ArticleOrphanet journal of rare diseases2013
The legal imperative for treating rare disorders.
Article in Orphanet journal of rare diseases, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Caregiver-Reported Economic Impacts of Pediatric Rare Diseases-A Scoping Review.Healthcare (Basel, Switzerland) · 2024Article
- Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research.Orphanet journal of rare diseases · 2024Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- The economic impact of living with a rare disease for children and their families: a scoping review protocol.HRB open research · 2023Article
- Heterogeneity in a large pedigree with Danon disease: Implications for pathogenesis and management.Molecular genetics and metabolism · 2018Article
- Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?Orphanet journal of rare diseases · 2017Article
- Orphan drugs: expensive yet necessary.QJM : monthly journal of the Association of Physicians · 2016Article
- Compassionate use of orphan drugs.Orphanet journal of rare diseases · 2015Article
- The European Gaucher Alliance: a survey of member patient organisations' activities, healthcare environments and concerns.Orphanet journal of rare diseases · 2014Article
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 at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLife-saving orphan drugs are some of the most expensive medicines. European Union governments aim to accommodate their provision within stretched healthcare budgets but face pressure to reduce funding of such treatments. Patients struggle to retain or gain access to them as their special status is questioned, causing distress and in some cases, fears of premature death. In the UK and EU reimbursement and pricing model of drugs, and orphan drugs in particular, is being re-evaluated.
methodsUsing the United Kingdom as a case study we present, for the first time, legal arguments which compel governments to provide orphan medicinal products. These include (i) disability legislation, (ii) national and organisational constitutions, (iii) judicial review, (iv) tort law and (v) human rights legislation. We then address directly potential objections to our analysis and counter arguments which aim to limit provision of orphan drugs to the intended patient recipients.
resultsWe demonstrate that a compelling case can be made that the law demands the treatment of orphan diseases.
conclusionsOur legal framework will assist doctors and patients in ensuring the continued provision of treatments despite significant economic pressure to reduce funding. These legal avenues will empower stakeholders in drafting funding guidelines throughout the EU. The legal right to treatment extends beyond rare diseases and our analysis may therefore affect allocation of healthcare budgets throughout the EU.
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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.