They ranged in age from 15 to 61—four African American women, all with stories to tell about their struggles with sickle cell disease, all with stories about a common experience that helped them through those struggles: participating in clinical trials.
It mattered, said the women, all of whom had joined trials funded by the National Institutes of Health (NIH). Their participation helped them better manage their condition, they said. It allowed promising treatments to be tested, and at least in one case, it led to a virtual cure.
The trial participants spoke to a standing-room-only session at the Annual Sickle Cell Disease Clinical Research Meeting, hosted in August by the National Heart, Lung, Blood Institute (NHLBI), Division of Blood Diseases and Resources (DBDR). The audience included researchers, health professionals, and patients and their families, and most were acutely familiar with the problem of diversity in clinical trials generally, and for sickle cell disease patients, in particular.
The challenge, the patients said, is getting more people like them—that is, people of color—to join clinical trials. But meeting that challenge, they noted, requires aggressive efforts: creative recruitment drives, a laser focus on building trust, and a commitment to educating patients about the many benefits of signing up.
More than 100,000 people in the United States and millions more worldwide have sickle disease, an inherited, often life-threatening blood disorder that mainly affects African Americans, Hispanics and Asians. Yet fewer than 10 percent of U.S. clinical trial participants come from these populations. Indeed, Hispanics—17 percent of the U.S. population and the second group most affected by sickle cell—make up just one percent of trial participants.
Marlene Peters-Lawrence, BSN, RN, RRT, the organizer of the Promoting Diversity in Clinical Trials and Recruitment in Sickle Cell Disease session, said this dearth of participation stymies medical progress more than people realize. “The studies are delayed and our ability to test hypotheses is severely limited,” she said. But the research community, she added, must play a key role in finding ways to help turn the tide.
The problem is daunting. A law passed by Congress in 1993 requires all medical research conducted and funded by the NIH to include enough racial and ethnic minorities and women to allow researchers the ability to assess differences in response to drugs and treatments. However, NIH only pays for a small percentage of clinical research in the U.S., and the rest are not subjected to this requirement.
NIH funds clinical trials in institutions throughout the country, but poverty, geographic proximity, lack of knowledge about and trust in research, as well as language barriers can all challenge diversity in recruitment for studies.
At the meeting, Ann Farrell, M.D., director of the FDA Division of Hematology Products, discussed the agency’s work with the research community to develop methods to refine its approach to the conduct and analysis of trials to provide the best estimates of treatment effects for diverse populations.
But the trial participants at the session had of suggestions of their own.
First, get creative with recruitment, they said. Shirley Miller, 61, the spokesperson for the FDA’s Year of Diversity in Clinical Trials Campaign, called for using current study participants to recruit others. “They are going to believe them better than someone who doesn’t experience the pain,” she said.
Second, build trust by talking about the research in detail. “When I consider participating in a trial, I want you [theresearchers] to be transparent about successes and failures, so the patient knows what to expect,” said Jennifer Nsenkyire, 44, who in 2009 received an experimental bone marrow transplant that transformed her life.
“Explain how I can stop my participation in the middle of the study, and what happens if I have a bad reaction to the treatment tested. Can I receive lifetime care for that or would I be on my own? I want to understand the benefits to me and to future generations, and the risks involved,” she said.
It is common to hear that minorities mistrust researchers and are often not willing to participate, afraid of being treated as “guinea pigs”.
According to Sherrie Flynt Wallington, Ph.D., assistant professor of oncology at Georgetown University Medical Center, the mistrust is not about past abuses such as the infamous Tuskegee study in which black men were not given effective treatment against syphilis in order to study the progression of the disease – but about how patients and their communities are treated by researchers today.
The answer, Wallington said, is engaging the community in the research.
She encouraged researchers to report back to the communities with study results and to develop lay audience materials. “At the core of all community-engaged research is the understanding that the community will be involved in a meaningful way,” Wallington said.
Trust and involvement is also key at the patient level, and explaining with candor the multi-layered benefits of the research—that it is for the greater good, not just for the individual patient—is part of building that trust, one patient said.
“There is a misunderstanding as to what patients consider to be a benefit to them,” said Nkechinyem (Nke) Nwabuzor, 39, attorney and advocate. “Part of it is contributing to the knowledge of the disease as a whole.”
This view was validated by the panelists’ unanimous “yes” when asked if they would allow, with their consent, the use of their personal data for research of other diseases and other trials.
Francine Baker, who joined her daughter Jasmin, the 15-year old sickle cell disease patient and advocate, put it best. “Giving back is rewarding,” she said. “Having my children participate in clinical trials has been rewarding for them as well, and they have learned so much about their disease.”
And with that, Jasmin offered a rousing sendoff message for researchers. “Thank you!” she said. “Even if you don’t see change, there is change. Don’t give up!”
The audience rose to its feet.