Search

Drug reimbursement decisions that spark public controversy are potential signals that processes used to reach such decisions do not adequately reflect society's goals. Such controversial decisions appear to be a characteristic of Quality-Adjusted Life Year (QALY)-based Incremental Cost Effectiveness Ratio (ICER)-dominated decision-making systems. QALY-based ICER-heavy systems have several known weaknesses that lead to individual and societal preferences being either ignored or considered in an unsystematic and inconsistent manner.We reprise some of the key inadequacies of QALY-based ICER analyses and suggest that there are other means including multicriteria decision analysis (MCDA) and cost-benefit analysis based on willingness to pay (WTP) measures by which to partially mitigate these weaknesses.For long, the inadequacies of QALY-based ICER-heavy decision-making systems have been rationalized with the answer: 'while the method is a second best, it is the best we currently have.' In light of the equally well-developed and widely utilized alternatives available, this resistance to improve assessment processes should not be accepted by policy makers. Health technology assessment bodies should consider and, with appropriate modifications, adopt these alternatives as they have the potential to result in more comprehensive, systematic, and accountable decision-making.

Conventional cost-effectiveness analysis [CEA] using cost per QALY thresholds may counteract other incentives introduced to foster development of treatments for rare and ultra-rare diseases. Therefore, alternative economic evaluation methods were explored, namely Discrete Choice Experiment Willingness to Pay (DCE-WTP) and Relative Social Willingness to Pay (RS-WTP), to value interventions for an ultra-rare childhood disease, Neuronal Ceroid Lipofuscinosis type 2 (CLN2). Treatment for CLN2 was valued from a citizen’s (‘social’) perspective using DCE-WTP and RS-WTP in a survey of 4,009 United Kingdom [UK] adults. Three attributes (initial quality of life, treatment effect, and life expectancy) were used in both analyses. For DCE-WTP, a cost attribute (marginal income tax increase) was also included. Optimal econometric models were identified. DCE-WTP indicated that UK adults are willing to pay incremental increases through taxation for improvements in CLN2 attributes. RS-WTP identified a willingness to allocate >40% of a pre-assigned healthcare budget to prevent child mortality and approximately 15% for improved health status. Both techniques illustrate substantive social WTP for CLN2 interventions, despite the small number of children benefitting. This highlights a gap between UK citizens’ willingness to spend on rare disease interventions and current funding policies.