Achieving equitable representation in clinical trials

Leveraging real-world evidence to align with FDA diversity action plan guidance

Clinical trials are evolving to reflect the diverse populations they serve. Recognising this, the FDA’s draft guidance on diversity action plans provides a roadmap for recruiting trial participants who are representative of the patient population as a whole. The draft guidance applies to phase 3 studies (or any other pivotal clinical study) and is expected to take effect in late 2025. Currently there is no similar comparable guidance from the EMA.

The guidance requires sponsors to document their plans to enrol representative samples by age, sex, race and ethnicity with respect to the treated condition. In some cases, the profile of patients suffering from the condition to be treated will be well established and available from published epidemiological studies or reports. But in the absence of published data the required breakdown of patient populations by age, sex race and ethnicity will only be available from primary research: this is where real-world evidence (RWE) comes in.

Real-world evidence

RWE refers to clinical insights derived from real-world data (RWD) – data collected outside of controlled clinical trials, usually for purposes other than research. Handled correctly, these data - from electronic health records (EHRs), claims databases, patient registries and wearable devices for example - can provide a more comprehensive insight into healthcare dynamics in the real world. Incorporating RWE into diversity action plans offers sponsors a way to characterise the patient population and perhaps to understand and address the specific needs and barriers faced by underrepresented groups.

The analysis of health data is, however, a non-trivial task. Access to patient data is tightly controlled and the analysis requires a certified secure environment. There are limits to how the data may be used to protect patient privacy and, in the UK, NHS data may only be used in the public interest. Specialist health analytics teams are required for robust data management and scientifically valid research questions.

With the right data, RWE can be used to fill the gaps left by more conventional epidemiological research, with the advantage of being fast and relatively low-cost. Disease patterns and disparities across age, sex, race and ethnicity can be rapidly highlighted and used to inform the target population for a clinical trial. But the FDA guidance also requires trial planners to consider barriers to trial participation in underrepresented groups. Focused research can help uncover these challenges by identifying logistical challenges (e.g. transportation issues) or cultural factors (e.g. mistrust in medical research). Strategically selecting trial sites and investigators is crucial for improving diversity and RWE can help identify regions with high disease prevalence in underserved populations. For example, claims data might highlight specific geographic areas for trial site placement, helping to build the case for proximity to target populations or decentralisation of trials.

Developing FDA-compliant diversity action plans

LCP Health Analytics is uniquely positioned to guide the development of FDA-compliant diversity action plans, leveraging its multidisciplinary expertise in epidemiology, actuarial science, and health economics. With a proven track record of delivering data-driven solutions, LCP combines real-world evidence generation, advanced analytics, and strategic planning to meet the FDA's guidelines on promoting diversity in healthcare research. Our tailored approach ensures actionable strategies that address health disparities, enhance inclusivity, and align with current and developing regulatory standards, making us the trusted partner for transforming diversity goals into measurable outcomes.

Real-world evidence is revolutionising how clinical trials approach diversity. By integrating RWE into diversity action plans, sponsors not only comply with FDA guidance but also create studies that truly reflect the populations affected by the conditions they aim to treat. This data-driven approach ensures equitable representation, fosters trust in clinical research, and ultimately enhances healthcare outcomes for all. Now more than ever, the strategic use of RWE is essential for shaping the future of inclusive medical research.