By incorporating health economic data collection into trial designs, we aim to provide standardised, evidence-based decision-making tools that help address the ethical and logistical challenges of medicine implementation across the UK.
To optimise rare disease trial development and regulatory science, we will conduct international benchmarking through the UK Rare Disease International Mirror Action Group (RD-IMAG), identifying best practices from global health systems. Additionally, this research area will establish a citizen’s jury, ensuring that patients and the public contribute to early access policy discussions. The work will closely align with trial design (WP3), trial delivery (WP4), and recruitment portal research areasto ensure a seamless translation of research findings into NHS practice.
By the end of this project, we will have developed a Value of Information (VoI) framework to refine trial design, completed policy recommendations for NHS medicine adoption, and trialed a patient-informed risk-benefit trade-off tool in exemplar rare disease studies. This initiative will help create a sustainable, patient-focused pathway for bringing innovative treatments into NHS care, ensuring faster, more equitable access to life-changing therapies.
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).
