ArticleJournal of medical ethics2005
Ethical issues in funding orphan drug research and development.
Article in Journal of medical ethics, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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
23 citing papers in PubMed.
- How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases.Orphanet journal of rare diseases · 2025Article
- Enhancing evidence-informed policymaking in medicine and healthcare: stakeholder involvement in the Commons Project for rare diseases in Japan.Research involvement and engagement · 2023Article
- Real-world disparities and ethical considerations with access to CFTR modulator drugs: Mind the gap!Frontiers in pharmacology · 2023Review
- Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?Orphanet journal of rare diseases · 2022Review
- A Call to Action Against Persistent Lack of Transparency in Vaccine Pricing Practices During the COVID-19 Pandemic.Annals of global health · 2022Article
- Rare Diseases and Otorhinolaryngology, Head and Neck Surgery.Laryngo- rhino- otologie · 2021Article
- Evaluating New Zealanders' Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions.PharmacoEconomics · 2021Article
- Ethical, legal, and social issues (ELSI) in rare diseases: a landscape analysis from funders.European journal of human genetics : EJHG · 2020Article
- Ethical Questions Linked to Rare Diseases and Orphan Drugs - A Systematic Review.Risk management and healthcare policy · 2020Article
- 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
- Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA.BMC cancer · 2017Article
- Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?Orphanet journal of rare diseases · 2017Article
- Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation.PharmacoEconomics · 2016Article
- CRISPR/Cas9 and mitochondrial gene replacement therapy: promising techniques and ethical considerations.American journal of stem cells · 2016Review
- Challenges in measuring the societal value of orphan drugs: insights from a canadian stated preference survey.The patient · 2015Article
- Reimbursement of orphan drugs in Belgium: what (else) matters?Orphanet journal of rare diseases · 2014Article
- The legal imperative for treating rare disorders.Orphanet journal of rare diseases · 2013Article
- Rare diseases: Canada's "research orphans".Open medicine : a peer-reviewed, independent, open-access journal · 2012Article
- Should EOAD patients be included in clinical trials?Alzheimer's research & therapy · 2011Article
- Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia.PharmacoEconomics · 2011Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This essay outlines the moral dilemma of funding orphan drug research and development. To date, ethical aspects of priority setting for research funding have not been an issue of discussion in the bioethics debate. Conflicting moral obligations of beneficence and distributive justice appear to demand very different levels of funding for orphan drug research. The two types of orphan disease, rare diseases and tropical diseases, however, present very different ethical challenges to questions about allocation of research funds. The dilemma is analysed considering utilitarian and rights based theories of justice and moral obligations of non-abandonment and a professional obligation to advance medical science. The limitations of standard economic evaluation tools and other priority setting tools used to inform health policy decision makers on research funding decisions are outlined.
Indexed as
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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.