By working closely with patients, advocacy groups, and stakeholders, we will establish consensus-based guidance to standardise PRO collection and ensure its acceptability, accessibility, and impact on trial design.
Through multistakeholder workshops, literature reviews, focus groups, and Delphi surveys, we will identify barriers and facilitators to PRO collection in rare disease trials. These insights will support the development of a PRO design support service, providing rare disease researchers with the tools and guidance needed to effectively implement patient-focused outcome measures in their studies.
By embedding PRO methodologies into trial design, this initiative will help ensure that clinical research captures what truly matters to patients, leading to more meaningful trial outcomes, improved patient engagement, and enhanced treatment decision-making.
We are always looking for talented researchers, clinicians, and industry partners to join our efforts in accelerating rare disease trials. Whether you’re interested in research collaboration, clinical trial design, or patient engagement, we’d love to hear from you.
