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FDA and drug makers deepen their commitment to clinical-trial diversity. Will it work?

By Jaimy Lee

Stephanie Walker, a Black retired nurse who was diagnosed with metastatic breast cancer in 2015, has never been asked to participate in a clinical trial. But if the opportunity to do so came up, she would take it in a heartbeat. 

“Give me the chance to say no after I’ve received all the information,” Walker, 63, told MarketWatch. “Let me make that informed decision.”

Black Americans like Walker are rarely asked to participate in clinical research. In fact, a survey of about 400 people she recently conducted for the Metastatic Breast Cancer Alliance found that 83% of Black patients with metastatic breast cancer said they would consider participating in a clinical trial, but 40% said no one on their care team had asked them about participating in a study. 

“Nobody’s telling us about clinical trials,” Walker said. “Why is that?”

An estimated 8% of people who participated in clinical trials for new drugs approved in 2020 identify as African American or Black; roughly 3% or 4% of people who participate in cancer trials in the U.S. are Black; and only 3% of participants in studies for new cardiovascular therapies are Black.

This means that Black patients rarely get the option to try potentially groundbreaking therapies being tested in clinical research, and the treatments that become the standard of medical care in the U.S. are seldom tested in the 13% of the U.S. population that identifies as Black. And that raises questions about which patients qualify for new drugs, whether due to federal health guidelines or health insurance coverage, and how the lack of inclusion can damage trust in medicine.

“Large swaths of the U.S. population, and those that often face the greatest health challenges, are less able to benefit from these discoveries because they are not adequately represented in clinical research studies,” the authors of a congressionally mandated National Academies of Sciences, Engineering, and Medicine report wrote this spring.

Though Black mistrust of the U.S. medical system is often blamed for racial disparities in clinical trials, there are several less-cited and perhaps more influential reasons behind the lack of representation in medical research, including a lack of federal incentives, lackluster efforts by pharmaceutical companies, a clinical-trial workforce that is largely white, and physicians who have stuck to longstanding and racially biased beliefs about Black people’s unwillingness to participate in medical research. 

But the COVID-19 pandemic and its disproportionate impact on people of color turned clinical trials into a well-known issue and created greater public awareness about the lack of people of color in medical research. That heightened interest, experts say, may fuel the kind of systemic change that has long been needed to ensure that clinical trials reflect the U.S. population.

“All the publicity about, and now literacy about, medical research and trials and what they mean and how the process works in terms of FDA approvals has opened a window that may not remain open for a long time,” said Dr. James Hildreth, the president and CEO of Meharry Medical College, a historically Black medical school in Nashville, Tenn. “But at least right now is a great time to continue to build on the momentum that was put into place by the response to COVID-19.”

The problem with ‘preconceived notions’

There are two buckets of issues that have to be addressed to ensure that clinical trials adequately represent any patient population, with respect to everything from race, ethnicity and gender to breastfeeding and socioeconomic status. 

There’s the operational stuff, like the location of the trial sites; whether participants are paid; whether the sponsor covers the cost of parking, meals, or child care; and whether it’s possible for follow-up appointments to be done remotely using telehealth or at local clinics, rather than at the clinical-trial site. Making these options available to participants can ease concerns for people who have less-flexible work or child-care schedules and may not have extra money or time. 

But what’s much more complicated is addressing why most clinical-trial participants are still white. Doing so raises uncomfortable questions about individual and institutional bias among the specialist physicians, clinical-trial investigators and pharmaceutical executives responsible for enrolling Americans in clinical research. 

“Principal investigators and study staff also bring their own biases to the research enterprise,” the authors of the National Academies report wrote. “Their perceptions about a potential participant’s reliability, health literacy, language skills, and social support, among other factors, all play into whether the potential participant will be offered information on enrollment.”

When it comes to explaining why fewer Black people than white people are included in clinical trials, the go-to answer is often the infamous Tuskegee study, which purposely and without consent infected poor Black men with syphilis starting in the 1930s, withhold treatment, and never told them about their illnesses. It’s considered one of the most unethical studies ever conducted in the United States. 

But medical racism still shows up in exam rooms across the country every day. Some medical students and residents falsely believe Black people feel less pain than white people, based on centuries-old and debunked thinking that there are biological differences between races, and Black patients may be less likely to comply with medical treatment plans. This means Black patients have been historically and systematically undertreated for pain compared with their white counterparts.

Also read: Black and Hispanic communities say doctors and hospitals treat them differently from their white counterparts  

It also means Black patients are more likely to report that their doctors don’t believe them . For example, when tennis superstar Serena Williams had an embolism after undergoing a C-section, she had to insist on medical attention, later writing in Elle that Black maternal mortality rates would differ “if the medical establishment listened to every Black woman’s experience.”

A Pew Research Center survey from November 2021 found that Black Americans are less likely than white or Hispanic Americans to say science has had a good impact on society.

In a similar vein, clinical research has long preferred the “male norm,” aka young and middle-aged healthy white men, according to the same National Academies report. That preference, which extends far beyond the clinic and into everyday medicine, comes at the exclusion of women and people of color. 

“Some physicians may think Black patients don’t know what clinical trials are, or can’t understand them, or won’t complete a clinical trial, or can’t understand the elements of one,” Walker said. “These little preconceived notions that you have that are monumental … keep us from even participating in a clinical trial.”

These biases set off a chain reaction that highlights how the design of clinical trials can be more prohibitive than Black mistrust of medicine: If physicians don’t tell Black patients about medical studies, they can’t decide whether to enroll in them. If regulators don’t require pharmaceutical companies to diversify their trials, they may choose not to. And if drug makers only partner with the same academic medical centers in the same cities to conduct medical research, that eliminates patients who are cared for in community health centers or live in rural communities.

“Our research establishment is too much concentrated in major medical centers,” Dr. Janet Woodcock, the then-acting commissioner of the Food and Drug Administration, said in January . “We had people dying in community hospitals everywhere, and yet they did not have access to clinical trials. And we couldn’t enroll them in order to learn faster about what worked for COVID and what didn’t work. And, even in the vaccine trials, specific efforts had to be made to have real representation of America in those trials. So I think what we learned is we have a long way to go.”

What we learned in 2020

Back in 2020, the FDA “strongly” encouraged the COVID-19 vaccine makers to design clinical trials that reflected the U.S. populations most affected by the virus. 

In the race to have the first FDA-authorized COVID-19 shot, Moderna (NAS:MRNA) and Pfizer (NYS:PFE) began sharing the demographics of participants enrolled in the Phase 3 clinical trials for the COVID-19 vaccines on their websites at a time when many Americans were closely watching the clinical trials, learning how medical research worked, and paying attention to differences in efficacy rates. About 10% of the participants in Moderna’s Phase 3 clinical trial in 2020 were Black, while 9.3% of the participants in the BioNTech and Pfizer study identified as Black or African American.

By the first half of 2021, most Americans knew getting vaccinated was safer than getting COVID-19, and that the shots were safe and effective, according to the Annenberg Science Knowledge survey.

“People paid attention to the science for the first time,” said Mary Stutts, a longtime pharmaceutical executive and the chief inclusion officer at Real Chemistry, a healthcare communications and innovation firm. “You had people talking about the difference between the J&J vaccine vs. Pfizer vs. Moderna. Talking to their primary-care doctors. Asking the questions about the difference.”

(Pfizer, for the record, still tracks enrollment demographics in its medical studies. “It’s embedded into the way that we work,” said Judy Sewards, the company’s head of clinical trial experience.)

That level of public awareness combined with corporate interest is fueling much of the activity we’re seeing today. Meharry’s Hildreth said more drug makers are contacting the medical college about clinical trials than they have in the past, shaking up a tradition in which some companies made lackluster attempts to enroll and retain people of color in pharmaceutical research. 

What’s happening now, Hildreth noted, is much more intentional than what has happened in the past. “I’m happy to see that some companies have been very aggressive in trying to leverage the moment that we’re in,” he said. 

Pfizer last year shared a decade’s worth of data on clinical-trial diversity for the first time in its history (and perhaps the first time in the industry), finding that the percentage of Black patients and women who participated in Pfizer’s clinical trials between 2011 and 2020 was equal to U.S. Census population levels. However, the company also discovered that there was “marked underrepresentation” of Black and Latino populations in cancer trials, which made up about 35% of the studies included in the retrospective analysis .  

Novartis (NYS:NVS) in 2021 gave about $13 million to the Morehouse School of Medicine, a historically Black medical school, to establish a clinical trial center of excellence. (It just announced plans to invest an additional $17 million in similar centers at three more historically Black medical schools.) Roche Holding’s (SWX:CH:ROG) Genentech set up a network of cancer trial sites in historically Latino and Black cities like San Antonio and Birmingham, Ala., while the Bristol Myers Squibb (NYS:BMY) Foundation is now providing clinical-research training to 250 medical students and physicians of color in varying areas of medical expertise. 

“What we’re trying to do is assure that our clinical trials reflect a population that mirrors the real world and is aligned with epidemiology of the disease,” Lorena Kuri, Bristol’s director of clinical-trial diversity strategy. “We need to think about that from the very beginning of the design of our trials.” 

While all of these programs are impressive in scope and detail, the recent involvement of regulators is what may put heat on companies and how they approach representation in clinical trials. 

The Centers for Medicare and Medicaid Services, which decides whether Medicare will pay for therapies or procedures, this year told the makers of the Alzheimer’s disease treatment Aduhelm and similar monoclonal antibodies in development that clinical-trial participants need to be representative of Alzheimer’s patients in the U.S. This came after the FDA approved Biogen’s (NAS:BIIB) Aduhelm using data from two clinical trials that enrolled 19 Black patients out of a total of 3,285 participants. That’s less than 1% of the total participants, though roughly 14% of seniors with Alzheimer’s in the U.S. are Black. (Biogen disagreed with the CMS’s decision, though it said it supports the agency’s efforts toward “better diversity in clinical trials.”)

The FDA is firming up some of its own policies. The regulator this spring put out draft rules recommending drug makers present plans to enroll people of color in research for new drugs before they begin the large, pivotal Phase 3 clinical trials used to inform FDA applications. It also recently chose not to approve at least two cancer therapies based on clinical trials conducted in China. The FDA told one of those companies — Hutchmed (NAS:HCM) , a Chinese drug maker — that it needs to conduct a clinical trial in more than one region or country to get approval for its pancreatic tumor treatment in the U.S.

Walgreens Boots Alliance (NAS:WBA) cited the FDA’s guidance when it announced the launch of a new business that will use its stores and clinics as clinical-trial sites, particularly in underserved or rural communities. A proposed joint venture between HCA Healthcare (NYS:HCA) , a publicly traded hospital company, and healthcare distribution giant McKesson (NYS:MCK) announced in June aims to enroll people in “underserved communities” in cancer trials.

Corporations and regulators appear to be speaking the same language, and that’s happening at a time in which the pandemic forced the stagnant U.S. healthcare system to adopt telehealth and move as much care as possible out of hospitals.  

No longer do potential clinical-trial participants have to live within miles of a major academic medical center. They can use telehealth for some follow-up visits and get lab work or imaging scans close to home and have those results shared remotely with the trial team. Plus, millions more people now understand how clinical trials work and why it’s important to participate in them, and companies have paid close attention to that engagement.

“We have a long way to go,” said Walker, the patient advocate for the Metastatic Breast Cancer Alliance. “COVID, on the other hand, has taught us a lot that we can utilize with clinical trials.”

An earlier version of this story incorrectly described the Bristol Myers Squibb effort. It is a program of the Bristol Myers Squibb Foundation.

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