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For too long, clinical trials have failed to adequately represent the diverse patient populations that will ultimately take the drugs and treatments being tested. This has created gaps in understanding how different people may respond to therapies based on factors like age, gender, race, and ethnicity. The lack of diversity in clinical research means medicines are not studied in representative populations, which can negatively impact healthcare decision-making and patient outcomes.
Regulatory bodies around the world are now taking steps to ensure clinical trials enroll participants that better reflect the real-world patient populations for the disease being studied. A more holistic, inclusive approach to clinical research is being formalized through new guidance and regulations.
Global Regulations on Clinical Trial Diversity
Regulatory agencies across different regions are introducing new guidance to drive more inclusive and representative enrollment in clinical trials.
Fulfilling a mandate under the Food and Drug Omnibus Reform Act (2022; FDORA) the U.S. Food and Drug Administration (FDA) published its draft guidance – Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies – in June 2024. Importantly, when this guidance is finalized, its requirements will be binding on study sponsors. This is in contrast with most FDA guidance documents that simply provide recommendations.
The FDA will require a Diversity Action Plan (DAP) for Phase III clinical trials, or other pivotal studies as appropriate, and for a drug or biological product. A DAP must specify the sponsor’s rationale and goals for clinical study enrollment, separated by age, ethnicity, sex and race of clinically relevant study populations, and describe how these goals will be met. Sponsors should aim to enroll trial participants who represent the patients that will be treated with the product if it is approved.
Health Canada and the European Medicines Agency (EMA) are also formalizing expectations around clinical trial diversity. Health Canada requires organizations to verify if sex, age, and race data was collected and expects participants to mirror the populations that will use the product. The EU’s new Clinical Trials Regulation emphasizes inclusive enrollment strategies so the study population reflects the patient demographic. Sponsors must justify inclusion/exclusion criteria based on gender and age.
In a similar vein, the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Health Research Authority are developing guidance that will likely require an Inclusion and Diversity Plan (IDP) for all clinical studies, not just Phase III. The draft guidance advises that studies should be reflective of the real world and an IDP should clearly show that sponsors have considered potential underserved groups in their trial design.
Opening the Door to Inclusivity
These regulatory moves are crucial for developing a more comprehensive evidence base that captures how different subgroups respond to therapies. More inclusive trials will generate better population-specific data to support more informed treatment decisions and better healthcare outcomes across diverse patient groups.
However, achieving representative enrollment is a difficult operational challenge. Creative approaches like decentralized or hybrid trials that combine remote and in-clinic elements may be needed to enable participation from individuals who would otherwise face barriers to attending academic research sites.
Still, the new regulatory guidance signals an important philosophical shift – drug development should benefit all people, not just certain populations. After years of suboptimal representation in clinical trials, regulators are now formalizing expectations around diversity across the entire drug development process.
While meeting these new requirements will be operationally complex, the impact could be transformative. Increasing diversity will strengthen clinical trials by making them more reflective of real-world patient populations. And that will ultimately lead to safer, more effective therapies for all people who need them.
To learn how H1’s data insights can help you design more representative clinical trials globally, request a demo.
