Signaling Demand with Cost-Effectiveness Analysis: A Case Study of PCSK9 Inhibitors.

OBJECTIVES: Healthcare decision-makers increasingly account for heterogeneity in cost-effectiveness across patient populations. We aimed to characterize the demand curve implied by this heterogeneity. This framework was applied in an analysis of proprotein convertase subtilisin/kexin type 9 inhibitor (PCSK9-I) therapy for Scottish adults with familial hypercholesterolemia (FH). METHODS: We showed how individual and subgroup level treatment value can be estimated by combining incremental health benefits and non-drug costs with a cost-effectiveness threshold, then annualized to obtain value-based prices. Ordering individuals or subgroups by value-based price generates a demand curve that maps treatment price to the proportion of the population that is cost-effective to treat. The framework was applied to PCSK9-I therapy for Scottish adults with FH. The Scottish CVD Policy Model, a previously published decision-analytic model, estimated quality-adjusted life years and healthcare costs. Uncertainty was explored with probabilistic sensitivity analysis. RESULTS: At the current list price of £4,410 per year, PCSK9-I therapy is not cost-effective for Scottish adults with FH (ICER: £350,000/QALY). A decision-maker should treat approximately 10% of the prospective patient population at an annual price of £612 (95% UI: £438-£888) and 50% of the patient population at a price of £276 (95% UI: £200-£390). CONCLUSIONS: We developed a framework that characterizes the demand curve implied by heterogeneity in cost-effectiveness and demonstrates how treatment recommendations can be used to signal demand to manufacturers. Applied to PCSK9-I therapy, the framework characterized the relationship between treatment price and eligibility and its implications for treatment access, surplus distribution, and value-based pricing.