Last week, in the midst of the Biden Administration’s coordinated release of more than 90 “Equity Action Plans” by various federal agencies, the FDA issued a new draft guidance on enhancing clinical trial diversity. In a statement accompanying the guidance, FDA Commissioner Robert Califf reiterated the agency’s commitment to this goal: “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.”
This draft guidance, titled “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials,” is the FDA’s latest effort in recent years designed to encourage industry to focus on the goal of improving diversity in clinical trials. As the FDA notes, “Consistent implementation of actions to improve racial and ethnic diversity in clinical trials and studies can support early access to medical discoveries and innovations, improve the generalizability of results across all patient populations, improve our understanding of the disease and/or medical product under study, and inform the safe and effective use of the medical product for all patients who are expected to use the medical product if approved.”
The guidance lays out a framework detailing how and when sponsors should submit “Race and Ethnicity Diversity Plans” (REDPs) designed to set forth a strategy for enrolling “representative numbers of participants from underrepresented racial and ethnic populations in the United States.” Notably, the guidance focuses only on racial and ethnic study populations, although the FDA encourages sponsors to continue to work toward adequate participation by other underrepresented populations defined by demographics such as sex, gender identity, race and others. The Oncology Center of Excellence prepared the guidance, but the other medical product centers contributed to the effort, as it applies to all medical devices and drugs for which clinical studies are intended to support the marketing submission.
The REDP for drugs should be submitted “as soon as practicable” but no later than when the sponsor is seeking FDA feedback on the pivotal trial (end of phase 2 meeting); for medical devices, the REDP should be submitted as part of the investigational device exemption (IDE) application. The guidance sets forth five elements that sponsors should include in the REDP:
- Overview of the Disease/Condition: Assess the data that indicates the potential for differential safety or effectiveness that is associated with race or ethnicity.
- Scope of Medical Product Development Program: Describe the trials that will support the ultimate approval of the product, including how the study will address inclusion of underrepresented racial and ethnic populations and any differential findings that might be associated with such populations.
- Goals for Enrollment of Underrepresented Racial and Ethnic Participants: Define and justify planned enrollment of participants from underrepresented racial and ethnic populations.
- Specific Plan of Action to Enroll and Retain Diverse Participants: Describe the operational measures to enroll underrepresented racial and ethnic populations, including strategies such as site location and access, sustained community engagement, and addressing burdens of study participation.
- Status of Meeting Enrollment Goals (as applicable): As progress is made, update the REDP to discuss status of meeting enrollment goals and potential for post-market data collection if goals are not met.
The draft guidance was issued amid a flurry of activity on Capitol Hill with respect to clinical trial diversity in the context of the FDA user fee reauthorization process. At a March 17 legislative hearing in the Energy and Commerce Committee Subcommittee on Health, four bills that would address the goal of enhanced clinical trial diversity were discussed, including one introduced by Subcommittee Chair Anna Eshoo with similarities to the guidance just issued. But the “Diverse and Equitable Participation in Clinical Trials Act,” or the “DEPICT Act” (H.R. 6584), goes further than the guidance by requiring the submission of a “diversity action plan” with an Investigational New Drug Application (IND) or IDE containing similar information to the REDP. The bill would also give FDA new authority to require post-market studies for “demographic subgroup or subgroups” if the enrollment targets set forth in the diversity action plans are not met.
It remains to be seen whether the DEPICT Act or any other bills will move forward with the user fee legislative package. But the FDA guidance, along with the Biden Administration’s general emphasis on diversity and equity, is yet another reminder to pharmaceutical and medical device companies that the time is now to ensure that every effort is made to improve racial and ethnic diversity in clinical trials.