Cancer Clinical Trials Save Lives, and Diversity Matters

It’s not only a matter of social justice. Diverse clinical-trial participation also means better science.

“Different groups of people may respond to a given medication in different ways,” including efficacy and side effects, the U.S. Food & Drug Administration says. When certain populations are underrepresented in medical research, the result can be further health disparities because treatments work better for some people than others. And more diverse trial enrollment has been shown to increase public trust in research results as well as broaden acceptance of the resulting treatments.

In early 2023, Congress instructed the FDA to require that researchers submit plans ensuring participant diversity before the agency can give the go-ahead to late-stage clinical trials that can lead to approval of new drugs.

From left: Christopher Lieu, MD; Evelinn Borrayo, PhD; and Jessica McDermott, MD, of the CU Cancer Center leadership team.

A long-standing priority

At these meetings, investigators present their clinical trials so that community members can advise them. That advice can include reviewing proposals and study materials to ensure they are relevant to Colorado’s diverse communities, guidance on recruitment and retention of diverse trial participants, and input on how to disseminate research findings.

Anthony Elias, MD, a CU Cancer Center member and medical oncology professor, briefed the group on sarcoma studies at its February meeting. He says COE-CARES will be a “terrific” resource on clinical trials and spoke to members of the importance of having patient advocates “review our consent forms, our goals, and our rationale for doing the trials that we’re doing. That’s really valuable.”

A participation gap

The vast majority of cancer patients don’t participate in clinical trials for various reasons. Often, patients don’t understand that a clinical trial can offer a treatment option, they don’t know about available trials, there isn’t a suitable trial underway, they aren’t eligible for a trial for medical reasons, or they’re worried about cost, side effects or logistical issues. In a National Cancer Institute (NCI) survey, 41% of Americans said they didn’t know what a clinical trial was.

A 2021 study of data from 1,200 U.S. cancer institutions calculated that only 6.3% of cancer patients participate in clinical trials, although the percentage for patients at NCI-designated comprehensive cancer centers like the CU Cancer Center is higher: 18.9%.

The participation gap is more pronounced for communities that are traditionally underrepresented in American society. For example, a 2019 study published in JAMA Oncology, examining 230 trials from 2008 to 2018 that led to FDA cancer drug approvals, found that Black and Hispanic people were "consistently underrepresented compared with their burden of cancer incidence” in those trials.

A 2022 report from the American Association for Cancer Research reported similar underrepresentation of Black, Hispanic, and Native American patients.

And a 2022 report to Congress by the U.S. Government Accountability Office (GAO) said various other groups — including adolescents, older adults, women, low-income people, and residents of rural areas — also were underrepresented in cancer clinical trials.

In addition to the factors contributing to the overall shortage of trial participants, the GAO report cites other factors that can contribute to underrepresentation of certain groups. They include bias impacting a health care provider’s decision to mention the option of clinical trial participation to a patient, logistical barriers such as distance to a trial site or inability to take time off work, a lack of trial staff “that shares cultural characteristics with the patients,” and language barriers.

Challenges and solutions

In interviews, CU Cancer Center leaders detailed several approaches underway to meet challenges in the way of greater and more diverse participation in cancer clinical trials. At the top of the list is access.

“Many of our underserved populations are uninsured or under-insured,” Lieu says. “Part of accessing our clinical trials is our patients’ ability to access the health care system in the first place. We need to make sure they have access. This has been a collaboration between the cancer center and UCHealth University of Colorado Hospital to help provide financial support and backing for patients.” Uninsured patients are referred to a navigator at the hospital for a medical review to see if they qualify for discounted care.

McDermott notes that Wells Messersmith, MD, associate director for clinical services at the CU Cancer Center and chief medical officer of oncology services at UCHealth, has worked with Khari Reed, metro-Denver senior director of oncology services at UCHealth, the cancer center’s primary clinical partner, to expand eligibility among underinsured patients to be seen by cancer center providers.

Education and outreach to patients and community health care providers also are top priorities for COE and other cancer center segments.

“Whether it’s a patient’s knowledge of what clinical trials are or whether they’re eligible for them, or whether their primary care doctors or community oncologists know what trials are available, we’re working on that,” McDermott says. In 2023, COE took part in 36 health fairs in 25 counties across Colorado, targeting underserved communities. The office also runs clinical trials education programs, partners on outreach groups like the Colorado Cancer Coalition, and meets with local providers.

“Physicians and providers can take time out of their schedule to meet with advocacy groups and community organizations,” Lieu says. “Community events that bring patients and providers together, not in a hospital setting, are really helpful.”

Patient navigators

Language can also be a significant barrier. “Given Colorado’s demographics, many of our patients are Spanish speaking,” Lieu says. “So we need to provide those patients with Spanish-speaking support. These are critical to making sure these patients can navigate a very complex system.”

Borrayo notes that her office helps investigators translate trial protocols and strategies to help trials teams with diverse patients. The COE has a cultural linguistic coordinator as a consultant and plans to hire for the position soon. The CU Cancer Center also recently hired a COE liaison to the cancer center’s Cancer Clinical Trials Office (CCTO).

“We’re increasing access to bilingual navigators and nurses and physicians and social workers who can help people get through the process,” adds McDermott. “And we’re trying to make sure that if a patient might be eligible for a trial, all the paperwork that they would need to have eyes on is translated into a language they understand.”

COE already has hired two bilingual patient navigators to serve underrepresented communities and people, Borrayo says. It’s the navigators’ role to help steer patients “from the community through the hospital and to clinical trials and completion,” she says.

Carlo Caballero, LPC, CCM, is one of the patient navigators. “Last year, we fleshed out the process of how to get a patient into a clinical trial,” he says. “We realized there were lots of steps, from getting in the door to getting into a clinical trial to remaining in the trial, and a lot of patients would fall through the cracks.”

Daniel Pacheco, MBA, PMP, COE program manager, worked with Caballero and others to develop the patient navigator program out of a 2020 pilot project, led by McDermott, called E3, for Esperanza en Español (“hope in Spanish”), formed in part to help Spanish speakers access cancer clinical trials.

“We thought it would be a real easy thing,” Pacheco says. “But we realized we had to create a new system to get patients into trials. Now we have a whole group of people from every cancer center department working on it.”

From left: Sindy Gonzalez, MPH, COE-CARES program coordinator; Dan Pacheco, COE program coordinator; Carlo Caballero, LPC, CCM, patient navigator.

There’s a geographic challenge, too. “If patients live in rural or remote areas, that makes it a lot more difficult” to take part in clinical trials in metro Denver, McDermott says. She notes that rural counties in south and east Colorado tend to rank high on the state’s “cancer disparity index” —a combination of high cancer rates, mortality, and frequent late-stage diagnosis.

“An ongoing goal for us is to figure out how to decentralize some of these trials,” she says. “That could mean opening up trials at some distant community health centers, or if that isn’t possible, having patients enroll in trials through the main campus, but get some of their tests and studies done closer to home.”

Looking inward

While these and other CU Cancer Center initiatives are focused outward, its leaders are also looking inward — at ways to rethink clinical trials so they are open to a broader spectrum of participants, and at keeping better track of inclusiveness in trials.

“We’ve been working on the design of trials to make it more realistic for a large population to be enrolled,” McDermott says. “Some trials that are designed by pharma companies have really strict inclusion-exclusion criteria, like tight blood pressure parameters or not allowing patients with HIV, things that might exclude a large portion of a certain population, things that sometimes aren’t that important to a particular drug or study. We’ve been trying to work both internally and externally to broaden those criteria as well.”

Redesigning trials also can mean altering schedules for blood tests and appointments to make them less burdensome on participants, she says. “Some people can’t miss work or school two or three times a week to get these kinds of things done.”

Meanwhile, the CU Cancer Center has a Protocol Review and Monitoring System (PRMS), a committee that reviews protocols for oncology clinical trials on campus. Part of its mission is to review accrual of participants.

“For the past couple of years, the PRMS committee has been collecting data for each tumor type — how many patients are enrolling in trials and the demographics of those patients, and then presenting it back to the investigators so we can see how we’re doing,” McDermott says.

That data “can sometimes be eye opening,” Lieu says. “You may think that you’re enrolling a diverse patient population, but the reports highlight areas of success and where there can be improvement. I think that has helped our investigators ensure who they’re enrolling reflects our state’s population.”