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Dual efforts focused on more inclusive research and more diverse enrollment in pre- and post-approval studies.
FDA recently issued a new guidance on how to make clinical research more inclusive, building on earlier, broader advisories on the topic. At the same time, Congress is looking to advance legislation promoting clinical trial diversity in pre- and post-market studies, while the latest revision to drug user fee programs includes provisions for advancing clinical trial diversity in sponsors’ R&D programs. These initiatives fit the Biden administration’s broader goal of addressing racial and ethnic health care disparities through a range of public research and coverage programs.
This latest FDA draft guidance follows its advisory on “Enhancing the Diversity of Clinical Trial Populations” published in November 2020. That final guidance supported efforts to expand eligibility criteria and improve enrollment practices and trial designs so that clinical studies would better reflect the population most likely to use the drug if approved.1
The specific aim here is to clarify how sponsors should submit a “Race and Ethnicity Diversity Plan” with applications proposing clinical research or continuing with trials to support the approval of drugs, biologics, and medical devices.2 The draft guidance encourages early submission of such plans to present strategies for enrolling and retaining diverse patient populations in trials. FDA advises sponsors to discuss such research proposals as early as possible during the investigational new drug (IND) stage, and certainly before launching pivotal Phase III studies.3
FDA defines a “diverse population” as including demographic factors such as race, ethnicity, sex, gender identity, age, pregnancy, or lactation status, and the presence of additional comorbidities. Sponsors should set enrollment goals for a full range of populations in order to obtain relevant pharmacological and genomic data that can help design larger studies appropriately. The guidance also outlines patient retention strategies, which may involve collaboration with community health workers and patient advocates and reimbursement for expenses to participants in need. FDA advises sponsors to consider the location and accessibility of research sites as part of diversity planning and to design programs for reaching out to patients often reluctant to participate in medical research.
Efforts to achieve greater diversity in research “will be a key focus throughout the FDA,” stated FDA Commissioner Robert Califf, noting the importance of effectively fighting diseases that often disproportionately impact minority communities in announcing the new proposal. These regulatory efforts support the Biden administration’s Cancer Moonshot initiative, which aims to address inequities in access to cancer screening, diagnostics, and treatment across race, gender, region, and resources. The new guidance was developed by staff of Project Equity at FDA’s Oncology Center of Excellence (OCE), which seeks data supporting the approval of oncology therapies that fully represent all patients in need of treatment.
Congress also is likely to consider legislation on this issue as part of a broader measure to reauthorize FDA user fees that is moving forward on Capitol Hill. A bipartisan bill supported by leading members of the House Energy and Commerce Committee aims to enhance FDA’s authority to require that drug and device applications include demographic information on the population to be treated and to enroll such groups in clinical research programs. The Diverse and Equitable Participation in Clinical Trials (DEPICT) Act further authorizes FDA to require sponsors that fail to meet diversity enrollment targets to obtain that data in post-marketing studies.
The 2.0 update to the 21st Century Cures Act similarly calls for FDA and other federal health agencies to advance diversity in clinical trials through added research and policy-making activities. And the latest version of the Prescription Drug User Fee Act (PDUFA VII) sets goals for sponsors to enhance clinical trial diversity, particularly in the development of new cancer therapies.
FDA aims to further encourage diverse clinical trials through research projects to explore best practices for achieving these goals. One proposal is to learn more about which populations are most affected by research that fails to address diversity issues. Others look to better define “health diversity” and how to determine when a specific population or group is most adversely affected by inequities in treatment and research. Sponsors would like more advice from FDA on how to respond when unable to enroll fully representative populations in studies, and what data is most important to collect. Goals may differ for research programs that involve very few patients, as seen in efforts to extrapolate race and ethnicity data from limited studies. An important issue is how FDA and sponsors should consider clinical data from research sites outside the US. FDA has requested that stakeholders submit comments by June 13 on these and other topics raised in the guidance document.