Pat Conley, a 72-year-old retired business analyst whose multiple myeloma relapsed last year, has never participated in a clinical trial. She was interested several times and didn’t meet the criteria. Now she’s eligible for one, but worries about the burden of regular hour-long trips from her home in Fayetteville, Georgia, to the trial site in Atlanta, as well as the copays for medical tests. “They’ll want a new biopsy, and Lordy, biopsies are not cheap,” she said.
Still, two drug regimens haven’t halted her cancer, and she wants something positive to come from her illness. “If they don’t have African Americans to test it on, how will they know it’s going to work?” she asked. “If it doesn’t help me, it’ll help my children; it’ll help somebody else.”
Pharmaceutical companies contacted by ProPublica all said diversity in clinical trials is important to ensure that drugs meet patients’ needs. The issue “is not elevated high enough in the discussion on clinical studies,” said John Maraganore, PhD, chair of the industry group Biotechnology Innovation Organization. But he added that enrolling minorities is challenging, often for reasons beyond the manufacturer’s control, and that it would require a “public-private partnership, working with the FDA and NIH [National Institutes of Health].”
Black participation reached 10% in trials for only 2 of the 31 cancer drugs: the multiple myeloma drug Darzalex (daratumumab), where the figure was exactly 10%, and Yondelis (trabectedin), which treats two types of soft-tissue cancer. A total of 12% of patients in the Yondelis trial were black, the highest proportion in the ProPublica study. Both drugs are made by Johnson & Johnson, which said it has an internal working group on trial diversity. The working group trains site leaders in best practices for diverse recruitment and seeks minority physicians to help run trials, since some patients prefer being treated by doctors of their own race.
Not enrolling in clinical trials is just one of many ways that African Americans trail white Americans in the quality of their health care. From diagnosis to death, they often experience inferior care and worse outcomes. Because some black Americans can’t afford the health insurance mandated under the Affordable Care Act, they remain less likely to have coverage than do non-Hispanic white Americans. African Americans are 30% more likely than white Americans to die from heart disease. Black mothers are three to four times as likely as white mothers to die in pregnancy or childbirth, and black children are diagnosed with autism later than white children are.
While the scarcity of African Americans stands out in ProPublica’s analysis, there appear to be gaps in participation of other minority groups as well. Asians were well represented in trials held in some foreign countries, but they made up only 1.7% of patients for drugs for which at least 70% of trials were conducted within the United States. By comparison, about 6% of the U.S. population identifies as Asian. Almost two-thirds of the trials didn’t report any Native Americans or Alaska Natives, who together make up about 2%of the U.S. population. ProPublica’s analysis excluded Hispanics, because the FDA reports did not have a separate category for them until 2017 and do not distinguish between white and nonwhite Hispanics.
The very relationship of race to drug development is fraught with controversy. Race is primarily seen as a social concept, rather than as a product of measurable biological traits. Yet there’s growing evidence that, whether for environmental or genetic reasons, drugs may have different effects on different populations.
In 2014, the state of Hawaii sued Bristol-Myers Squibb and Sanofi, the manufacturers of the blood thinner Plavix, accusing them of deceptive marketing for failing to disclose that the drug was less effective for some patients of East Asian or Pacific Islander descent. The drugmakers have denied allegations of misconduct and argue that genetic traits have not been proven to affect how well Plavix works. That case is pending. In the meantime, the FDA has added a warning to the label saying that Chinese patients are more likely to have a gene variation that renders the drug ineffective. Researchers at the University of California, San Francisco, have found that a commonly used asthma medication, albuterol, doesn’t work as well for black and Puerto Rican children as it does for European American or Mexican children.
Inadequate minority representation in drug trials means that “we aren’t doing good science,” said Jonathan Jackson, MD, founding director of the Community Access, Recruitment and Engagement Research Center at Massachusetts General Hospital in Boston. “If we aren’t doing good science and releasing these drugs out into the public, then we are at best being inefficient, at worst being irresponsible.”
The National Black Church Initiative, a coalition of 34,000 U.S. churches, urged the FDA in 2017 to mandate diversity in all clinical trials before approving a drug or device. “Simply put, the pharmaceutical community is not going to improve minority participation in clinical trials until the FDA compels them to do so via regulations,” it wrote to Commissioner Scott Gottlieb.
The FDA hasn’t done so. Although it noted in a 2013 report that a lack of diversity betrays a key ethical principle of medical research – equal justice for all – the agency has shied away from setting quotas or numerical guidelines for participation by race.
Instead, it has relied on persuasion. In its 2014 Action Plan, it said it aimed to “share best practices,” to “support” industry efforts at improving diversity in clinical trials, and to “encourage” patients to participate in trials via social media, emails, and blog posts.
“The FDA believes that enrollment should reflect the patients most likely to use a medical product,” spokeswoman Gloria Sanchez-Contreras said. The FDA “does not have the regulatory authority to require specific levels of minority representation in clinical trials,” although it may ask drugmakers for additional data, she said.