METHODOLOGICAL CHALLENGES ASSOCIATED WITH EARLY ECONOMIC EVALUATION: A CASE OF AN RNA-BASED THERAPY FOR FISTULIZING CROHN’S DISEASE...
Author(s)
HANG SU, BSc, Dyfrig Hughes, PhD.
Bangor University, Bangor, United Kingdom.
Bangor University, Bangor, United Kingdom.
OBJECTIVES: To estimate the price at which an RNA-based therapy that is in pre-clinical development for Crohn’s disease, represents good value to a healthcare payer.
METHODS: A de novo Markov model was constructed to evaluate the value-based price of the RNA therapy compared with upadacitinib from a UK healthcare perspective. The model used monthly cycles representing induction and maintenance phases over a 12-month time horizon. Transition probabilities were derived from published clinical trials and structured expert elicitation by fitting large-period matrices to observed state distributions using constrained numerical optimisation with an additional recurrence rate and new onset constraint, followed by matrix root extraction. To address local optima, 10 random-restart solutions were generated per treatment arm. Transition matrices were weighted inversely proportional to their final trace-fitting error for aggregation. The value-based price was determined from a threshold analysis for zero net monetary benefit. A two-way sensitivity analysis jointly varied drug price and utility gain to pricing boundaries.
RESULTS: In the base-case analysis, RNA therapy (£900/month) was dominated by upadacitinib with higher costs (+£413.74) and fewer QALYs (−0.0102). A value-based price of £836-£847/month achieved a net monetary benefit of zero at a threshold of £25,000/QALY, reducing to £823-£828/month at £50,000/QALY. Two-way sensitivity analysis indicated that at £900/month, the RNA therapy required a utility gain of at least +0.030 across all health states to achieve zero net monetary benefit at £25,000/QALY, and +0.025 at £35,000/QALY.
CONCLUSIONS: Based on structured expert elicitation estimates of benefit, the RNA therapy is predicted to be dominated at a price matching upadacitinib. Equivalence in cost-effectiveness could be achieved with a price reduction, or a gain in utility, potentially achieved with fewer adverse reactions or improved patient convenience.
METHODS: A de novo Markov model was constructed to evaluate the value-based price of the RNA therapy compared with upadacitinib from a UK healthcare perspective. The model used monthly cycles representing induction and maintenance phases over a 12-month time horizon. Transition probabilities were derived from published clinical trials and structured expert elicitation by fitting large-period matrices to observed state distributions using constrained numerical optimisation with an additional recurrence rate and new onset constraint, followed by matrix root extraction. To address local optima, 10 random-restart solutions were generated per treatment arm. Transition matrices were weighted inversely proportional to their final trace-fitting error for aggregation. The value-based price was determined from a threshold analysis for zero net monetary benefit. A two-way sensitivity analysis jointly varied drug price and utility gain to pricing boundaries.
RESULTS: In the base-case analysis, RNA therapy (£900/month) was dominated by upadacitinib with higher costs (+£413.74) and fewer QALYs (−0.0102). A value-based price of £836-£847/month achieved a net monetary benefit of zero at a threshold of £25,000/QALY, reducing to £823-£828/month at £50,000/QALY. Two-way sensitivity analysis indicated that at £900/month, the RNA therapy required a utility gain of at least +0.030 across all health states to achieve zero net monetary benefit at £25,000/QALY, and +0.025 at £35,000/QALY.
CONCLUSIONS: Based on structured expert elicitation estimates of benefit, the RNA therapy is predicted to be dominated at a price matching upadacitinib. Equivalence in cost-effectiveness could be achieved with a price reduction, or a gain in utility, potentially achieved with fewer adverse reactions or improved patient convenience.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
EE47
Topic
Economic Evaluation
Disease
SDC: Gastrointestinal Disorders