{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T21:56:22Z","timestamp":1793051782806,"version":"4.5.0"},"reference-count":65,"publisher":"Informa UK Limited","issue":"5","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Current Medical Research and Opinion"],"published-print":{"date-parts":[[2009,5,1]]},"DOI":"10.1185\/03007990902892633","type":"journal-article","created":{"date-parts":[[2009,4,14]],"date-time":"2009-04-14T15:15:16Z","timestamp":1239722116000},"page":"1285-1293","source":"Crossref","is-referenced-by-count":47,"title":["Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI"],"prefix":"10.1185","volume":"25","author":[{"given":"M.","family":"Schlander","sequence":"first","affiliation":[{"name":"Institute for Innovation & Valuation in Health Care (InnoVal\u2009HC), Wiesbaden, Germany"},{"name":"University of Heidelberg, Mannheim Institute for Public Health, Social and Preventive Medicine, Mannheim, Germany"},{"name":"University of Applied Economic Sciences Ludwigshafen, Ludwigshafen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Beck","sequence":"additional","affiliation":[{"name":"University of Heidelberg, Mannheim Institute for Public Health, Social and Preventive Medicine, Mannheim, Germany"},{"name":"University of Mainz, Children's Hospital, Mainz, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2009,4,7]]},"reference":[{"key":"CIT0001","doi-asserted-by":"publisher","DOI":"10.1001\/jama.281.3.249"},{"key":"CIT0002","doi-asserted-by":"publisher","DOI":"10.1007\/s004390050641"},{"key":"CIT0003","doi-asserted-by":"publisher","DOI":"10.1007\/s004399900075"},{"key":"CIT0004","doi-asserted-by":"publisher","DOI":"10.1038\/sj.ejhg.5201044"},{"key":"CIT0005","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-005-0112-z"},{"key":"CIT0006","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.30579"},{"key":"CIT0007","unstructured":"Useful information on rare diseases from an EU perspective. Available at http:\/\/ec.europa.eu\/health\/ph_information\/documents\/ev20040705_rd05_en.pdf [Last accessed 31 October 2008]"},{"key":"CIT0008","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60875-2"},{"key":"CIT0009","doi-asserted-by":"crossref","DOI":"10.1093\/oso\/9780198529446.001.0001","volume-title":"Methods for the Economic Evaluation of Health Care Programmes","author":"Drummond M","year":"2005","edition":"3"},{"key":"CIT0010","volume-title":"A Qualitative Study","author":"Schlander M","year":"2007"},{"key":"CIT0011","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2184"},{"key":"CIT0012","doi-asserted-by":"publisher","DOI":"10.1016\/j.cccn.2004.10.009"},{"key":"CIT0013","doi-asserted-by":"publisher","DOI":"10.1016\/S1096-7192(03)00007-6"},{"key":"CIT0014","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2007.00693.x"},{"key":"CIT0015","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(95)92597-X"},{"key":"CIT0016","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.03.018"},{"key":"CIT0017","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2004-1023"},{"key":"CIT0018","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2005.12.014"},{"key":"CIT0019","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.04.001"},{"key":"CIT0020","unstructured":"MPS\u2009VI Clinical Surveillance Program. Available at http:\/\/clinicaltrials.gov\/ct2\/show\/NCT00214773 [Last accessed 31 October 2008]"},{"key":"CIT0021","doi-asserted-by":"publisher","DOI":"10.1042\/BA20070093"},{"key":"CIT0022","doi-asserted-by":"publisher","DOI":"10.1517\/14656566.6.3.489"},{"key":"CIT0023","volume-title":"Presented at the Annual Symposium of the Society for the study of inborn errors of metabolism","author":"Scarpa M"},{"key":"CIT0024","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.08.007"},{"key":"CIT0025","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-006-0490-x"},{"issue":"18","key":"CIT0026","first-page":"1","volume":"43","year":"2000","journal-title":"Official Journal of the European Communities"},{"key":"CIT0027","first-page":"5","volume":"103","journal-title":"Official Journal of the European Communities 2000"},{"key":"CIT0028","unstructured":"COMP. Report to the commission in relation to article 10 of regulation 141\/2000 on orphan medicinal products. Doc Ref. EMEA\/35218\/2005. Available at: http:\/\/www.emea.europa.eu\/pdfs\/human\/comp\/3521805en.pdf [Last accessed 25 July 2008]"},{"key":"CIT0029","unstructured":"Register of designated orphan medicinal products (25\/8\/08). Available at http:\/\/ec.europa.eu\/enterprise\/pharmaceuticals\/register\/orphreg.htm [Last accessed 29 August 2008]"},{"key":"CIT0030","unstructured":"National Commissioning Group for Highly Specialised Services. Commissioning intentions for 2008\/09. Available at: http:\/\/www.ncg.nhs.uk\/documents\/cp_ncg_commissioning_intentions_for_2008-09.pdf [Last accessed 25 July 2008]"},{"key":"CIT0031","unstructured":"Jessop E. National service standards for care of people with LSDs. 2005. Available at: http:\/\/www.ncg.nhs.uk\/documents\/lsd_national_service_standards_for_care_of_people_with_lsds-010805.pdf [Last accessed 25 July 2008]"},{"key":"CIT0032","unstructured":"Wraith JE, Vellodi A, Cleary MA, et al. Guidelines for the investigations and management of mucopolysaccharidosis type VI. 2006. Available at: http:\/\/www.ncg.nhs.uk\/documents\/lsd_guidelines_for_mucopolysaccharidosis_type_IV-210306.pdf [Last accessed 25 July 2008]"},{"key":"CIT0033","unstructured":"Sozialgesetzbuch V, \u00a7 73d. Available at: http:\/\/www.gesetze-im-internet.de\/sgb_5\/__73d.html [Last accessed 9 January 2009]"},{"key":"CIT0034","doi-asserted-by":"publisher","DOI":"10.1017\/S0266462307051550"},{"key":"CIT0035","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2125.2006.02579.x"},{"key":"CIT0036","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.314.7081.667"},{"key":"CIT0037","doi-asserted-by":"publisher","DOI":"10.1023\/A:1011387524579"},{"key":"CIT0038","doi-asserted-by":"publisher","DOI":"10.1093\/qjmed\/hci128"},{"key":"CIT0039","doi-asserted-by":"publisher","DOI":"10.1136\/jme.2007.021683"},{"key":"CIT0040","doi-asserted-by":"publisher","DOI":"10.1016\/0168-8510(93)90042-N"},{"key":"CIT0041","first-page":"2218","volume":"265","author":"Hadorn DC","year":"1991","journal-title":"Cost-effectiveness meets the rule of rescue. JAMA"},{"key":"CIT0042","doi-asserted-by":"publisher","DOI":"10.1016\/S0277-9536(02)00244-7"},{"key":"CIT0043","doi-asserted-by":"publisher","DOI":"10.1002\/hec.924"},{"key":"CIT0044","doi-asserted-by":"publisher","DOI":"10.1136\/jme.2003.007138"},{"key":"CIT0045","doi-asserted-by":"publisher","DOI":"10.1016\/j.socscimed.2004.04.034"},{"key":"CIT0046","unstructured":"Richardson J, Iezzi A, Sinha K, McKie J. The Relative Social-Willingness to Pay Instrument: Justification and Initial Results. Melbourne, Vic: Monash University, Centre for Health Economics. Research Paper 22, April 2008. Available at: http:\/\/arrow.monash.edu.au\/vital\/access\/manager\/Repository\/monash:7512 [Last accessed 9 January 2009]."},{"key":"CIT0047","doi-asserted-by":"publisher","DOI":"10.1016\/j.socscimed.2005.10.023"},{"key":"CIT0048","doi-asserted-by":"publisher","DOI":"10.2165\/00019053-200624110-00008"},{"key":"CIT0049","unstructured":"Media release of minister of health and ageing, Australia, May2008. Available at: http:\/\/www.health.gov.au\/internet\/budget\/publishing.nsf\/Content\/E87548B32DB8C0E8CA257446002B 6CF8\/$File\/08_health013.pdf [Last accessed 9 January 2009]"},{"key":"CIT0050","unstructured":"Alcimed. Study on orphan drugs. Part I. Paris: Alcimed, 2005. Available at: http:\/\/ec.europa.eu\/enterprise\/pharmaceuticals\/orphanmp\/doc\/pricestudy\/final_final_report_part_1_web.pdf [Last accessed 25 July 2008]"},{"key":"CIT0051","doi-asserted-by":"publisher","DOI":"10.1016\/S0167-6296(02)00126-1"},{"key":"CIT0052","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.331.7523.1016"},{"key":"CIT0053","doi-asserted-by":"publisher","DOI":"10.1136\/jme.2007.021790"},{"key":"CIT0054","doi-asserted-by":"publisher","DOI":"10.1002\/hec.762"},{"key":"CIT0055","doi-asserted-by":"publisher","DOI":"10.1016\/S0168-8510(96)00851-2"},{"key":"CIT0056","doi-asserted-by":"publisher","DOI":"10.1023\/A:1009439917796"},{"key":"CIT0057","doi-asserted-by":"publisher","DOI":"10.4337\/9781845428914.00050"},{"key":"CIT0058","unstructured":"Sen A. Rights as goals. The Austin Lecture, April 6, 1984"},{"key":"CIT0059","author":"Schokkaert E","year":"1992","journal-title":"Justice: Interdisciplinary Perspectives"},{"key":"CIT0060","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.331.7525.1144-c"},{"key":"CIT0061","doi-asserted-by":"publisher","DOI":"10.1017\/CBO9780511609145"},{"key":"CIT0062","doi-asserted-by":"publisher","DOI":"10.2165\/00019053-200826010-00001"},{"key":"CIT0063","doi-asserted-by":"publisher","DOI":"10.1093\/qjmed\/hcl033"},{"key":"CIT0064","unstructured":"Alcimed. Study on orphan drugs. Part II. Paris: Alcimed, 2005. Available at: http:\/\/ec.europa.eu\/enterprise\/pharmaceuticals\/orphanmp\/doc\/pricestudy\/final_final_report_part_2_web.pdf [Last accessed 9 January 2009]"},{"key":"CIT0065","doi-asserted-by":"publisher","DOI":"10.2165\/00019053-200624040-00001"}],"container-title":["Current Medical Research and Opinion"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1185\/03007990902892633","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,3,12]],"date-time":"2024-03-12T16:14:35Z","timestamp":1710260075000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1185\/03007990902892633"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,4,7]]},"references-count":65,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2009,5,1]]}},"alternative-id":["10.1185\/03007990902892633"],"URL":"https:\/\/doi.org\/10.1185\/03007990902892633","relation":{},"ISSN":["0300-7995","1473-4877"],"issn-type":[{"value":"0300-7995","type":"print"},{"value":"1473-4877","type":"electronic"}],"subject":[],"published":{"date-parts":[[2009,4,7]]}}}