Addressing the health economic challenges of evaluating the full costs and effects of genome-targeted therapies
This project is part of the Therapeutic Genomics Centre, a multidisciplinary programme of innovative approaches to treating rare genetic disorders and providing an enhanced training experience for DPhil applicants.
Academic Supervisors: Prof. Sarah Wordsworth, Assoc. Prof. Nicky Whiffin
Other Supervisors: Dr. Padraig Dixon
Host Dept: Nuffield Department of Population Health (NDPH), Oxford
A major hurdle for moving genome-targeted therapies such as antisense oligonucleotides (ASO) and CRISPR-editing into routine care is the health technology assessment (HTA) process and demonstrating cost-effectiveness to agencies such as NICE. Few health economic assessments have been carried out in this area, with methodological challenges in evaluating genome-targeted therapies noted. Challenges include deciding which health and non-health outcomes to assess and which measurement instruments to use, dealing with small sample sizes, whether to apply an inflationary factor to quality-adjusted life years (QALYs) or to use a higher cost-effectiveness threshold and considering other forms of economic evaluation, including cost-benefit analysis.
This DPhil project will perform much-needed early health economic analysis, including exploring hospital episode statistics data from Genomics England (GEL) patients to examine how healthcare costs could be avoided or reduced if genome-targeted therapies are used. We will test potential solutions to methodological challenges to prepare the evaluation pipeline for HTA agencies. Finally, stakeholder preferences for using genome-targeted therapies will be elicited to identify barriers influencing therapy uptake.
This DPhil project is important to the Therapeutic Genomics Centre to help ensure that any significant scientific and clinical breakthroughs are not left in an endless cycle of reimbursement rejections due to limitations in the current HTA process.
Studentship code: MRCTGCORE2024003
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For more information on DPhil in Paediatrics: DPhil in Paediatrics | University of Oxford
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