ArticleThe patient2015
Challenges in measuring the societal value of orphan drugs: insights from a canadian stated preference survey.
Article in The patient, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
15 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Public preferences for allocating health system resources in Canada: a systematic review.Systematic reviews · 2025Pooled it
- Valuation of Treatments for Rare Diseases: A Systematic Literature Review of Societal Preference Studies.Advances in therapy · 2023Pooled it
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.Orphanet journal of rare diseases · 2016Pooled it
- Societal Preferences for Assessment Pathways of Rare Disease Drugs: A Discrete Choice Experiment.Applied health economics and health policy · 2026Article
- What Drives Public Preference for Rare Drugs Coverage in China? Insights From a Multi-Center Discrete Choice Experiment.Health expectations : an international journal of public participation in health care and health policy · 2026Article
- Views and opinions of the general public about the reimbursement of expensive medicines in the Netherlands.PloS one · 2025Article
- Comparing Preferences for Disease Profiles: A Discrete Choice Experiment from a US Societal Perspective.Applied health economics and health policy · 2024Article
- General public's understanding of rare diseases and their opinions on medical resource allocation in Japan: a cross-sectional study.Orphanet journal of rare diseases · 2023Article
- Comparison of Rare and Common Diseases in the Setting of Healthcare Priorities: Evidence of Social Preferences Based on a Systematic Review.Patient preference and adherence · 2023Review
- Moving beyond the Court of Public Opinion: A Citizens' Jury Exploring the Public's Values around Funding Decisions for Ultra-Orphan Drugs.International journal of environmental research and public health · 2022Article
- Societal preferences for funding orphan drugs in China: An application of the discrete choice experiment method.Frontiers in public health · 2022Review
- Moving Towards Accountability for Reasonableness - A Systematic Exploration of the Features of Legitimate Healthcare Coverage Decision-Making Processes Using Rare Diseases and Regenerative Therapies as a Case Study.International journal of health policy and management · 2019Article
- Communal Sharing and the Provision of Low-Volume High-Cost Health Services: Results of a Survey.PharmacoEconomics - open · 2017Article
- The imperative for patient-centred research to develop better quality services in rare diseases.The patient · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
backgroundExpensive drugs for rare diseases (i.e. orphan drugs) often do not meet traditional cost-effectiveness criteria and thus put further strain on limited healthcare budgets. Failing to provide medically necessary care to patients, however, violates one of the underlying tenets of most public health insurance systems-equity. This has led payers to consider the value that society places on the treatment of rare diseases, given the opportunity cost, when deciding on whether to fund specific treatments.
aimsIn this article we aim to illustrate two factors that make the measurement of societal value in this area particularly difficult: the low level of public awareness of, and engagement with, the orphan-drug issue, and the 'zero-sum' framing commonly used to describe the policy challenge posed by orphan drugs.
methodWe illustrate these challenges using data from an original survey of 2,005 Canadian adults. Respondents completed two tasks in which they were asked to choose between funding the treatment of patients suffering from either rare or common diseases.
resultsRespondents were more likely to display choice aversion and unstable preferences if they had not completed a university degree and when a 'zero-sum' frame was used to introduce the choice sets.
conclusionsThe results suggest that studies in which the stated opportunity costs of funding orphan drugs focus exclusively on reductions in funding for other drugs or treatments may only provide a limited understanding of citizens' policy preferences in the area of rare diseases.
Indexed as
Identifiers
25586645What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.