FDA Seeks to Enhance Clinical Trial Diversity with New Guidance

Because there are still significant obstacles to increasing enrollment diversity in clinical trials, the U.S. Food and Drug Administration has issued draft guidance to overcome this. According to FDA, “Failure to include complex participants in a development program may lead to a failure to discover important safety information about use of the investigation drug in patients who will take the drug after approval. Broadening eligibility criteria, when appropriate, maximizes the generalizability of trial results and the ability to understand the therapy’s benefit-risk profile across the patient population likely to use the drug in clinical practice, without jeopardizing patient safety.”

To broaden eligibility criteria:

• Sponsors can examine each exclusion criterion to determine whether it is needed to help protect people’s safety or to achieve the study objectives. If not, sponsors should consider eliminating the criteria or tailoring it as narrowly as possible. “For example, if there are unreasonable risks to participants with advanced heart failure, but enrollment of those with milder disease would be appropriate, the exclusion criteria should specifically define the population of heart failure participants that should be excluded,” FDA said.

• Sponsors can consider using an adaptive clinical trial method that allows for prespecified trial design changes during the trial, including altering the trial population. “An adaptive design can start with a narrow population if there are concerns about safety and can expand to a broader population based on interim data from the trial, as well as external data,” FDA said.

• Sponsors can account for logistical and other factors that could limit participation. For example, FDA suggested that when designing the study, sponsors could consider recruitment challenges that may occur because of the study’s schedule. “Reduce the frequency of study visits to those needed to appropriately monitor safety and efficacy and consider whether flexibility in visit windows is possible and whether electronic communication (e.g., telephone/mobile telephone, secured electronic mail, social media platforms) or mobile technology tools can be used to replace site visits and provide investigators with real-time data,” FDA said.

• Sponsors could offer financial reimbursements for travel and other costs associated with clinical trial participation. In addition, they should work directly with communities to address participants’ needs, and to involve patients, patient advocates and caregivers in designing clinical trial protocols. “Patients may provide valuable insight into challenges and burdens and may be more willing to accept risk for a potential benefit as long as the risks are clearly communicated in the informed consent and the research team explains the risks,” FDA said.

• Lastly, sponsors should consider recruiting clinical trial participants in diverse locations. “Make recruitment events accessible by holding them often, as well as offering them during evening and weekend hours,” FDA said. “Consider holding the events in nonclinical but trusted locations (such as houses of worship) and social commercial venues (such as barbershops and beauty salons) as a means of connecting with diverse populations.”