FDA Advocates for Diversity in Clinical Trials

Historically, the design of clinical trials to evaluate specific drugs has been in a controlled environment. The study population enrolled in a trial is very carefully chosen, with specific inclusion and exclusion criteria for trial subjects. However, in the real world, the drug will be prescribed to a diverse population of patients, each with a distinct genetic profile that can result in significant differences in drug exposure, absorption, metabolism, outcomes, and adverse effects.

Several studies have noted the lack of diversity in clinical trials. Physicians usually base their treatment decisions by extrapolating the results of trial data from a relatively homogenous population of white males. With a near-equal split in the percentage of males and females in the US, and with racial and ethnic minorities making up nearly 40% of the population, heterogeneity in clinical trials would be extremely valuable. Another population that is usually underrepresented in clinical trials are the elderly. Medications developed for a lot of chronic conditions are most often than not used by the elderly, and the 65-plus is the fastest growing segment of the global population.

A study conducted by researchers at the University of California, Davis, found that while cancer clinical trial participation is low for all adult groups (3-5%), the percentage of minorities who participate in clinical trials is not representative of the US population affected by cancer. For example, blacks experience the highest incidence of cancer (593.7 cases per 100,000 people) but, along with Hispanics, have the lowest rates of cancer clinical trials participation at 1.3%.

Responding to these concerns, the FDA, under direction by the Congress, has been paying greater attention to study subject diversity in clinical trials. The directive from the Congress was included in Section 907 of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, and in response, the FDA created the Drug Trial Snapshot, which provides details on the demographic profiles of participants in clinical trials of approved drugs. The Snapshot would thereby be the go-to page for patients and providers alike to understand the responses of specific groups of patients to these drugs.

This effort can have a tremendous impact on the quality of care as well as downstream costs of care for the health system as a whole. Inclusion of more women, minorities, and the older population in a drug trial can help drug developers as well as providers better understand clinical presentation and response to the drug being developed in a wider demographic. A skewed pool of participants could miss important drug-gene interactions. A clear understanding of a drug’s effects across the population can potentially reduce experimentation by physicians once the drug is approved for use in the clinic.

To that end, the FDA is planning numerous efforts in 2016 to push for greater inclusion in trial populations:

• FDA’s Office of Minority Health has developed tools to support clinical trial participation, including collaboration with the National Library of Medicine to raise consumer and patient awareness on clinical trials
• FDA’s Office of Women’s Health launched its Diverse Women in Clinical Trials initiative to raise awareness and share best practices about clinical research design, recruitment, and subpopulation analyses
• Ongoing efforts to engage patient advocacy groups in clinical trial design, feedback, and evaluation
• FDA staff will work with the research community to refine their approach to the conduct and analysis of trials to provide the best estimates of treatment effects for diverse populations.

What is needed is a concerted effort from various agencies and policy changes to push greater inclusion and avoid disparities in clinical trials.