DDW Editor Reece Armstrong examines whether recent FDA plans to boost diversity within clinical trials are enough to tackle the scale of the problem.

In June this year, the US Food and Drug Administration (FDA) issued draft guidance designed to help boost the engagement of underrepresented communities within clinical trials.

The agency’s ‘Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies’, were released in an effort to increase the number of people from historically underrepresented communities enrolling in clinical trials and to assist the FDA in gathering data on the types of patients who may eventually use the therapy being tested.

Having diverse patient groups is essential for ensuring that therapies represent the broad populations of people who use them. For the FDA and pharmaceutical industry, it can also help inform how diseases and treatments vary in their usage and occurrence within different communities.

It is an issue in which historical actions have greatly impacted trust between certain communities and healthcare providers. Last year, DDW spoke to Shantanu Dhamija, VP of Strategy and Innovation at Molecular Devices about the issue of a lack of diversity in clinical trials. Dhamija pointed to the Tuskegee study – a trial where researchers studied the effects of untreated syphilis in African American men without informed consent – as discouraging minority participation in clinical trials.

Recent efforts to improve inclusion within clinical trials haven’t exactly been met with great results. For instance, clinical trial inclusion rates for Black/African American and Hispanic patients have failed to match US demographic levels over the past decade, according to IQVIA’s latest ‘Global Trends in R&D 2024 report.

Across therapy areas the statistics are slightly better, particularly in the US where areas such as endocrinology, neurology, cardiovascular and vaccines saw higher levels of patient inclusion compared to other therapy areas.

Within oncology, 2023 US-only clinical trials saw around 7% of their patients come from black and African American backgrounds, highlighting the work needed to engage more with communities across various therapeutic areas.

This is particularly concerning when you consider how various diseases and the healthcare provisions offered can affect outcomes. For instance, the American Cancer Society notes the healthcare disparities can affect black communities, stating how African Americans have a higher cancer burden and can face greater obstacles to cancer care. The organisation says that black people have the highest death rate and shortest survival of any racial group in for most cancers in the US.

And whilst efforts have been made previously to address diversity and inclusion within clinical trials, they have so far not yielded the kind of results that agencies would hope for. In 2022, the FDA released draft guidance to address underrepresentation in clinical trials. However, IQVIA’s report notes that between April 2022 and April 2023, the FDA found that only 6% of studies contained the required diversity information that would be acceptable with IQVIA’s analysis, despite 87% having completed a diversity and inclusivity plan.

Discussing the FDA’s new plans, Susan Shorter, Senior Quality, Risk and Regulatory Compliance Specialist at Greenphire said:

“The Diversity Action plan is currently focused on Phase III clinical trials and is intended to document the sponsor’s enrollment goals for their clinical trials. It also calls for indicating the rationale for enrollment goals as well as details behind how sponsors intend to meet those goals. For example, for rare disease trials, the FDA recommends using sources such as patient registries to assist in targeting participants.

“Ultimately, the Diversity Action Plan guidance aims to acknowledge and address the inequalities that are present in healthcare, with an interest in improving access to treatment and clinical studies amongst underrepresented populations. Increasing diversity in clinical research is critical as this effort will help to ensure heightened safety and efficacy of treatments and drugs across a broader sample of potential consumers – especially those populations that traditionally lack appropriate representation.”

It will be some time until we’re able to ascertain how much impact this new draft guidance has. It’s another step by the FDA which sees it replace previous guidance released in 2022.

Importantly, the new draft guidance not only requires clinical trial sponsors to submit diversity action plans for their studies, but also recommends that companies begin to develop and implement comprehensive diversity plans for clinical development programmes, including early studies.

Determining how therapies might affect different populations at earlier stages of the development process can both assist with the early detection of risks of a particular molecule, but also potentially encourage greater diversity in later clinical trials.

Shantanu Dhamija explained to DDW how utilising induced pluripotent stem cells (iPSCs) could be used to assuage concerns in clinical trials.

“Beyond the obvious benefits associated with the early detection of risks associated with a drug development programme, in vitro screening strategies that incorporate population differences may encourage greater diversity in clinical trials.

“For example, an Asian male might have concerns about whether or not a drug is safe for him. But if someone can say: “We’ve tested this on a human-relevant cell models and results show no-to-low-risk for Asian people,” this individual may be encouraged to enrol in a clinical trial.

“This work can also help expand eligibility. Often, for example, clinical researchers do not want to enrol pregnant women because they are unclear about developmental toxicity. Now, because iPSCs basically mimic developmental cues, you can start to assess developmental toxicity in iPSC-driven organoid models.”

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