573 Effects of policy on enrollment of populations facing multimorbid conditions: A systematic analysis

Objectives/Goals: To identify gaps in policy that influence enrollment trends of patients with multimorbidity in Phase III clinical trials. We aim to propose policy recommendations for increasing use of real-world evidence (RWE) that increases safety and efficacy information for patients with multimorbidities. Methods/Study Population: Conduct a systematic policy analysis on the current regulatory landscape of RWE, referencing the 2020 FDA Guidance “Enhancing the Diversity of Clinical Trial Populations.” Evaluate guidelines using the Department of Health and Human Services’ (HHS) 2016 Regulatory Impact Assessment (RIA) Framework. Utilize the Center for Drug Evaluation and Research’s New Molecular Entity Database to identify novel hypertensive drugs approved after 2006, and assess clinical studies’ alignment with the 2020 Guidance. Review additional policies, FDA guidelines, and ICH documents to establish baseline compliance. Two case studies will evaluate past policy impacts on drug development. Assess costs and benefits of increasing multimorbid patient enrollment to inform a policy framework. Results/Anticipated Results: Anticipated results include all components of the HHS’s RIA and a policy framework informed by the assessment. To identify problems, an analysis of clinical trial exclusion criteria in novel hypertensive drugs will be conducted to show diversity and enrollment gaps in regulatory policy, referencing the FDA’s 2020 Guidance. The RIA’s cost–benefit analysis will highlight costs faced for utilizing RWE and expanding enrollment criteria in Phase III studies. The cost–benefit analysis, RIA, and case studies will inform a policy framework that explains dynamics between stakeholders and outline policies that increase clinical trial representation in ways that are less burdensome to sponsors and patients. Discussion/Significance of Impact: By understanding the barriers to enrolling participants with multimorbid conditions, we can outline incentives to increase diverse trial populations, helping healthcare providers choose more treatments for complex conditions. This research supports policy recommendations to make drugs more representative of conditions the population faces.