艾昆纬-实现双赢：通过战略规划设计更具代表性的试验（英）

White PaperAchieving a Win-Win: Designing More Representative Trials Through Strategic Planning RACHAEL FONES, Senior Director, Government & Public Affairs and Strategic Advisor, Representative Clinical Trials ROBERT STOLPER, Managing Principal, Strategy Consulting and Head of Enterprise Transformation Strategy SHAHRZAD SALMASI, PH.D., Associate Epidemiology DirectorLESLIE HENDERSON-WILLIAMS, Director, Clinical Trial Access and Representation MONICA GOINS, Senior Director, Therapeutic Analytics ERIC MARTINUSEN, Medical Writing ManagerTable of contentsIntroduction 3How does prioritizing representativeness benefit my trial? 4Scientific benefits 4Operational benefits 4Regulatory benefits 5Commercial benefits 5How should I approach planning for a representative trial? 6Characterize differences within the patient population 6Generate distribution estimates 7Set strategic goals 7Leverage insights to craft successful operational strategies 7What should I look for in a strategic partner? 8What’s next? 9About IQVIA 9About the authors 10 iqvia.com | 3In the world of clinical trials, the ultimate goal is to execute a well-designed trial that yields high-quality, robust evidence that sufficiently characterizes and demonstrates the therapy’s efficacy and safety on an efficient timeline. En route to this goal, sponsors should not underestimate the importance of planning for representative enrollment, a process that aims to study populations and generate data representative of the real-world patient population. The FDA reinforced the importance of planning for representative enrollment in 2022 and again in 2024 when they issued draft guidance on how to effectively develop and submit a Diversity Action Plan (DAP) for clinical studies. As stated by the FDA in its guidance, “actions to improve representativeness in clinical studies can support more equitable and timely access to medical discoveries and innovations, improve the generalizability of results across the intended patient populations, improve our understanding of the disease and/or medical product under study, and inform the safe and effective use of the medical product for all patients.1” In addition to the understanding and insight provided by achieving representativeness, the FDA-outlined approach for DAPs allows for accelerated enrollment by expanding a trial’s reach and enabling more tailored recruitment. While there is currently uncertainty around whether the FDA’s expectations for submitting DAPs will continue, what remains certain is that understanding the demographic makeup of a program’s intended use population and tailoring operational plans with that knowledge improves a trial’s ability to achieve representative enrollment and generalizable data, which is an enduring regulatory, scientific, medical, and commercial concept. In the following text, we explore the benefits we have seen from work on DAPs and how sponsors should pursue planning for representativeness to gain those ad