Use incentives to increase diversity in clinical trials

TLack of diversity in clinical trials unfairly biases American medicine and costs billions of dollars a year in premature deaths and ill health. Proven economic incentives to recruit more diverse participants can dramatically change the image, if the will to employ them is there.

The scope of the problem was raised in a new report that Congress asked the National Academies of Sciences, Engineering, and Medicine, and that we, as committee members, helped write.

Each year, new pharmaceutical agents and devices are tested for safety and efficacy by government and industry before they are allowed on the market. In drug trials, one group of participants usually gets the drug and another gets a placebo or the standard of care for the condition being studied.

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Large sections of the US population have not been adequately represented in these studies, so doctors lack data on new discoveries that could prevent disease and extend the lives of many of their patients. This includes underrepresented racial and ethnic populations; older adults; LGBTQIA+ communities; People with disabilities; and those who are pregnant, of reproductive age or lactating. Confidence in the safety and efficacy of a drug cannot be assured for these populations unless they are properly represented in the research.

A recent analysis of Food and Drug Administration drug approvals between 2014 and 2021 found that less than 20% of those drugs had clinical trial data on treatment benefits or side effects for black patients. The 2022 National Academies report showed progress had been made in representing white women in trials, but had stalled for people from underserved racial and ethnic populations.

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The report details several adverse impacts of low diversity in clinical trials. These include lack of access to effective medical interventions, exacerbating health disparities in excluded and underrepresented populations, and hampering innovation.

The report shed new light on a key impact: the economic cost, which affects not only underrepresented populations but society as a whole. An analysis created for the NASEM report using the future elder 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, shorter disability-free lives, and fewer working years among populations that are not are proportionally represented in clinical trials. If just 1% of health disparities were alleviated through greater diversity in clinical trials, Schaeffer’s model estimates that would result in more than $40 billion in profits for diabetes and $60 billion for heart disease. heart.

Following the money trail also leads to one of the best ways to reverse the problem: financial incentives. Currently, clinical trial sponsors have limited ability to reimburse participants, often by institutional review boards of the study. Compensating participants for lost wages, transportation costs, dependent care, and housing would help.

But greater results can be expected if industry players are given incentives to take action. Among its package of recommendations, the National Academies committee urged the FDA to study new incentives for sponsors who meet criteria for representativeness of clinical trials, including tax credits, expedited eligibility, waiver of some FDA application and expanded market exclusivity for drugs.

These recommendations mirror those included in the Orphan Drug Act of 1983, that completely changed the face of therapeutics for rare diseases. Before the law, millions of people lived with diseases that did not attract significant research funding because the patient population for each was so small that studies could take a long time and financial recovery was unlikely. Diseases were left out of the line of innovation, hence the term “orphan.” Many people with orphan diseases are still waiting for cures. However, the incentives of the 1983 legislation led the industry to develop more than 5,000 medications which received orphan drug designation between 1983 and 2019.

How might incentives for better representation in clinical trials work? A good example is the Alzheimer’s drug Aduhelm, which the FDA approved in 2021, but was rejected by Medicare for its broad coverage and 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 representation standards that the FDA would designate.

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 could help enroll more participants from underserved racial and ethnic populations in trials. 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 the fight against disease.

The stagnant state of underrepresentation in clinical research is costing the US too much and contributing to widespread health inequities. A change of course can be achieved by instituting incentives that spur the industry to action.

Dana Goldman is dean of the USC Price School of Public Policy and co-director of the USC Schaeffer Center for Health Policy and Economics. Edith A. Pérez is a hematologist/oncologist, medical director of Bolt Therapeutics, Inc., and professor of medicine at Mayo Clinic. Carlos del Rio is an infectious disease physician, Distinguished Professor of Medicine at Emory University School of Medicine, and Professor of Epidemiology and Global Health at Emory University Rollins School of Public Health. 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.”

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