Addressing the health economic challenges of evaluating the full costs and effects of genome-targeted therapies
2025/59
external supervisor
Dr Padraig Dixon, Nuffield Department of Primary Care Health Sciences
background
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. These technologies could be beneficial in the future for the treatment of immunodeficiencies, retinal blindness, neurodevelopmental disorders, and neurodegenerative disorders.
Few health economic assessments have been carried out in this area, with methodological challenges of evaluating gene therapies noted. Challenges include deciding which health and non-health outcomes to assess and 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 patients to examine how health care 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 on using genome-targeted therapies will be elicited to identify barriers influencing therapy uptake.
This DPhil project is part of a large long-term study for the MRC Centre for Research Excellence in Therapeutic Genomics. The DPhil will hopefully help to 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.
The aims of the project are to:
- review all relevant health economics evaluations of genome-targeted therapies to understand their methods and challenges
- explore whether current evaluation approaches are sufficient for genome-targeted therapies
- undertake analyses of health economic data from Genomics England rare disease patients to highlight where genome-targeted therapies could provide economic value
- undertake survey work to elicit preferences of relevant stakeholders such as patients for the use of genome-targeted therapies.
RESEARCH EXPERIENCE, RESEARCH METHODS AND TRAINING
The student will gain experience in health economics, genomics, rare disease research, population health and analysis of large-scale data. They will develop skills in conducting systematic literature reviews, analytical techniques, research planning, data analysis, and presentation skills. The student will be supported to publish peer-reviewed papers during their DPhil.
FIELD WORK, SECONDMENTS, INDUSTRY PLACEMENTS AND TRAINING
Training in health economics, genomics and scientific writing will be provided as needed. Attendance at seminars, workshops and courses provided by NDPH and the wider University will also be encouraged. The candidate will have the opportunity to present their research work at relevant international/national conferences.
PROSPECTIVE STUDENT
Candidates should have a Master's degree in economics, health economics, genetic/epidemiology, medical statistics or a closely related discipline. Proficiency in conducting analyses with R, Stata and Excel are desirable.