After regulatory approval, clinical trials serve several important purposes, such as demonstrating the value of the medicine to payers, reimbursement agencies, healthcare professionals, and other stakeholders. Patient-generated data, including for example, patient-reported outcomes (PROs) are vital, offering insights directly from patients about their disease and treatment experiences, and the impact of these on their health-related quality of life. Clinical trials provide essential data for economic models required by many reimbursement agencies. Ensuring trials can be used for comparative effectiveness research—comparing new treatments with the existing standard of care—is crucial at the design stage. While statistical methods are advancing, they cannot always overcome challenges posed by trials not designed for this purpose.
A broad range of topics will be considered in relation to clinical trial design, including anticipated stakeholder needs and planning for future value message generation, exploring the patient perspective, and considering future inputs for economic evaluations such as utility estimates and comparative efficacy estimates. Additionally, the need for single-arm trials and approaches to select and generate external control arms will be discussed.
Kati Copley-Merriman will moderate the session, providing insights into trial design with a focus on value message generation, the importance of considering current available evidence, and creating an evidence strategy. Lynda Doward will emphasize the need for patient-centered trial design and data collection, ensuring trials include study endpoints that address key areas of disease and treatment impact from the patient perspective while being less burdensome. Emma Hawe will discuss considerations for ensuring the future feasibility of analyses to generate inputs for cost-effectiveness models, with a focus on relative efficacy estimates, and utility estimates by health state. Shannon Cope will discuss and offer practical considerations based on past submissions of ECAs for reimbursement.
