Food and Drug Administration’s (FDA’s) Draft Guidance on Enhancing the Diversity of Clinical Trial Populations


The National Health Council (NHC) appreciates the opportunity to provide comments on the Food and Drug Administration’s (FDA’s) draft guidance on Enhancing the Diversity of Clinical Trial Populations. While industry has moved toward increased diversity in clinical trial enrollment, more work needs to be done. We support FDA’s guidance to industry on this important issue. The NHC urges FDA to ensure that its final guidance leads to enrollment plans that are deliberate, guided by patient involvement, and fit for purpose, while avoiding creation of a set of new requirements that could disrupt clinical trial efficiency or delay patient access to promising new treatments.

Founded in 1920, the NHC brings diverse organizations together to forge consensus and drive patient-centered health policy. The NHC provides a united voice for the more than 160 million people in the United States with chronic diseases and disabilities and their family caregivers. Made up of more than 125 diverse national health-related organizations and businesses, the NHC’s core membership includes the nation’s leading patient advocacy organizations, which control its governance and policy-making process. Other members include professional and membership associations; nonprofit organizations with an interest in health; and representatives from the pharmaceutical, generic drug, health insurance, device, and biotechnology industries.

The NHC strongly supports any efforts to ensure that individuals with chronic diseases and disabilities have the opportunity to participate in clinical trials to facilitate the availability of new and innovative treatment options. We agree with FDA’s statement that “failure to include complex participants in a development program may lead to a failure to discover important safety information about use of the investigational drug in patients who will take the drug after approval.” Similarly, for many individuals, the ability to take part in the drug-development process is an empowering step that aligns with their health care goals and could allow earlier access to treatments to significantly improve health outcomes.

The NHC has actively engaged with the FDA and other stakeholders over the past several years to help ensure the patient voice is meaningfully represented, including in – but not limited to – the development of clinical-trial protocols. The NHC has worked to build consensus across a range of issues to advance the dialogue on patient engagement, from identifying key priority areas and topics for guidance development to providing feedback and suggestions on a common glossary of terms.
Our comments reflect our continuing commitment to ensuring a range of patient voices is meaningfully represented. We focus on the need for early, meaningful, proactive patient engagement to ensure a balance between the benefits of increased participant diversity and the risk of unduly complicating clinical-trial design or delaying access to treatment improvements. The NHC’s overarching comments are:

  • Clinical trial sponsors should engage patient organizations in the design of trials;
  • Representativeness in patient engagement is key;
  • Clinical-trial sponsor efforts to enroll a diverse set of participants should be condition specific;

The NHC strongly supports guidance provisions focused on reducing the burden of clinical-trial participation;

We appreciate the FDA’s additional considerations for clinical-trial design for rare-disease products; and

This guidance offers clear direction without imposing overly-burdensome requirements on sponsors that could result in unintended consequences.

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