title: The legal imperative for treating rare disorders.
abstract: BACKGROUND
  Life-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.
  
  
  METHODS
  Using 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.
  
  
  RESULTS
  We demonstrate that a compelling case can be made that the law demands the treatment of orphan diseases.
  
  
  CONCLUSIONS
  Our 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.
authors.affiliation: Department of Medicine, University of Cambridge, Cambridge CB2 0QQ, UK. hannaihyry@gmail.com.
authors.forename: Hanna I
authors.initials: HI
authors.lastname: Hyry
authors.forename: Jonathan C P
authors.initials: JC
authors.lastname: Roos
authors.forename: Jeremy
authors.initials: J
authors.lastname: Manuel
authors.forename: Timothy M
authors.initials: TM
authors.lastname: Cox
mesh_terms: D005062:European Union; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy*