ArticleDrugs2012
Orphan drugs for rare diseases: is it time to revisit their special market access status?
Article in Drugs, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers, 3 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
33 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Global insight into rare disease and orphan drug definitions: a systematic literature review.BMJ open · 2025Pooled 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
- Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025Review
- Pharmaceutical governance system for costly drugs through human rights due diligence.BMJ (Clinical research ed.) · 2025Article
- Multi-criteria decision analysis of value evaluation of rare disease medical insurance drugs in China.BMC public health · 2025Article
- Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes.Healthcare (Basel, Switzerland) · 2025Article
- Orphan medical devices: addressing the regulatory and access gaps in the EU and US.Frontiers in public health · 2025Article
- The mechanistic functional landscape of retinitis pigmentosa: a machine learning-driven approach to therapeutic target discovery.Journal of translational medicine · 2024Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Voluntariness or legal obligation? An ethical analysis of two instruments for fairer global access to COVID-19 vaccines.Frontiers in public health · 2023Article
- Review
- Article
- How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.Frontiers in pharmacology · 2022Article
- Analysis of patient access to orphan drugs in Turkey.Orphanet journal of rare diseases · 2021Article
- Research and Management of Rare Diseases in the COVID-19 Pandemic Era: Challenges and Countermeasures.Frontiers in public health · 2021Review
- How to Value Orphan Drugs? A Review of European Value Assessment Frameworks.Frontiers in pharmacology · 2021Review
- Brief Overview of Approaches and Challenges in New Antibiotic Development: A Focus On Drug Repurposing.Frontiers in cellular and infection microbiology · 2021Review
- Unrealized potential of drug repositioning in europe during COVID-19 and beyond: a physcian's perspective.Journal of pharmaceutical policy and practice · 2020Review
- Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.Frontiers in medicine · 2020Article
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
No grant is acknowledged in the PubMed record.
Abstract
Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support market access of orphan drugs. We argue that it is time to revisit the special market access status of orphan drugs. Indeed, evidence suggests that there is no societal preference for treating rare diseases. Although society appears to assign a greater value to severity of disease, this criterion is equally relevant to many common diseases. Furthermore, the criterion of equity in access to treatment, which underpins orphan drug legislation, puts more value on health improvement in rare diseases than in common diseases and implies that population health is not maximized. Finally, incentives for the development, pricing and reimbursement of orphan drugs have created market failures, including monopolistic prices and the artificial creation of rare diseases. We argue that, instead of awarding special market access status to orphan drugs, there is scope to optimize research and development (R&D) of orphan drugs and to control prices of orphan drugs by means of, for example, patent auctions, advance purchase commitments, pay-as-you-go schemes and dose-modification studies. Governments should consider carefully the right incentive strategy for R&D of orphan drugs in rare diseases.
Indexed as
Identifiers
22747423What 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.