ArticleOrphanet journal of rare diseases2025
Using a discrete choice experiments to explore societal preferences for valuing new drugs for rare diseases.
Article in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Transplantation as disease modifying therapy in the era of gene therapy medicinal products - health policy considerations.Orphanet journal of rare diseases · 2026Review
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Authors and funding
5 authors.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundRare diseases affect few people, but collectively they affect a substantial proportion of the population. Limited treatment options and the additional challenges of securing public funding make reimbursement decision making particularly complex, mainly due to the inherent evidence uncertainty, lack of understanding of these diseases and the greater impact on non-health outcomes. This study explored societal preferences regarding which factors should be valued when considering public subsidy of drugs to treat rare diseases.
methodsA discrete choice experiment was developed, and respondents were asked to assume the role of a Government advisor and decide on funding between 2 drugs to treat a rare disease. Attributes were identified from the literature and focus groups, including uncertainty of the evidence, unmet need, magnitude of the clinical benefit, magnitude in quality of life and total cost to government. A representative sample of Australians (n = 1099) completed the online survey. Data were analysed using mixed logit regression and latent class models to examine heterogeneity. Willingness to pay was also estimated.
resultsIn general, respondents had a greater preference for drugs that increase survival, where there was greater confidence in the effectiveness of the new drug and which increased patients' capacity to do their usual activities. Preferences were not homogenous, the latent class analysis identified three groups: Class 3 (58%) demonstrated a strong preference for improvements in survival; Class 2 (21%) showed a strong preference for confidence in the evidence; and Class 1 (21%) positively valued increased government expenditure.
conclusionThese results are consistent with previous studies that used different methodologies in showing a preference for drugs with improved survival and quality of life. However, addressing a societal preference for greater confidence in the evidence - reducing evidential uncertainty - represents a methodological and policy challenge for the evaluation of drugs in rare diseases. CLINICAL TRIAL NUMBER: Not applicable.
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