The lack of diversity in clinical trials unfairly skews American medicine and costs billions of dollars a year in early deaths and poor health. Proven economic incentives to recruit more diverse participants can dramatically change the picture — if the will exists to employ them.
The scope of the problem was laid out in a new report that Congress requested from the National Academies of Sciences, Engineering, and Medicine, which we as committee members helped write.
Each year, government and industry test the safety and efficacy of new pharmaceutical agents and devices before they are allowed on the market. In drug trials, one group of participants typically receives the drug and another gets a placebo or the standard of care for the condition being studied.
Large swaths of the U.S. population have not been adequately represented in those studies, so clinicians lack data on new discoveries that might prevent disease and extend life in many of their patients. This includes underrepresented racial and ethnic populations; older adults; LGTBQIA+ communities; individuals with disabilities; and those who are pregnant, of reproductive age, or lactating. Confidence in the safety and effectiveness of a drug for those populations can’t be assured unless they are properly represented in the research.
A recent analysis of Food and Drug Administration drug approvals between 2014 and 2021 found that fewer than 20% of those drugs had clinical trial data regarding treatment benefits or side effects for Black patients. The 2022 National Academies report showed that progress had been made on representation of white women in trials, but had stalled for people in underserved racial and ethnic populations.
The report details several adverse impacts from poor diversity in clinical trials. These include lack of access to effective medical interventions, compounding of health disparities in underrepresented and excluded populations, and hindering innovation.
The report shed new light on one key impact: the economic cost, which affects not just the underrepresented populations but society at large. An analysis created for the NASEM report using the Future Elderly Model developed by the USC Schaeffer Center found that hundreds of billions of dollars will be lost over the next 25 years due to reduced life expectancy, shortened disability-free lives, and fewer years working among populations that are not proportionately represented in clinical trials. If just 1% of health disparities were alleviated by improved diversity in clinical trials, the Schaeffer model estimates that would result in more than $40 billion in gains for diabetes and $60 billion for heart disease.
Following the money also leads to one of the best ways to turn the problem around: financial incentives. Currently, clinical trial sponsors are limited in their ability to reimburse participants, often by study institutional review boards. Remunerating participants for lost wages, transportation costs, dependent care, and housing would help.
But bigger results can be expected if industry players are offered incentives to take action. Among its bundle of recommendations, the National Academies’ committee urged the FDA to study new inducements for sponsors that meet clinical trial representativeness criteria, including tax credits, fast-track eligibility, exemption from some FDA application fees, and extended market exclusivity for drugs.
These recommendations mirror ones included in the Orphan Drug Act of 1983, which completely changed the face of therapeutics for rare disorders. Before the act, millions of people were living with diseases that were not attracting significant research funding because the patient population for each one was so small that studies could take a long time and financial recovery was unlikely. The diseases were left out of the innovation pipeline, hence the term “orphan.” Many people with orphan diseases still await cures. Nevertheless, inducements in the 1983 legislation led industry to develop more than 5,000 drugs that received orphan drug designation between 1983 and 2019.
How might incentives for better representation work in clinical trials? A good example is the Alzheimer’s drug Aduhelm, which the FDA approved in 2021 but was rejected for broad coverage by Medicare and was sent back for additional, more representative trials. Congress could create a strong incentive for more inclusive trials by guaranteeing Medicare coverage for drugs that meet standards of representation that would be designated by the FDA.
The National Academies report recommends that the Department of Health and Human Services, the FDA, the National Institutes of Health, and other federal agencies write new regulations and impose new requirements for diversity in clinical trials. Some of these might indeed help enroll in trials more participants from underserved racial and ethnic populations. But the success of the Orphan Drug Act should be clear proof that incentives are strong and effective levers for driving change in research and in fighting diseases.
The stagnant state of underrepresentation in clinical research is costing the U.S. too much and contributing to widespread health inequities. A turnaround can be achieved by instituting incentives that will spur industry to action.
Dana Goldman is the dean of the USC Price School of Public Policy and codirector of the USC Schaeffer Center for Health Policy and Economics. Edith A. Perez is a hematologist/oncologist, chief medical officer of Bolt Therapeutics, Inc., and professor of medicine at the Mayo Clinic. Carlos del Rio is an infectious disease physician, a distinguished professor of medicine at Emory University School of Medicine, and professor of epidemiology and global health at the Rollins School of Public Health of Emory University. The authors were members of the committee that wrote the National Academies of Sciences, Medicine, and Engineering report “Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups.”