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Implementing Innovation in NHS

Using health economic data collection to support regulatory approval, we will seek to break down barriers in the NHS around rare disease therapies, whilst working with international partners.

Research lead

Prof Amy Jayne McKnight

As a professor of molecular epidemiology and public health, Prof. McKnight’s research is focused on molecular factors that influence human disease and promote health ageing. She runs the NI rare disease research centre and with colleagues, she has helped develop powerful approaches to data analysis and data integration to identify novel biological pathways for disease, discovering many risk factors and biological networks for rare diseases. As an award-winning researcher, her interests are around translating high quality research into tangible benefits from the NHS, industry and academia collaborations. Her efforts in the All-Ireland Rare disease Interdisciplinary research Network (RAiN), and the RD-IMAG and RD research landscape groups will be critical in the success of the centre

University lead

Victoria Hedley - Newcastle University

Victoria Hedley is a Rare Disease Policy Manager at Newcastle University and has a broad role dealing with many health and research-related topics under the banner of ‘rare diseases’. Her work involves paediatric clinical data standardisation as well as being co-lead for the Newcastle Centre for Rare Disease.

Prof Devin Piepert– Birmingham University

Professor Devin Peipert’s research focuses on use of patient reported outcomes in drug development and to optimise treatment decision-making. Devin has been appointed as a 125th Anniversary Chair at the University of Birmingham, which recognises global talent with an outstanding record of research excellence.

Area Overview

Ensuring that innovative rare disease treatments are effectively integrated into the NHS requires a strong focus on health economics, regulatory decision-making, and equitable access to medicines. This research area will develop new frameworks for evaluating risk-benefit trade-offs, supporting regulatory approvals and NHS adoption of novel therapies.

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. 

Research News

Interested in working with the team?

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.

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