Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2]

被引:2
|
作者
Moro, Domenico [1 ,2 ,3 ]
Schlander, Michael [4 ,5 ,6 ]
Telser, Harry [7 ,8 ]
Sola-Morales, Oriol [9 ]
Clark, Michael David [3 ]
Olaye, Andrew [10 ]
Camp, Charlotte [10 ]
Jain, Mohit [10 ]
Butt, Thomas [10 ]
Bakshi, Sumeet [2 ]
机构
[1] Univ Birmingham, Dept Econ, Birmingham, W Midlands, England
[2] Certara Evidence & Access, London, England
[3] Apple Educ Ltd, Birmingham, W Midlands, England
[4] Inst Innovat & Valuat Hlth Care InnoValHC, Wiesbaden, Germany
[5] German Canc Res Ctr, Div Hlth Econ, Heidelberg, Germany
[6] Heidelberg Univ, Heidelberg, Germany
[7] Polynomics, Olten, Switzerland
[8] Univ Lucerne, Ctr Hlth Policy & Econ, Luzern, Switzerland
[9] HiTT, Barcelona, Spain
[10] BioMarin Europe Ltd, London, England
关键词
CLN2; Rare Disease; Relative Social Willingness to Pay; RS-WTP; Discrete Choice Experiment Willingness to Pay; DCE-WTP; Discrete Choice Experiment; DCE; Willingness to Pay; Conjoint Analysis; NEURONAL CEROID-LIPOFUSCINOSES; PERSON TRADE-OFF; ORPHAN DRUGS; ELICITING PREFERENCES; DIFFERENT PERSPECTIVES; PRIORITIZING TREATMENT; ECONOMIC-EVALUATION; CONJOINT-ANALYSIS; COST; SERVICES;
D O I
10.1080/14737167.2022.2014324
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Background 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). Research Design and Methods 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. Results 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. Conclusions 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.
引用
收藏
页码:581 / 598
页数:18
相关论文
empty
未找到相关数据