Early Economic Modeling to Estimate Cost-Effectiveness and Value-Based Price of a Gene Therapy for Urofacial Syndrome
Author(s)
Sainan Chang, MSc1, Neil Roberts, PhD2, Adrian S. Woolf, PhD2, Sean P. Gavan, PhD1.
1Manchester Centre for Health Economics, The University of Manchester, Manchester, United Kingdom, 2The University of Manchester, Manchester, United Kingdom.
1Manchester Centre for Health Economics, The University of Manchester, Manchester, United Kingdom, 2The University of Manchester, Manchester, United Kingdom.
OBJECTIVES: Many rare conditions are caused by genetic variants, and gene therapy offers a potential one-time curative treatment. Urofacial syndrome is a rare but devastating disorder featuring an inability to empty the urinary bladder, leading to recurrent urinary infections and kidney failure. It is usually caused by biallelic HPSE2 variants. A recent preclinical gene therapy model shows promise. Understanding its economic viability is essential to support further research and development. This study estimated the value-based price (VBP) of a hypothetical gene therapy for urofacial syndrome, and its cost-effectiveness compared with standard of care from a UK healthcare system perspective.
METHODS: An early economic evaluation used a Markov model to estimate lifetime costs(£ price-year 2024) and quality-adjusted life years (QALYs, EQ-5D-3L UK tariff) for gene therapy versus standard care. Gene therapy was modelled as a one-time curative intervention. VBP was calculated using a £100,000 per QALY threshold, following the NICE Highly Specialised Technologies decision-making criteria. One-way sensitivity analysis identified key drivers of cost-effectiveness. Scenario analyses were performed to examine the impact of varying assumptions around treatment effectiveness on the estimated VBP.
RESULTS: Gene therapy gained a mean lifetime QALYs of 21.26 (19.02-22.57) versus 16.67 (13.63-18.62) for standard care, with an incremental gain of 4.59 QALYs (3.16-7.26). Standard care costs were £336,166 (£248,176-£402,039), while VBP of gene therapy was estimated to be £795,008 (£597,816-£1,076,718). Scenario analyses indicated that under assumptions regarding the duration of treatment effectiveness, the VBP reduced between £262,902 and £750,549. For the base case VBP, probabilistic analysis indicated that the probability of a gene therapy being cost-effective was 42%.
CONCLUSIONS: This early health economic evaluation estimated the cost-effectiveness and value-based price of a gene therapy under development for urofacial syndrome. The sensitivity analyses highlight critical parameters that influence economic viability and identify priority areas for future research to reduce decision uncertainty.
METHODS: An early economic evaluation used a Markov model to estimate lifetime costs(£ price-year 2024) and quality-adjusted life years (QALYs, EQ-5D-3L UK tariff) for gene therapy versus standard care. Gene therapy was modelled as a one-time curative intervention. VBP was calculated using a £100,000 per QALY threshold, following the NICE Highly Specialised Technologies decision-making criteria. One-way sensitivity analysis identified key drivers of cost-effectiveness. Scenario analyses were performed to examine the impact of varying assumptions around treatment effectiveness on the estimated VBP.
RESULTS: Gene therapy gained a mean lifetime QALYs of 21.26 (19.02-22.57) versus 16.67 (13.63-18.62) for standard care, with an incremental gain of 4.59 QALYs (3.16-7.26). Standard care costs were £336,166 (£248,176-£402,039), while VBP of gene therapy was estimated to be £795,008 (£597,816-£1,076,718). Scenario analyses indicated that under assumptions regarding the duration of treatment effectiveness, the VBP reduced between £262,902 and £750,549. For the base case VBP, probabilistic analysis indicated that the probability of a gene therapy being cost-effective was 42%.
CONCLUSIONS: This early health economic evaluation estimated the cost-effectiveness and value-based price of a gene therapy under development for urofacial syndrome. The sensitivity analyses highlight critical parameters that influence economic viability and identify priority areas for future research to reduce decision uncertainty.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
P44
Topic
Economic Evaluation
Disease
Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases, Urinary/Kidney Disorders