Food and Drug Administration to Require Diversity in Clinical Drug Trials

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According to Nature, the U.S. Food and Drug Administration (FDA) will soon require researchers and pharmaceutical companies seeking approval for late-stage clinical drug trials to submit a plan for ensuring diversity among trial participants.

This new regulatory push was telegraphed by statutory language in the end of year spending bill signed into law by President Biden at the end of December 2022. Broadening and diversifying the people who participate in clinical drug trials is important to make medicines more effective for the entire U.S. population, researchers explained to Nature. “[T] his is the biggest change in the regulatory landscape for clinical-trial diversity in the last three decades”, says Thomas Hwang, a physician at the Dana-Farber Cancer Institute in Boston, Massachusetts, who has expertise in pharmaceutical regulation.

As Nature explains:

“The diversity requirement arrives in the wake of a 2022 report from the US National Academies of Sciences, Engineering and Medicine, which found that, although the representation of white women in clinical trials has improved, progress has “largely stalled” for minority racial and ethnic groups. Older adults, pregnant people and individuals with disabilities remain severely under-represented — and, in some cases, excluded — from US clinical research, the report found.

For example, an analysis of cancer therapeutics approved by the FDA between 2012 and 2017 found that 79% of the clinical trials that were used to support the FDA’s decisions adequately represented women — but only 27% adequately represented older adults, and only 11% met the bar for minority racial and ethnic groups. (In this case, ‘adequately represented’ means that the percentage of, say, women enrolled in a cancer trial approximately matches the proportion of women in the United States who have that type of cancer.)

When the make-up of a pool of participants doesn’t reflect the population that could benefit from a particular drug, it suggests that the clinical-research findings will not be relevant for everyone, says Marian Knight, a perinatal epidemiologist at the University of Oxford, UK. It also undermines trust in the medical establishment, she adds.

… [T]o comply with the diversity requirement, researchers and pharmaceutical firms will need to list their demographic goals for the participant pool, their rationale for the goals and an explanation of how they intend to meet them. This process looks promising, says Jennifer Miller, a bioethicist at Yale School of Medicine in New Haven, Connecticut, and a co-author of the cancer-therapeutics analysis. ‘This is the first time we’re going to have companies proactively plan enrolment targets and submit them to regulators.’

… Once the FDA implements its diversity requirement, it will be up to companies and researchers to recruit people to take part in their trials. To achieve their goals, they will have to focus on the structural problems that underlie the lack of diversity in clinical-trial populations, Miller says. For instance, they’ll need to consider relocating research sites to places that are more accessible for members of under-represented groups, and should try to build trust in local communities made wary by past instances of exploitative medical research.

By itself, the legislation won’t be sufficient to address long-standing disparities in clinical-trial enrolment, Hwang says. But Knight says: “It’s fantastic the FDA is taking the approach.” It could even inspire countries such as the United Kingdom to introduce similar requirements, she adds. “Having these kinds of levers is the way to make researchers think about diversity.”

 

Clinical. trial participant diversification has been discussed for many years. However, the Biden administration is moving forward with such diversity as part of its overall health equity effort.

For example, in 2022, the U.S. Department of Health and Human Services released a health equity action plan, https://www.hhs.gov/sites/default/files/ hhs-equity-action-plan.pdf.

According to the equity action plan:

“[Biden administration] Executive Order 13985 calls on agencies to advance equity through identifying and addressing barriers to equal opportunity that underserved communities may face due to government policies and programs. This Equity Action Plan focuses on a main tenet of EO 13985, that advancing equity must be a central component of the decision-making framework that all agency functions are routed through. Using the definition of equity presented in EO 13985, this plan takes the perspective that it is incumbent on HHS to move urgently to assess and change policies, programs and processes that the Department administers to concretely advance equity and that for these efforts to last, HHS must simultaneously shift the culture, resources, and approaches available to HHS staff to institutionalize and sustain a focus on equity over time.”

In February 2023, HHS’s Centers for Medicare and Medicaid Services (CMS) released its CMS Framework for Health Equity, https://www.cms.gov/about-cms/agency-information/omh/health-equity-programs/cms-framework-for-health-equity. The framework is intended to advance health equity, expand coverage, and improve health outcomes for the more than 170 million individuals supported by CMS programs.

Indeed, according to CMS:

“CMS is the largest provider of health insurance in the United States, responsible for ensuring that more than 170 million individuals supported by CMS programs (i.e., Medicare, Medicaid, Children’s Health Insurance Program (CHIP), and the Health Insurance Marketplaces) are able to get the care and health coverage they need and deserve.

Consistent with the Department of Health and Human Services’ Healthy People 2030 Framework, CMS recognizes that addressing health and health care disparities and achieving health equity should underpin efforts to focus attention and drive action on our nation’s top health priorities. CMS defines health equity as the attainment of the highest level of health for all people, where everyone has a fair and just opportunity to attain their optimal health regardless of race, ethnicity, disability, sexual orientation, gender identity, socioeconomic status, geography, preferred language, or other factors that affect access to care and health outcomes.”

All of these plans and regulatory initiatives place a premium on providing linguistically and culturally appropriate communications with people who do not speak English as their primary language. In addition, people who are blind or deaf or have other communication disabilities also need effective communications, which can include sign language interpreters and Braille documents, to participate in newly diversified clinical trials.

The expectation is that such communication will be required by FDA as the clinical trial diversity requirements move forward from concept to federal regulation.

When working with diverse populations in future clinical drug trials, pharmaceutical companies and researchers should heed the following advice from Dr. Luz Maria Garcini, assistant professor in the Department of Psychological Sciences at Rice University:

“Cultural competence improves interpersonal interactions, helps to build trust, conveys respect, reduces biases that may lead to inaccurate diagnoses and treatments, and increases the chances that patients may be more compliant with the medical recommendations given.

[Cultural competency] requires time, self-awareness, and systemic change within our organizations. Also, [it] is an ongoing and continuous process that all of us need to be constantly working on. It requires humility and a willingness to listen and to learn from diverse perspectives, including learning from patients and community members.”

 

© Bruce L. Adelson 2023. All Rights Reserved The material herein is educational and informational only.  No legal advice is intended or conveyed.

Bruce L. Adelson, Esq., is nationally recognized for his compliance expertise.  Mr. Adelson is a former U.S Department of Justice Civil Rights Division Senior Trial Attorney.  Mr. Adelson is a faculty member at the Georgetown University School of Medicine and University of Pittsburgh School of Law where he teaches organizational culture, implicit bias, cultural and civil rights awareness.

Mr. Adelson’s blogs are a Bromberg exclusive.

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