The legal imperative for treating rare disordersReport as inadecuate

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Orphanet Journal of Rare Diseases

, 8:135

First Online: 06 September 2013Received: 25 March 2013Accepted: 12 June 2013


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.

KeywordsLaw Orphan Orphan drug Rare disease Human rights Disability Tort Judicial review Electronic supplementary materialThe online version of this article doi:10.1186-1750-1172-8-135 contains supplementary material, which is available to authorized users.

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Author: Hanna I Hyry - Jonathan CP Roos - Jeremy Manuel - Timothy M Cox


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