Racial and Ethnic Diversity in Clinical Trials – FDA Guidance and How it Impacts Patient Recruitment in Clinical Trials

November 29, 2023
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On April 13, 2022, The Food and Drug Administration (FDA) issued a draft guidance document – Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials – for the industry to submit with their Investigational New Drug (IND) applications.  Along with the guidance’s news release, the FDA stated that it “… recommends that sponsors of medical products develop and submit a Race and Ethnicity Diversity Plan to the agency early in clinical development, based on a framework outlined in the guidance.”   the FDA stated that it “recommends that sponsors of medical products develop and submit a Race and Ethnicity Diversity Plan to the agency early in clinical development, based on a framework outlined in the guidance.”  

The FDA states how the industry should follow their guidance through the administration’s: “Guidance documents are not binding for FDA or the public. Guidance should be viewed as recommendations unless specific regulatory or statutory requirements are cited. An alternative approach may be used if the approach satisfies the requirements of the applicable statute and regulations.” While guidance documents issued by the FDA are not technically binding, strict adherence to them is standard industry practice.  

In 2016, the FDA had issued another guidance document on the Collection of Race and Ethnicity Data in Clinical Trials, providing specific instruction on the type of diversity data to collect in clinical trials. As evidenced by the growing industry focus on diversity in clinical trials, it is likely that diverse patient recruitment and enrollment in clinical trials will be “must have” capabilities by the time a final guidance document is issued by the FDA.  

Growing Responsibilities for Industry Sponsors

When the FDA issued its first clinical trial diversity guidance document in 2016, clinical trial sponsors weren’t responsible in carrying out the outlined efforts.  The reason was simple: race and ethnicity data was collected at research sites as patients enrolled into clinical trials, and there were no specific diverse enrollment goals.  

However, once the finalized guidance is issued for the enrollment of participants from underrepresented racial and ethnic populations, industry sponsors will need to undertake both the enrollment of these participants and the collection of race and ethnicity data.

Race and Ethnicity Data Collection

The FDA’s recommendation for collecting race and ethnicity data is based on the Office of Management and Budget Directive 15 and was developed in accordance with section 4302 of the Affordable Care Act. The data collection recommendation applies to studies being conducted both inside and outside of the United States.  

The FDA recommends ethnicity and race questions be asked in the following order using the structure provided below:

Question 1:

Do you consider yourself Hispanic/Latino or Not Hispanic/Latino? [choose one]

Question 2:

Which of the following five racial designations best describe you? More than one choice is acceptable.

American Indian or Alaska Native: A person having origins in any of the original peoples of North and South America (including Central America), and who maintains tribal affiliation or community attachment.

Asian: A person having origins in any of the original peoples of the Far East, Southeast Asia, or the Indian subcontinent, including, for example, Cambodia, China, India, Japan, Korea, Malaysia, Pakistan, the Philippine Islands, Thailand, and Vietnam.  

Black or African American: A person having origins in any of the black racial groups of Africa. Terms such as “Haitian” can be used in addition to “Black or African American.”  

Native Hawaiian or Other Pacific Islander: A person having origins in any of the original peoples of Hawaii, Guam, Samoa, or other Pacific Islands.  

White: A person having origins in any of the original peoples of Europe, the Middle East, or North Africa.

The FDA provides guidance on collecting more detailed racial and ethnicity information as needed, but the above may be sufficient to confirm the race or ethnicity of clinical trial applicants.  

Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials

In its guidance document, the FDA recommends enrolling representative numbers of participants into clinical trials from underrepresented racial and ethnic populations in the United States. The FDA proposes that these parameters will ensure the data that’s generated in clinical research will reflect the racial and ethnic diversity of the patient population and will identify relevant safety and efficacy outcomes should the potential therapeutic receive approval.  

The FDA is requiring industry sponsors to submit a specific plan constituting of the following elements:

  1. Disease overview: Describe available data on the pathophysiology of the disease or condition in underrepresented racial and ethnic populations.  Explain how the disease impacts underrepresented minorities differently.  
  1. Program design: Outline how the study design impacts underrepresented minorities and how data might be different.
  1. Enrollment goals: Specify populations and enrollment goals.  
  1. Plan to enroll and retain diverse participants: Provide specific trial enrollment and retention strategies including but not limited to:
  1. Site location and access (e.g., language assistance for individuals with limited English proficiency, reasonable modifications for persons with disabilities, and other issues such as transportation)
  1. Sustained community engagement (e.g., community advisory boards and navigators, community health workers, patient advocacy groups, local healthcare providers, etc.)
  1. Reducing burden due to trial/study design/conduct (e.g., number/frequency of study-related procedures, use of local laboratory/imaging, telehealth)
  1. Metrics: Describe metrics to ensure goals are met and specify actions during the trial to meet goals if goals aren’t met.  
  1. Status: Update plan based on status and provide a plan to collect data post-marketing if sponsors are unable to achieve their diverse enrollment goals during the trial conduct.  

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