Estimating the family and wider societal health economic impacts of genomic therapeutics.

About the project

About the Project A major hurdle to integrating genome-targeted therapies, such as antisense oligonucleotides (ASOs) and CRISPR-based therapies, into routine care is the health technology assessment process and the need to demonstrate cost-effectiveness to agencies such as NICE. In many countries, reimbursement decisions have traditionally focused on narrow measures of economic benefit, with limited consideration of the wider societal impacts of health interventions. This includes the effects of illness on informal or unpaid caregivers and other family members, known as “family spillover effects.” Evidence of the impact of genetic diseases on families and caregivers has grown substantially, with research demonstrating both negative and potentially positive effects on siblings for example. However, the explicit incorporation of these effects into economic evaluations is particularly limited, with existing research largely focused on the cost-effectiveness of cascade screening for genetic testing. This DPhil will investigate how the impacts of treatments for rare genetic diseases on siblings and other family members can be measured and incorporated into health economic evaluations of genomic therapeutics. It will examine how accounting for family spillover effects could influence reimbursement decisions, ultimately supporting a broader and more comprehensive assessment of the value of innovative genomic therapies. Project Objectives To identify and characterise the family spillover effects of genomic therapeutics, including the health, social, and economic impacts experienced by informal caregivers and family members. To explore the experiences and perspectives of family members affected by genomic therapies through qualitative research, with particular attention to the impacts on caregivers and siblings. To quantify family spillover effects associated with genomic therapeutics using quantitative surveys to generate robust data on health, wellbeing, caregiving, productivity, and other societal consequences. To incorporate family spillover effects into health economic models and assess how their inclusion changes estimates of the costs, benefits, and overall value of genomic therapeutics. To assess the implications of incorporating family spillover effects into health technology assessment and reimbursement decisions, providing evidence to support more comprehensive and equitable approaches to evaluating genomic therapies. Research Methodologies Reviews Interviews Surveys Health economic decision modelling Cost-benefit analysis Potential Project Impact This project would provide much needed information to help the MRC Core Project and reimbursement agencies in the UK (NICE) and internationally to understand the impact of societal costs and benefits on the health economic value of genome-targeted therapies. Proposed Project Timelines Year 1: Review the health outcome literature on the impact of genomic treatments on family members AND identify and interview relevant groups of family members, especially siblings to understand the likely impact of genomic therapeutics; Year 2: Undertake survey work input to understand the preferences and barriers to the uptake of genome-targeted therapies of a range of stakeholders; Year 3: Develop several health economic decision models to evaluate the health system and wider societal costs of genomic therapeutics being developed in the MRC CoRE; Year 4: Provide a framework for decision makers, including reimbursement agencies to assess evidence provided from health economic models of the societal impacts of genomic therapeutics. Potential Internship/Exceptional Training Opportunities This DPhil would benefit hugely from having training on PPIE methods and being integrated with the PPIE team of the CoRE under Carols leadership. Opportunities for student participation in PPIE This DPhil is very closely linked with understanding the importance of PPIE in Genomic Therapeutics. MRC CoRE-TG Scholarships MRC CoRE-TG Scholarships Applicants to MRC CoRE-TG projects may be nominated for a CoRE-TG DPhil scholarship, which involves a second interview following the departmental interview. Due to UKRI limits on international student recruitment, only UK home students are eligible for these MRC CoRE-TG DPhil scholarships. For information on home student eligibility, please refer to UKRI training grants: standard terms and conditions of training grant sections TGC 5.2.4 & TGC 5.2.5. The MRC CoRE-TG DPhil scholarship provides funding for: Course fees for the duration of fee liability. A living stipend at the UKRI rate, paid for four years. A £20,000 Research Training Support Grant (RTSG) to support research and training costs. A £1,200 travel allowance to support research-related travel and development opportunities. The stipend rate is reviewed annually by UKRI/MRC. Overseas applicants are still welcome to apply for MRC CoRE-TG projects. They may be considered for other funding opportunities available through the relevant department and may also apply for external scholarship funding independently to support their DPhil. Ready to join us? Applications are competitive and will be assessed according to the admissions requirements of the relevant host department or university. For questions about the MRC CoRE in Therapeutic Genomics programme , please contact mrccoretg@paediatrics.ox.ac.uk Scholarship Code: CoRE-TG 2026-007

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