After the FDA released draft guidance on increasing diversity in clinical trials, companies have been left to figure out the details. Here’s what experts say is working.
Pictured: A child flexes his arms for a doctor in a clinic as his mother looks on/Getty
Last year, the FDA released a draft guidance on increasing diversity in clinical trials. Reflecting a surge in efforts to correct the historical exclusion of non-white communities, the guidance also suggests including people with varied gender identities, pregnancy statuses, ages and comorbidities.
Despite increased attention to the need for diversity, it appears that on the whole, clinical trial sponsors are fumbling when it comes to setting and meeting realistic diversity goals: A 2023 report by the IQVIA Institute for Human Data Science showed that the inclusion of Black/African American and Hispanic patients has dropped over the past 5 years, reaching a historic low in 2022.
In a bid to address this issue, the FDA guidelines were folded into the Food and Drug Omnibus Reform Act of 2022 (FDORA), which mandates that clinical trial sponsors submit diversity actions plans to the Secretary of the U.S. Department of Health and Human Services. These plans must lay out the sponsors’ enrollment goals, why they set these goals, and how they plan to meet the goals. As neither the draft guidance nor FDORA prescribe concrete steps, it falls to companies to figure out the details. In such cases, they often rely on clinical trial officers and contract organizations who specialize in diversity, equity and inclusion (DEI). BioSpace spoke with some of these specialists about some key elements of a successful plan.
Integrate DEI from Start to Finish
Most experts agree that diversity and representation must be prioritized from the very beginning of a trial. “The sooner you can do it in the trial planning process, the better because it takes so much more time,” said Rachel Rangel, a clinical trial specialist at Curavit. A contract research organization (CRO), Curavit helps companies determine who they should be enrolling, what has historically stopped these populations from being recruited, and how to best include them in trials today.
Denise Bronner, the director of Diversity, Equity and Inclusion in Clinical Trials at Johnson & Johnson Innovative Medicine, also emphasizes the importance of an early start. For instance, if the initial dosage and safety studies are done on participants who are mostly white and male, then “you really have nothing to inform you of what happens at the next stage,” she said.
Even before beginning trials, recruitment materials should be designed with accessibility in mind. Simple measures like using large font sizes or offering instructions in both written and audio form can go a long way in making trials accessible to a wider swathe of the population, said Courtney Firak, a consultant who helps companies come up with strategies to increase diversity in trials.
Once a diversity plan is set in place, the trial protocol can be optimized to enroll the right participants. This often involves revisiting stringent eligibility criteria like blood markers, which have been shown to restrict participation by members of some groups. For instance, Black/African American people tend to have higher rates of anemia and lower white blood cell counts than white people, rendering many potential participants ineligible for trials that have strict criteria involving these measures. By carefully combing through demographic data and comparing it with the enrollment profiles of old trials, companies can figure out which communities have been left out purely because they did not meet such criteria.
Picking the right study site can also allow people from different groups to participate. This can mean scouting locations in “diversity hotspots” with large numbers of patients, or even in countries outside of the U.S. “These drugs are going into a global market, so you should be thinking about ways to make sure that other countries and other groups are being represented,” Bronner said. Rangel endorses using patient data creatively to find good study sites. For a MedRhythms study on a treatment to help stroke victims walk better, she combined data on the geographic prevalence of stroke with data on populations known to suffer from the long-term effects of stroke. This enabled her to generate a map of potential study sites near people who could benefit from the trial.
One can cast a wide geographical net by conducting decentralized clinical trials, which effectively allow people to participate from their own homes and be monitored through devices or virtual check-ins. By removing the need to visit a trial site, decentralized trials not only expand the geographical reach of the study but allow people with disabilities and demanding responsibilities like caregiving to take part.
A good DEI plan is carried through until the end of, and even beyond, a trial. Every completed trial is a valuable data point that can help companies assess where they have done well and where they have fallen short. Bronner recalls looking back at old trials that fell short of diversity targets to pinpoint where in the pipeline DEI strategies should have been implemented but were not, and learning from those failures. “The trigger, so to speak, that made us think about doing this earlier was looking at those studies consistently and saying, ‘we’re coming in too late,’” she said.
Set Reasonable Benchmarks
One way to assess and facilitate the success of DEI strategies is to set realistic and attainable benchmarks, such as enrolling a target percentage of people from a given community. This can look different for different studies depending on the disease, patient population, geographic location and so on. Bronner recommends drawing from as many databases as possible to get a good sense of patient demographics and disease incidence. Firak further stresses the importance of thinking beyond race and ethnicity, urging companies to gather as much data as possible on intersectional identities that include gender, sexuality and disabilities to account for different perspectives and lived experiences. When collected early on, this data can give sponsors a good picture of the populations they should aim to enroll. “A good diversity goal is if it looks like the population as a whole,” Firak said.
Just this year, trial organizers at Johnson & Johnson realized they had overestimated the difficulty of enrolling diverse trial participants and set too small a goal. After originally aiming for about 35% non-white participants in some of its immunology trials, the company overshot its diversity goal by enrolling close to 50% non-white participants. Organizers also completed some of the trials ahead of schedule and saw that the drop-out rate of non-white participants was no higher than that of white participants. Bronner asserts that this dispels three longstanding myths: that recruiting diverse participants is difficult, that it will take too long, and that non-white participants are more likely to drop out of trials.
Forge Relationships with Communities
One of the most time-consuming aspects of a DEI plan is building relationships with communities, especially those with a history of mistrust in or exclusion by the medical system. A 2019 study showed that a lack of information and trust were critical barriers to participation in clinical trials. One of the ways Johnson & Johnson has tried to address this is by setting up websites like Research Includes Me, which lays out the clinical trial process and how people of color have not been included in the past. Bronner also cautions against “selling” community members a treatment. “You’re really trying to educate them and to be there to answer their questions,” she said. She herself has twice been a speaker at Black Health Matters summits, helping inform community members about Black representation in clinical trials and health disparities in heart disease and multiple myeloma.
A foolproof way of addressing the concerns of specific communities is to simply go to the source and ask people what they need. “It’s a matter of identifying the right partners who have already put that time in, to kind of work with them, get their expertise, and leverage that to your best advantage because we all know that we want to get trials off the ground,” Rangel said.
Firak recommends working with patient advocacy teams who are attuned to the needs of different communities and know how to manage expectations. “It’s likely going to be different strategies for different groups,” she said. “Especially when we’re talking about historically marginalized communities…it will take time and resources to gain that trust.”
Sruthi S. Balakrishnan is a freelance academic editor, science writer and fact-checker based in Santa Barbara, California. She can be reached at sruthisb@sciscriber.com.
