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.
LifeArc is a self-funded medical research charity. We take science ideas out of the lab and help turn them into medical breakthroughs that can be life-changing for patients. We have been doing this for more than 25 years and our work has resulted in five licensed medicines, including cancer drug pembrolizumab (Keytruda®), lecanemab for Alzheimer’s (Leqembi), and a diagnostic for antibiotic resistance.
Our teams are experts in drug and diagnostics discovery, technology transfer, and intellectual property. Our work is in translational science – bridging the gap between academic research and clinical development, providing funding, research and expert knowledge, all with a clear and unwavering commitment to having a positive impact on patient lives.
Find out more about our work on www.lifearc.org or follow us on LinkedIn or X.
The opinions and interpretations presented on this website are those of the authors and not LifeArc’s.
Newcastle University
Newcastle upon Tyne
Tyne and Wear
NE1 7RU
United Kingdom
The Centre is funded by LifeArc. LifeArc is a company limited by guarantee (registered in England and Wales under no. 2698321) and a charity (registered in England and Wales under no. 1015243 and in Scotland under no. SC037861).
