Achieving A Win-Win: Designing More Representative Trials Through Strategic Planning

By Rachael Fones, Robert Stolper, Shahrzad Salmasi, Ph.D., Leslie Henderson-Williams, Monica Goins, and Eric Martinusen

Designing clinical trials to reflect the diversity of real-world patient populations leads to stronger scientific validity, better regulatory alignment, faster recruitment, and improved commercial performance. Incorporating representativeness early in trial planning helps ensure that data are generalizable and inclusive of key subgroups.

Effective strategies begin with understanding demographic differences in disease patterns and treatment response, supported by literature reviews and real-world data. These insights allow for realistic diversity targets and guide critical operational decisions such as site selection, eligibility criteria, and tailored outreach to underrepresented groups.

Cross-functional collaboration is key, drawing on expertise from epidemiology, clinical development, regulatory strategy, and data science. Aligning trial design with the demographic realities of the target population enables more thoughtful protocol development and operational execution.

FDA guidance on Diversity Action Plans emphasizes the importance of representative enrollment. While regulatory requirements may evolve, the strategic benefits of inclusion are well established. High-quality, generalizable data contribute to more effective therapies and support favorable regulatory review, market differentiation, and patient trust.

Planning with representativeness in mind creates a win-win: supporting equitable access to clinical trials while increasing the likelihood of success across the development lifecycle.