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]

Domenico Moro, Michael Schlander, Harry Telser, Oriol Sola-Morales, Michael David Clark, Andrew Olaye, Charlotte Camp, Mohit Jain, Thomas Butt, Sumeet Bakshi

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Abstract

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.

Original languageEnglish
Pages (from-to)1-18
Number of pages18
JournalExpert Review of Pharmacoeconomics & Outcomes Research
Early online date23 Feb 2022
DOIs
Publication statusE-pub ahead of print - 23 Feb 2022

Keywords

  • 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

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