverse population, when applicable, to be inclusive of all populations as defined by demographic factors such as race, ethnicity, sex, gender identity, age, pregnancy status, lactation status, and by the presence of certain clinical characteristics such as multiple comorbidities,” the FDA noted.  “Some individuals from these groups have often been underrepresented in medical product development and FDA considers their representation in clinical trials and studies to be a priority.”

The guidance recommends sponsors define enrollment goals for underrepresented populations as early as practicable and those goals should be based in part on pre-specified protocol objectives.  Deciding on enrollment goals may be difficult because it may not be clear what factors may affect different populations differently.  However, the FDA says collecting data such as pharmacokinetic (PK), pharmacodynamic (PD), and pharmacogenomic data from a diverse population for drug development, and phenotypic, anatomical or biological data for devices, may help sponsors hone down the necessary data for their cohort.

The FDA also acknowledges that some groups may be wary of participating in clinical research, alluding to mistrust in the African-American community of medical research due to unethical experiments on Black men with syphilis conducted by the Tuskegee Institute.  They also cite other factors such as socioeconomic, language and cultural barriers.  “Barriers to participation among racial and ethnic groups may include mistrust of the clinical research system due to historical abuses, aspects of the trial design such as inadequate recruitment and retention efforts, frequency of study visits, time and resource constraints for participants, transportation and participation conflicting with caregiver or family responsibilities,” the Agency said.  “In addition, language and cultural differences, health literacy, religion, limited access within the health care system and a lack of awareness and knowledge about what a clinical trial is and what it means to participate may impact clinical trial participation among racial and ethnic minority populations.”

REFERENCE:  RAPS (Regulatory Affairs Professional Society) Regulatory Focus News Article (2022 4); 13 APR 2022; Ferdous Al-Faruque

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