Related Experiment Video
Updated: Jun 19, 2026
Measuring the Subjective Value of Risky and Ambiguous Options using Experimental Economics and Functional MRI Methods
Published on: September 19, 2012
Using a discrete choice experiments to explore societal preferences for valuing new drugs for rare diseases
Constanza Vargas1, Stephen Goodall2, Deborah J Street2
1Centre for Health Economics and Evaluation, University of Technology Sydney, Building 20, 100 Broadway, Chippendale, Sydney, NSW, 2008, Australia. constanza.vargas@uts.edu.au.
Background:
Rare 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.
Methods:
A 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.
Results:
In 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.
Conclusion:
These 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.
Related Concept Videos
01:16Crossover Experiments
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
01:26Drug Discovery: Overview
01:02Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
01:12Analysis of Population Pharmacokinetic Data
01:28Structure-Activity Relationships and Drug Design
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence its...
01:15Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions