How Much Diversity is Required for FDA Approval?   

Clinical trial populations are not very diverse. Minority populations form a very small fraction of the trial populations, much below their proportion in the US population leading to concerns about the effectiveness of approved products in populations under-represented in clinical trials. Since 2014, FDA’s Action Plan to Enhance the Collection and Availability of Subgroup Data requires 1) improved demographic subgroup data within medical product applications, 2) identification of barriers to enrolling members of demographic subgroups into clinical trials and strategies to address those barriers, and 3) making demographic subgroup data from market approval applications more readily available to the public. To meet the first criteria FDA now requires all sponsors to create clinical trial enrollment plans to assure a study population that includes diverse demographic subgroups (age, sex, ethnicity/race), consider any disparities in outcomes observed across demographic subgroups, and assure that the population be reflective of the likely U.S. patient population for whom the agent will be prescribed following approval for its use. The net result of this requirement is that sponsors have to show a reasonable effort to recruit minority populations, although lack of minority populations in the clinical trials cannot be a reason for denial of market approval. The second criteria of the Action Plan lead to several initiatives at the FDA to educate and encourage minorities to participate in clinical trials. This effort has had a limited effect so far with positive trends. The last criteria of the Action Plan lead to the Drug Trial Snapshot program whereby each approved drug gets a summary page on FDA’s web-site describing the demographic composition of the clinical trial populations that formed the basis for approval. A review of the Drug Trial Snapshots, show that there is a long road for reasonable minority participation in clinical trials. Overall, efforts to encourage minority populations in clinical trials pose a tough challenge to any sponsor. One way to get reasonable data in populations other than Caucasians is to include sites in other countries. That adds its own logistical challenges and complexities but may be worth the effort if FDA makes it mandatory to include diversity representative of the US population in market approval applications. At present, diversity is desired but not required. That may change.