ArticlePharmacoEconomics2015
Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.
Article in PharmacoEconomics, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers, 5 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
41 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- The VBHC big picture: an umbrella review.BMC health services research · 2025Pooled it
- Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review.International journal of health policy and management · 2024Pooled it
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.Orphanet journal of rare diseases · 2016Pooled it
- Article
- 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
- Using a lottery to resolve indeterminacy when allocating resources for drugs for rare diseases.Journal of medical ethics · 2026Article
- Assessing the Value Contribution of VyvgartAdvances in therapy · 2026Article
- Development of an multicriteria decision analysis framework for rare disease reimbursement prioritization in Malaysia.International journal of technology assessment in health care · 2024Article
- Unraveling elements of value-based pricing from a pharmaceutical industry's perspective: a scoping review.Frontiers in pharmacology · 2024Article
- The Broader Opportunity Costs in the Broader Cost-Effectiveness Analysis Framework.Applied health economics and health policy · 2023Article
- The Value of New: Consideration of Product Novelty in Health Technology Assessments of Pharmaceuticals.Applied health economics and health policy · 2023Review
- Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019.Frontiers in medicine · 2022Article
- A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study).Orphanet journal of rare diseases · 2021Article
- How to Value Orphan Drugs? A Review of European Value Assessment Frameworks.Frontiers in pharmacology · 2021Review
- Evaluating New Zealanders' Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions.PharmacoEconomics · 2021Article
- Cost-Effectiveness of Cannabidiol Adjunct Therapy versus Usual Care for the Treatment of Seizures in Lennox-Gastaut Syndrome.PharmacoEconomics · 2020Article
- Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019.Orphanet journal of rare diseases · 2020Article
- The implementation of health technology assessment principles in public decisions concerning orphan drugs.European journal of clinical pharmacology · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
backgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.
objectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.
methodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.
resultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or 'opportunity cost'-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.
conclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.
Indexed as
Identifiers
What 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.