Drug pricing for a novel treatment for wet macular degeneration: using incremental cost-effectiveness ratios to ensure societal value.

摘要

Objective: Health economic models can assist policy-makers in determining the value of novel treatments from the viewpoint of society. In this context, value is defined as the benefit of treatment, given its cost. A new treatment for wet age-related macular degeneration (AMD), juxtascleral administration of anecortave acetate, 15 mg for depot suspension (Retaane), is now in a late-phase clinical trial. In a theoretical analysis, we sought to determine the cost at which this treatment might offer economic value to society, using incremental cost-effectiveness ratios (ICERs).Methods: A series of 1-year cost-utility models was created for the investigational treatment and standard treatment (photodynamic therapy [PDT] with verteporfin [Visudyne]). Value to society was defined in terms of theoretical associated ICERs (in US dollars): Dollars 100 000 per quality-adjusted life-year (QALY), Dollars 50 000/QALY, Dollars 20 000/QALY and Dollars 0/QALY, the point of economic indifference. Models were created from the societal perspective and included a patient-derived utility assessment involving regression equations to estimate time trade-off preferences, event probabilities derived from a randomized clinical trial comparing the safety and efficacy of anecortave administration and PDT with verteporfin, decision analysis and relevant costing information.Results: An ICER of Dollars 100 000/QALY would be associated with an anecortave cost of Dollars 3022/vial, an ICER of Dollars 50 000/QALY with an anecortave cost of Dollars 2986/vial and an ICER of Dollars 20 000/QALY with an anecortave cost of Dollars 2964/vial. The point of economic indifference between anecortave administration and standard therapy would occur with an anecortave cost of Dollars 2950/vial.Interpretation: In theory, an anecortave cost of Dollars 2986/vial is associated with an ICER of Dollars 50 000/QALY, the threshold used by many health technology assessment and reimbursement agencies.