The legal imperative for treating rare disorders

Orphanet Journal of Rare Diseases

Timothy M Cox²  Jeremy Manuel¹  Jonathan CP Roos²  Hanna I Hyry² 

[1] European Gaucher Alliance, UK Gauchers Association, 340 West End Lane, London NW6 1LN, UK;Department of Medicine, University of Cambridge, Cambridge CB2 0QQ, UK

关键词: Judicial review;    Tort;    Disability;    Human rights;    Rare disease;    Orphan drug;    Orphan;    Law;   

Others  :  863570
DOI  :  10.1186/1750-1172-8-135

 received in 2013-03-25, accepted in 2013-06-12,  发布年份 2013

【 摘 要 】

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.

【 授权许可】

   
2013 Hyry et al.; licensee BioMed Central Ltd.

【 参考文献 】

• [1]Walsh B: The tier 4 phenomenon: shifting the high cost of drugs to consumers. Washington DC: AARP; 2009. http://assets.aarp.org/rgcenter/health/tierfour.pdf webcite
• [2]Testori Coggi P, Director General of the Directorate-General for Health and Consumers of the European Commission: Orphan Products and Rare Disease Therapies. Brussels: 6th European Conference on Rare Diseases & Orphan Products; 2012.
• [3]European Commission: Competition DG: Pharmaceutical sector inquiry – final report. http://ec.europa.eu/competition/sectors/pharmaceuticals/inquiry/staff_working_paper_part1.pdf webcite
• [4]The Swedish Dental and Pharmaceutical Benefits Agency: Assessment of treatment for Gaucher disease ends. 2012. [(in Swedish)] http://www.tlv.se/lakemedel/omprovning-av-lakemedel/avslutade-omprovningar/omprovning-av-lakemedel-for-gauchers-sjukdom-avslutad/ webcite
• [5]Sheldon T: Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs. BMJ 2012, 345:e5461.
• [6]Department of Health: A new value-based approach to the pricing of branded medicines – Government response to consultation. 2011. http://webarchive.nationalarchives.gov.uk/20130107105354/http://www.dh.gov.uk/en/Consultations/Responsestoconsultations/DH_128226 webcite
• [7]Hawkes N: Why is the press so nasty to NICE? BMJ 2008, 337:a1906.
• [8]Nuffield Council on Bioethics: Forward Look 19–20 May 2011 - Hyper-expensive treatments - background paper. http://www.nuffieldbioethics.org/sites/default/files/files/Hyper_expensive_treatments_background_paper.pdf webcite
• [9]Anand G: Uncertain miracle—a biotech drug extends a life, but at what price? Wall Street Journal 2005.
• [10]Jack A, Hope K: Greek crisis gets under skin of vulnerable. Financial Times 2012.
• [11]European Commission: Rare diseases – what are they?. http://ec.europa.eu/health/rare_diseases/policy/index_en.htm webcite
• [12]Council Recommendation of 8 June 2009 on an action in the field of rare diseases. (2009/C 151/02). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF webcite
• [13]Directive 2011/24/EU of The European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2011:088:0045:0065:EN:PDF webcite
• [14]Roos J, Hyry H, Cox TM: Orphan drug pricing may warrant a competition law investigation. BMJ 2010, 341:c6471.
• [15]NICE to assess high cost drugs for rare conditions. 2012. http://www.nice.org.uk/newsroom/news/NICEToAssessHighCostDrugsForRareConditions.jsp webcite
• [16]UN Convention on the Rights of Persons with Disabilities. http://www.un.org/disabilities/convention/conventionfull.shtml webcite
• [17]UN Convention on the Rights of Persons with Disabilities. [Article 1 and Recital (j)] http://www.un.org/disabilities/convention/conventionfull.shtml webcite
• [18]UK Department for Work and Pensions: UN Convention on the Rights of Disabled People. http://odi.dwp.gov.uk/disabled-people-and-legislation/un-convention-on-the-rights-of-disabled-people.php webcite
• [19]UN Convention on the Rights of Persons with Disabilities. [Article 25] http://www.un.org/disabilities/convention/conventionfull.shtml webcite
• [20]United Nations: Optional Protocol to the Convention on the Rights of Persons with Disabilities. http://www.un.org/disabilities/default.asp?id=311 webcite
• [21]Proposal for a Council Directive on implementing the principle of equal treatment between persons irrespective of religion or belief, disability, age or sexual orientation {SEC(2008) 2180} {SEC(2008) 2181} /* COM/2008/0426 final - CNS 2008/0140 */. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=COM:2008:0426:FIN:EN:HTML webcite
• [22]Explanatory Notes to Equality Act 2010. http://www.legislation.gov.uk/ukpga/2010/15/notes/contents webcite
• [23]Equality Act 2010. http://www.legislation.gov.uk/ukpga/2010/15/contents webcite
• [24]Disability Discrimination Act – Guidance on matters to be taken into account in determining questions relating to the definition of disability. http://www.equalityhumanrights.com/uploaded_files/guidance_on_matters_to_be_taken_into_account_in_determining_questions_relating_to_the_definition_of_disability.pdf webcite
• [25]Equality Act 2010. [s6(1)] http://www.legislation.gov.uk/ukpga/2010/15/contents webcite
• [26]Department of Health: Securing best value for NHS patients. 2012. https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/156122/PCCR-consultation-Final.pdf.pdf webcite
• [27]Eisai Ltd v National Institute for Health and Clinical Excellence. 2007. EWHC 1941
• [28]Ac v West Primary Care Trust. 2010. EWHC 1162
• [29]R (on the application of W) v Birmingham City Council. 2011. EWHC 1147 (Admin)
• [30]R (on the application of Chavda and others) v Harrow London Borough Council. 2007. EWHC 3064 (Admin):para 40 (concerning access by elderly disabled persons to community care)
• [31]Gericke CA, Riesberg A, Busse R: Ethical issues in funding orphan drug research and development. J Med Ethics 2005, 31:164-168.
• [32]R (SB) v Governors of Denbigh High School. 2006. UKHL 15:para 68
• [33]R (on the application of De Almeida) v Royal Borough of Kensington and Chelsea. 2012. EWHC 1082
• [34]Associated Provincial Picture Houses Ltd v Wednesbury Corporation. 1948. 1 KB 223
• [35]Manchester City Council v Pinnock. 2011. UKSC 6:paras 45 to 73
• [36]R (on the application of Rogers) v Swindon NHS Primary Care Trust and another. 2006. EWCA Civ:392
• [37]Keenan v United Kingdom. 2001. 33 E.H.R.R. 913:para 11, applying İlhan v. Turkey [GC], no. 22277/93, § 87, ECHR 2000-VII
• [38]R (on the application of (HA) (Nigeria)) v Secretary of State for the Home Department. 2012. EWHC 979 (Admin). The Court accepted that inter alia absence of “appropriate medical treatment” to alleviate the detainee’s mental illness breached Article 3 (paras 179 and 181)
• [39]R (on the application of D) v Secretary of State for the Home Department. 2012. Unreported
• [40]R (on the application of Booker) v NHS Oldham. 2010. EWHC 2593
• [41]R (on the application of Otley) v Barking and Dagenham NHS Primary Care Trust. 2007. EWHC 1927, especially para 26 which sets out further reasons for granting judicial review
• [42]Regina v North West Lancashire Health Authority (Ex parte A, D and G). 2000. 1 WLR 977
• [43]R (on the application of Oliver Leslie Burke) v the General Medical Council. 2005. EWCA Civ 1003
• [44]Gladwell v Steggall. 1839. 5 Bing NC 733
• [45]Clerk and Lindsell on Torts. 20th edition. [Sweet & Maxwell]
• [46]Roe v Ministry of Health. 1954. 2 QB 66
• [47]Cassidy v Ministry of Health. 1951. 2 KB 343
• [48]Wilsher v Essex AHA. 1987. 1 QB 730 (hospital had only four ventilators and none was made available to a baby who needed one: no negligence in the health authority merely because it did not have more at its disposal)
• [49]Nitecki v Poland. ECHR. Application 65653/01
• [50]HM Revenue & Customs: National and Official Satistics. http://hmrc.gov.uk/stats/income_distribution/3-1table-feb2012.pdf webcite
• [51]Osman v UK. [(Case 87/1997/871/1083) 1999] 1 FLR 193
• [52]McCabe C, Stafinski T, Menon D: Is it time to revisit orphan drug policies? BMJ 2010, 341:c4777.
• [53]United States Congress: Orphan drug reauthorization: hearing before the Subcommittee on Health and the Environment of the Committee on Energy and Commerce, House of Representatives, One Hundred Third Congress, second session on H.R. 4160, a bill to amend the Federal Food, Drug, and Cosmetic Act, the Public Health Service Act, and the Orphan Drug Act to revise the provisions of such acts relating to orphan drugs. Washington; 1994:2.
• [54]US National Organization for Rare Disorders: Social Security Disability Assistance. http://www.rarediseases.org/advocacy/initiatives-updates/ssa-disability webcite
• [55]U.S. Social Security Administration: Disability Planner: What We Mean By Disability. http://www.ssa.gov/dibplan/dqualify4.htm webcite
• [56]R v Cambridge Health Authority, Ex parte B. 1995. 1 WLR 898
• [57]R v North and East Devon Health Authority, ex p Coughlan. 2000. 2 WLR 622
• [58]Dunoyer M, GlaxoSmithKline: Value & Specificity of Rare Diseases Business Model. Brussels: 6th European Conference on Rare Diseases & Orphan Products; 2012.
• [59]NHS Act 2006. [s172]
• [60]National Health Service (Charges for Drugs and Appliances) Regulations. 2000.
• [61]NHS Constitution: appendix page 118.
• [62]Weale A, Clark S: Co-payments in the NHS: an analysis of the normative arguments. Health Economics, Policy and Law 2010, 5:225-246.
• [63]D v United Kingdom. 1997. 24 EHRR 423
• [64]R (on the application of Condliff) v North Staffordshire Primary Care Trust. 2011. EWHC 2011:895 (Admin)