women, ensuring adequate sample sizes based on the intended use population and appropriate (novel) clinical endpoints and assessment scales. In cases where the data is inconclusive, trials must include sufficient participants from both sexes to allow for meaningful analysis. Additionally, adaptive trial designs can improve efficiency by enabling real-time adjustments, reducing the patient burden while ensuring diverse populations are adequately studied. –Educate investigators, developers, clinical trial staff and patients to improve research quality and outcomes. Requiring an education plan ensures that investigators, developers and clinical trial staff receive proper training, and that patients and their families are well-informed through accessible formats such as videos rather than dense text. Programmes such as the University of Maryland School of Pharmacy PATIENTS Professors Academy exemplify this approach, enhancing patient comprehension and compliance to promote more effective and inclusive research participation.49 –Ensure access to clinical trials through proactive strategies to recruit and retain women, particularly those from underserved communities. Requiring a recruitment-and-retention plan that addresses barriers – such as childcare, transport and financial security – can significantly improve the participation of women in clinical trials, particularly from low- and middle- income backgrounds. Remote access options and partnerships with community organizations can further build trust and engagement. Additionally, analysing clinical trial dropout rates by sex and underlying causes can provide valuable insights to refine study designs and enhance not only recruitment but also retention of diverse populations in future research. Education and access are essential for meeting ethical standards and for ensuring informed consent but also for making clinical trials more inclusive, thereby advancing medical research. Achieving this requires a multistakeholder approach that brings together investigators, industry leaders and other experts to drive change. However, better study design is what ultimately ensures that research produces meaningful, achievable results. When prior preclinical and clinical studies reveal sex-based differences, clinical trials must be designed to address them, with regulators setting clear requirements for sex-specific analyses. Only once these differences are understood and integrated into clinical research can more effective, personalized treatments be achieved. Embedding sex-based biological differences into clinical trial design is a scientific and ethical imperative – policy must lead the way to make this the norm, not the exception. Victor Dzau, President, National Academy of Medicine Prescription for Change: Policy Recommendations for Women’s Health Research 19

Prescription for Change 2025