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    Strategies from the FDA for Designing More Inclusive Clinical Trials

    By Hannah Drake • February 24, 2021
    Inclusivity for clinical trials

    The call to improve diversity in clinical trials has been growing louder in recent months, and rightfully so. The accuracy and success of any trial depends on how well its participants reflect the population of patients who will ultimately use the drug or treatment under investigation. The Food and Drug Administration itself has weighed in on the subject.


    In the FDA’s November 2020 guidance document, “Enhancing the Diversity of Clinical Trial Populations—Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry,” the FDA stresses that inclusive clinical trials begin with intentional trial design. Most often, the agency says, this can be accomplished by broadening eligibility criteria. Consider implementing some or all of these strategies that the FDA recommends.

    • Start with a goal of ensuring that subjects reflect a representative sample of the population who will benefit from the drug under development. In the past, patients with the very illness or condition a drug would treat have been excluded from clinical studies based on concerns about their risk for adverse events. The FDA advises researchers to quantify what degree of medical dysfunction would create unacceptable risk and accept in their studies patients who have milder levels of illness.

    • Likewise, enrollees should mirror the racial, ethnic, gender and age characteristics of the population who would benefit from the drug under investigation to the extent possible. This could mean including children and adolescents when appropriate, as well as women, who have often been left out of important research. 

    • When conducting pharmacokinetic sampling, consider including women who become pregnant during the course of the study, if the risks to the women and fetuses are reasonable, particularly when balanced against the potential benefits to them.

    • In early clinical development, work to understand how different populations might metabolize or clear the drug differently. This will help avoid unnecessary exclusions later, and will permit dose adjustments based on efficacy and safety in different groups of people.

    • Use an adaptive trial design—one that lets you modify the trial as results emerge. That way, any concerns about safety can be tested with a narrow population early on, and you can broaden the population once safety concerns are allayed.

    • When using prognostic and predictive enrichment strategies during trial enrollment—i.e., enrolling only people who have a certain severity, subset or clinical marker of a disease, or who are likely to respond to a treatment—consider including people who don’t meet all the enrichment criteria, especially if entire demographic groups would otherwise be left out. The FDA suggests that including people who have the disease but not the marker being studied could yield important information about efficacy in people with differing levels of disease severity.

    • The FDA also notes that trial participation can be burdensome for patients who have challenges with transportation, childcare, finances, and other practical concerns. To ease these hardships, the agency suggests reducing the number of site visits required, and embracing video and telephone visits, as well as digital health tools, when appropriate. Sponsors could also consider reimbursing participants for travel and lodging expenses. The FDA does not consider such reimbursements “undue influence,” so they are perfectly allowable. In some circumstances, you might consider paying subjects for participation, though your institutional review board should clarify the amounts and conditions that make this permissible.

    • Conduct public outreach to educate the broader population about clinical trials, as well as to seek input from disease-affected groups about ways to make participation less burdensome and more meaningful to them. Provide cultural competency education for your team so they understand how best to engage with participants from different backgrounds. Consider providing this outreach not just at health sites, but at locations throughout the community, and even on social media, to make recruitment more accessible to more people. 

    • Consider using real-world data from electronic health records to identify eligible participants. Of course, patient privacy and consent must be ensured, but this approach can help researchers find previously untapped sources of diverse patients. par8o Research Network’s partnerships with America’s community health centers are a great example of how this can work. 


    While this guidance from the FDA is nonbinding, the smart money is on implementing these suggestions as though they might be mandated at any time, especially as a new federal administration takes shape. But even more important than being prepared to follow any new rules is embracing the imperative to design trials for the highest benefit of all.

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