Evolving Endpoint Strategies: Navigating New Therapies and Regulatory Acceptance

SINGLE clinical trial endpoints often fall short in capturing the full scope of patient experience, particularly in complex conditions like sickle cell disease and haematologic malignancies. A European Hematology Association (EHA)-Patient Joint Symposium presented at the EHA2025 Congress, held in Milan, Italy, created space for multistakeholder discussions on topics with policy and regulatory implications. These topics are valuable to, and chosen by, patients. This particular session brought together perspectives from patient advocates, clinicians, regulators, industry, and health technology assessment (HTA) bodies to examine the limitations of single endpoints and explore how integrating patient-reported outcomes (PRO), demographic-specific measures, and multiple sources, including real-world data, can lead to more comprehensive and informative patient-centred evaluation frameworks.