FDA releases new draft guidance to enhance diversity in clinical trials

The US Food and Drug Administration (FDA) has issued draft guidance to increase underrepresented population enrollment in clinical trials, enhancing data relevance and patient representation.

The FDA has issued a draft guidance titled, “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.” This guidance is designed to assist medical product sponsors in developing and submitting diversity action plans for certain clinical studies, aiming to increase the participation of historically underrepresented populations.

Enhancing diversity within clinical studies is a significant step towards ensuring that clinical data more accurately reflects the patients who may potentially use the medical product. As FDA Commissioner Robert M Califf, MD, emphasized,

“Participants in clinical trials should be representative of the patients who will use the medical products.”

He added that, “The agency’s draft guidance is an important step – and one of many ongoing efforts – to address the participation of underrepresented populations in clinical trials to help improve the data we have about patients who will use the medical products if approved.”

The guidance outlines the format and content of diversity action plans, specifying which medical products and clinical studies require such plans. It also details the timing and process for submitting these plans to the FDA, as well as the criteria for evaluating waiver requests.

According to the guidance, diversity action plans must include the sponsor’s rationale and goals for clinical study enrollment, disaggregated by age group, ethnicity, sex, and race of clinically relevant study populations. The plans should also outline how sponsors intend to achieve these goals. The FDA urges sponsors and investigators to consider various dimensions of clinical trial diversity, extending beyond age, ethnicity, sex, and race, to ensure the enrollment of populations that accurately represent the patients likely to be treated if the product is approved. As noted in the draft guidance,

“Some populations in the US are frequently underrepresented in biomedical research, including clinical studies, even when they bear a disproportionate burden for certain conditions or diseases relative to their proportional representation in the general population.”

Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, noted, “Generating data for a broader and more representative population early in the clinical development program is among the FDA’s priorities to bring innovative medical products to the public.” He stressed the importance of sponsors critically and intentionally considering the characteristics of the patient population when designing their clinical studies.

This requirement for diversity action plans stems from new provisions in the Federal Food, Drug, and Cosmetic Act, introduced by the Food and Drug Omnibus Reform Act (FDORA). The provisions apply to phase 3 clinical studies, other pivotal clinical studies of drugs or biological products, and certain device clinical studies, that begin 180 days after the final guidance’s publication. The guidance was led by the FDA Oncology Center of Excellence Project Equity, which recently was elevated to a program, The OCE Equity Program.

This guidance aligns with ongoing White House efforts to ensure clinical trial diversity and underscores the FDA’s long-standing goal of promoting diversity in clinical trials. The FDA encourages sponsors to implement comprehensive diversity strategies across their entire clinical development programs, including early studies, whenever possible.

Comments on the draft guidance should be submitted within 90 days after its publication in the Federal Register, identified with the docket number and the title of the guidance document.