Increasing Diversity in Clinical Trials

The FDA issued guidance in November aimed at enhancing diversity and encouraging inclusivity in medical research, specifically in the development of medical products. FDA Commissioner Stephan M. Hahn, M.D., wrote that “in order to promote public health, it is important that people who are in clinical trials represent the populations likely to use the potential medical product.”

Last week, the industry trade group PhRMA issued voluntary guidelines to members aimed at enhancing racial and ethnic diversity among clinical trial participants. Those guidelines go into effect in April 2021.

The FDA guidance discusses:

  1. broadening eligibility criteria and avoiding unnecessary exclusions for clinical trials;
  2. developing eligibility criteria and improving trial recruitment so that the participants enrolled in trials will better reflect the population most likely to use the drug, if the drug is approved, while maintaining safety and effectiveness standards; and
  3. applying the recommendations for broadening eligibility criteria to clinical trials of drugs intended to treat rare diseases or conditions.

The final FDA guidance addresses demographic diversity as well as non-demographic diversity, including people with co-morbidities, disabilities and wider weight ranges. Broadening inclusion eligibility for clinical trials impacts protocol design and methodological approaches.

The PhRMA guidelines specifically focus on outreach to communities of color to reduce health disparities. Activities include outreach and education, as well as the reduction of barriers to participation.

Exclusion and eligibility criteria necessarily balances risk of adverse events against the potential benefit of the research. Pregnant women, people with chronic or severe kidney disease, or complex medical conditions are often excluded from clinical trials due to risk. However, the FDA cautions against excluding certain populations out of habit or template, rather than based on strong clinical or scientific justification. Both sets of guidelines encourage broadening eligibility criteria to increase diversity when scientifically and clinically appropriate.

The FDA guidance addresses the logistics of participation and offers strategies for minimizing the burden on enrollees. For example, certain populations may find it difficult to visit research sites frequently. Researchers and sponsors are encouraged to consider alternative methods of engaging with participants, including phone or virtual visits, email, social media and digital devices.

Investigators and sponsors are encouraged to adopt enrollment and retention practices that enhance inclusiveness. Engaging patient advocacy groups in the design of protocols; increasing outreach and education through community organizations; expanding research sites to ethnic or underserved neighborhoods; hosting frequent recruitment events; and developing recruitment materials in multiple languages are just some of the suggestions.

While both the FDA and PhRMA address sponsored clinical trials, it can also influence investigator-initiated trials.

Broadening eligibility criteria and adopting more-inclusive enrollment practices should improve the quality of studies by ensuring that the study population is more representative of the population that will use the medical product if it is approved.