Congress Should Take This Chance to Fix Clinical Trial Diversity

Recently, Congress missed a critical opportunity to ensure novel drugs are tested in diverse patient populations.

Ahead of its expiration on September 30 and despite bipartisan support for policies to improve clinical trial diversity, legislators passed a “clean” FDA user fee reauthorization bill, stripped of necessary public health provisions to advance a more equitable clinical research ecosystem.

But there might be another opportunity in the end-of-year spending bill for Congress to ensure that patients and their doctors know that clinical trials supporting Food and Drug Administration approvals are diverse.

Authorization Process

Every five years, Congress reauthorizes the user fee program, which allows the FDA to collect payments from pharmaceutical and medical device industry sponsors to fund its operations.

Historically, additional provisions have been added to this legislation, often in favor of the pharmaceutical and medical device industries, including establishing new programs for expediting FDA approval, relaxing evidentiary standards for approval, and the like.

This year’s authorization was different, driven by a controversial FDA decision to approve aducanumab, Biogen’s Alzheimer’s drug, and corresponding pressure on Medicare to cover the costly and potentially ineffective drug.

Many noted the drug’s clinical trials enrolled too few Black and Latinx-identifying patients in comparison to their representation among Alzheimer’s patients.

As a result, measures to improve clinical trial diversity were introduced into the user fee legislation. While they did not make it into the final user fee bill, leaders of the House Energy and Commerce and Senate Health, Education, Labor and Pensions Committees committed to incorporating these long-overdue provisions to improve trial diversity into the end-of-year spending bill.

Diversity Needed

Clinical trials enrolling diverse patients help demonstrate to patients and clinicians that new drugs and devices are safe and effective in the diverse patient population expected to use them.

Clinical trial diversity is important because research suggests there can be differential response to drugs by sex and age due to biological differences in how drugs are metabolized, and drug toxicity.

While there are no inherent biological differences by race and ethnicity, patients of different races and ethnicities may respond differently to drugs and biologics due to health disparities driven by structural racism and social determinants of health.

Since the 1980s, FDA has made efforts to encourage clinical trial diversity. More recently, in 2014, FDA introduced a five-year action plan to “encourage greater participation of diverse populations in clinical trials” including establishment of the Drug Trials Snapshots program to increase transparency around the demographics of trial participants.

Data Gaps

However, despite these FDA efforts, Drug Trials Snapshots still has missing data. Poor data availability impedes the biomedical research community’s ability to benchmark representation and assess progress, particularly for smaller demographic groups of the US population—i.e. Native Hawaiians and Pacific Islanders, American Indians, and so forth.

Moreover, representation in trials across gender, age, and race supporting FDA approval of new medical products continues to be inadequate. One study of more than 200 cancer drugs approved between 2008 and 2018 found suboptimal reporting of race data and underrepresentation of Black and Latinx patients. Other studies have similarly found women and older adults are underrepresented in trials.

Diversity Plans

In April, the FDA released new draft guidance recommending that sponsors develop “diversity plans” prior to initiating clinical trials. In these plans, sponsors would include enrollment targets for racial and ethnic minorities and how they plan to recruit adequate numbers to meet these targets.

By asking for diversity plans, the FDA can provide feedback prior to the implementation of the clinical trial and offer guidance to sponsors seeking to improve diversity in their clinical trials.

However, FDA guidances are only recommendations and sponsors could simply choose to not follow them—similarly to how they have not done before for other such guidances from the FDA meant to improve representation in clinical trials.

Under the bipartisan House user fee legislation that passed over the summer, the FDA would have been granted the authority to require similar “diversity action plans” with specific enrollment targets.

Moreover, the FDA would also draft guidance specifying under which circumstances sponsors would have to run additional, more diverse clinical trials following FDA approval, indicating a possible enforcement mechanism when sponsors fail to meet their diversity enrollment targets.

The trade associations for drug manufacturers have argued submitting diversity action plans is burdensome and could slow clinical research. However, this is not much different from what sponsors already are required to do in submitting protocols and statistical analysis plans prior to study initiation.

Setting clear enrollment targets for diverse populations will make it more likely for the results of clinical trials to be useful for diverse populations in practice, mitigating the need for additional time-intensive studies in the future to determine if the treatment tested is beneficial across patients.

Proposed legislation also allows FDA to determine whether development and implementation of such plans on a case-by-case basis is truly a barrier and enables the agency to play an assistive role.

By passing measures to improve clinical trial diversity, patients will be reassured that FDA-approved drugs and devices are being tested in trial participants like them.

History has shown that despite FDA’s best efforts to encourage such diversity, representation in clinical trials has lagged. Congress must not let the end-of-year spending bill to be another missed opportunity to pass such meaningful FDA reforms.

This article does not necessarily reflect the opinion of Bloomberg Industry Group, Inc., the publisher of Bloomberg Law and Bloomberg Tax, or its owners.

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Author Information

Jennifer E. Miller is a bioethicist and associate professor at Yale School of Medicine and founder of Bioethics International. She led several studies examining representation within clinical trials supporting FDA approval.

Reshma Ramachandran is a family medicine physician and assistant professor at Yale School of Medicine. She co-directs the Yale Collaboration for Regulatory Rigor, Integrity, and Transparency and chairs the FDA Task Force for Doctors for America.

Tanvee Varma is a final-year medical student at Yale School of Medicine who has conducted and published research on clinical trial diversity.