The guidance, intended to promote more consistent demographic subgroup data collection practices, reflects a standardized approach to collecting race and ethnicity data in accordance with Affordable Care Act (ACA) and Food and Drug Administration Safety and Innovation Act (FDASIA) requirements.

The FDA published updated guidance, replacing a 2005 version, to provide input on how clinical trial sponsors can meet the requirements regarding presentation of demographic data on investigational new drug (IND) applications and new drug applications (NDAs) and the collection of race and ethnicity data in biologics license applications (BLAs) and medical device applications.

After an FDASIA-mandated report revealed diversity gaps in race and ethnicity data in clinical trial applications for FDA‐regulated medical products, the agency held a public hearing in 2014 soliciting industry feedback on the challenges associated with the collection, analysis and availability of demographic subgroup data. The updated guidance reflects the concerns and recommendations generated in public workshops by a variety of experts and stakeholders, which the FDA incorporated into an action plan intended to improve the completeness and quality of such data. The guidance supports the action plan by detailing the FDA’s standardized approach for the collection and availability of demographic subgroup data and the analysis of race and ethnicity data.

To maintain consistency with the current Office of Management and Budget directive and the National Institutes of Health guidance for collecting racial and ethnic data, the FDA recommends sponsors use a two-question approach to request race and ethnicity information from clinical trial participants:

  • Question 1 (answer first): Do you consider yourself Hispanic/Latino or not Hispanic/Latino?
  • Question 2 (answer second): Which of the following five racial designations (American Indian or Alaska Native; Asian; Black or African American; Native Hawaiian or Other Pacific Islander; White) best describes you? More than one choice is acceptable.

The guidance also provides the following FDA recommendations:

  • Self-reporting: The FDA recommends that trial participants self-report race and ethnicity information and be permitted to designate a multiracial identity. Race and ethnicity should not be assigned by the study team conducting the trial.
  • Ethnicity: The agency outlines the minimum recommended choices that should be offered to trial participants.
  • Race: The FDA recommends a list of minimum choices that should be offered to trial participants.
  • Use of more detailed racial and ethnic categories: In situations where appropriate, the FDA recommends using more detailed categories by geographic region to provide sponsors the flexibility to adequately characterize race and ethnicity. For INDs, NDAs and BLAs, the agency recommends the submission of tabulated demographic data based on the Demographic Rule for all clinical trials using the characterizations of race and ethnicity described in this guidance.

Per the updated guidance document, the FDA expects sponsors to enroll participants who reflect the demographics for clinically relevant populations with regard to age, gender, race and ethnicity. A plan to address inclusion of clinically relevant subpopulations should be submitted to the agency for discussion at the earliest phase of development and, for drugs and biologics, no later than the end of the Phase 2 meeting.