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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.

BackgroundOral anticoagulant therapy (VKA) is nowadays the mainstay of treatment in primary and secondary stroke prevention in patients with atrial fibrillation. Given the limited risk-benefit ratio of vitamin K antagonists, pharmacological research has been directed towards the development of products that could overcome these limits, new oral anticoagulants were recently introduced: dabigatran, rivaroxaban, apixaban, and edoxaban.AimScope of the present study was to examine patterns of use, effectiveness, safety and mean annual cost per patient of anticoagulant treatment for non-valvular AF in real clinical practice.MethodsA retrospective observational cohort study, by using administrative databases (drugs, hospitalizations, clinical visits, lab tests, population registry), was conducted in the Local Health Unit (LHU) of Treviso, Italy, from January 1, 2012 to December 31, 2016.Results5597 subjects were selected, 2171 of which satisfied all inclusion criteria. In particular 1355 patients were treated with VKA, 577 patients were treated with NOAC, and 239 patients were treated initially with VKA and subsequently switched to NOAC (switch group). NOAC treatment showed to be superior to VKA and this superiority was statistically significant on both end-points: patients in the NOAC group reported less cardiovascular events (9,9%) and less bleeding episodes (5,5%) versus VKA patients (14,6% and 11,4%; pConclusionIn this retrospective real-world study treatment with NOAC showed to be associated with significant reductions of CV events and bleeding events compared to VKA use, albeit at a higher NHS' direct cost per patient/year, mainly due to higher drug therapy cost.

Introduction:It is established that the current cost effectiveness health technology assessment (HTA) paradigm does not appropriately value rare disease technologies. Social willingness-to-pay (SWTP) has been suggested to be higher for rare disease technologies, it's inclusion into existing HTA framework could better reflect social preferences (SP) and enable a more equitable evaluation of rare disease technologies. Our study aims to estimate SP and SWTP for a rare disorder, CLN2 disease.Methods:Relevant attributes for CLN2 disease were developed and validated by literature review and focus groups with patient's primary caregivers. An elicitation survey (discrete choice experiment (DCE) WTP and relative social willingness to pay (RS-WTP)) was developed using the selected attributes and levels of each attribute. The survey instruments and attributes/levels were tested and validated in a pre-pilot survey (n = 103) and a subsequent pilot survey (n = 286) of the United Kingdom (UK) general population. Information about CLN2 disease was provided to respondents and their understanding of the disease was tested including assessment of framing effects. The main survey with the general UK population (n = 4,009) is now complete.Results:Eight hundred and twenty-six people were contacted, of which 286 completed the DCE, 113 abandoned the survey before the DCE and 92 abandoned after the DCE. There were no significant differences in characteristics of respondents completing the DCE and those starting but not completing the DCE. Median survey completion time was 20 minutes with 90 percent of participants completing in a single sitting. Median score for the understanding test was 3 (Min = 0 and Max = 4). Incoherencies in WTP estimates were analyzed enabling us introduce relevant modifications and select the most appropriate attribute levels.Conclusions:DCE and RS-WTP potentially are appropriate methods for assessment of social preferences. The selected attributes/levels for the experiment in CLN2 disease have been validated in this pilot.