{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,13]],"date-time":"2026-07-13T13:00:41Z","timestamp":1783947641432,"version":"3.55.0"},"reference-count":17,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2021,5,4]],"date-time":"2021-05-04T00:00:00Z","timestamp":1620086400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2021,5,4]],"date-time":"2021-05-04T00:00:00Z","timestamp":1620086400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["BMC Med Ethics"],"published-print":{"date-parts":[[2021,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background<\/jats:title>\n                <jats:p>Nusinersen is one of an increasing number of new, expensive orphan drugs to receive authorization. These drugs strain public healthcare budgets and challenge principles for resource allocation. Nusinersen was introduced in the Norwegian public healthcare system in 2018. A national expert group consisting of physicians was formed to oversee the introduction and continuation of treatment in light of specific start and stop criteria.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>We have studied experiences within the expert group with a special emphasis on their application of the start and stop criteria, rationing of treatment, and experienced moral dilemmas. A research interview with six members of the national expert group was performed, then analysed with manifest content analysis. The analysis was supplemented with publically available sources on priority setting and the process leading up to the introduction of nusinersen and the establishment of the expert group.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Sixty-six patients have received treatment within the first 25\u00a0months since the national expert group\u2019s establishment. Treatment has not been discontinued for any patient. No patients under 18\u00a0years of age have been denied treatment, as those who were referred at this age were all deemed to fulfill the start criteria. The expert group has, however, increased geographical treatment equity and facilitated important cooperation at the national level. Furthermore, it has enhanced open and critical discussions of both medical issues and new ethical dilemmas.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>Although facilitating equal access to treatment for SMA patients, the national expert group has not discontinued treatment for any patient. It is suggested that in order for clinicians to be able to ration care for individual patients, they require both adequate support and sufficient formal authority. Start and stop criteria need to be re-evaluated as more knowledge and experience are gained regarding the treatment.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s12910-021-00623-5","type":"journal-article","created":{"date-parts":[[2021,5,4]],"date-time":"2021-05-04T02:03:03Z","timestamp":1620093783000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Priority setting at the clinical level: the case of nusinersen and the Norwegian national expert group"],"prefix":"10.1186","volume":"22","author":[{"given":"Morten","family":"Magelssen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Magnhild","family":"Rasmussen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean","family":"Wallace","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reidun","family":"F\u00f8rde","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,5,4]]},"reference":[{"issue":"10149","key":"623_CR1","doi-asserted-by":"publisher","first-page":"791","DOI":"10.1016\/S0140-6736(18)31069-9","volume":"392","author":"L Luzzatto","year":"2018","unstructured":"Luzzatto L, Hyry HI, Schieppati A, Costa E, Simoens S, Schaefer F, et al. Outrageous prices of orphan drugs: a call for collaboration. The Lancet. 2018;392(10149):791\u20134.","journal-title":"The Lancet"},{"issue":"4","key":"623_CR2","doi-asserted-by":"publisher","first-page":"112","DOI":"10.1177\/1477750916657664","volume":"11","author":"M Magelssen","year":"2016","unstructured":"Magelssen M, Nortvedt P, Solbakk JH. Rationing at the bedside: Immoral or unavoidable? Clin Ethics. 2016;11(4):112\u201321.","journal-title":"Clin Ethics"},{"key":"623_CR3","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1093\/acprof:oso\/9780199989447.003.0015","volume-title":"Fair resource allocation and rationing at the bedside","author":"VB Wyller","year":"2014","unstructured":"Wyller VB. Give to the doctor what is due to the doctor! why \u201cfair rationing at the bedside\u201d is impossible. In: Danis M, Hurst S, Fleck L, F\u00f8rde R, Slowther A, editors. Fair resource allocation and rationing at the bedside. New York: Oxford University Press; 2014. p. 253\u201362."},{"issue":"8","key":"623_CR4","doi-asserted-by":"publisher","first-page":"488","DOI":"10.1136\/jme.2007.022285","volume":"35","author":"K B\u00e6r\u00f8e","year":"2009","unstructured":"B\u00e6r\u00f8e K. Priority-setting in healthcare: a framework for reasonable clinical judgements. J Med Ethics. 2009;35(8):488\u201396.","journal-title":"J Med Ethics"},{"issue":"9","key":"623_CR5","doi-asserted-by":"publisher","first-page":"2629","DOI":"10.1007\/s11606-020-05805-2","volume":"35","author":"JD Chambers","year":"2020","unstructured":"Chambers JD, Silver MC, Berklein FC, Cohen JT, Neumann PJ. Orphan drugs offer larger health gains but less favorable cost-effectiveness than non-orphan drugs. J Gen Intern Med. 2020;35(9):2629\u201336.","journal-title":"J Gen Intern Med"},{"issue":"1","key":"623_CR6","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1017\/S0266462307051550","volume":"23","author":"MF Drummond","year":"2007","unstructured":"Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007;23(1):36.","journal-title":"Int J Technol Assess Health Care"},{"issue":"6","key":"623_CR7","doi-asserted-by":"publisher","first-page":"845","DOI":"10.1007\/s40273-019-00769-6","volume":"37","author":"S Zuluaga-Sanchez","year":"2019","unstructured":"Zuluaga-Sanchez S, Teynor M, Knight C, Thompson R, Lundqvist T, Ekelund M, et al. Cost effectiveness of nusinersen in the treatment of patients with infantile-onset and later-onset spinal muscular atrophy in Sweden. Pharmacoeconomics. 2019;37(6):845\u201365.","journal-title":"Pharmacoeconomics"},{"key":"623_CR8","unstructured":"OECD Data. Health spending. https:\/\/data.oecd.org\/healthres\/health-spending.htm. Accessed 5 Mar 2021."},{"key":"623_CR9","unstructured":"Helse- og omsorgsdepartementet. Meld. St. 34 (2015\u20132016)\u00a0Verdier i pasientens helsetjeneste \u2013 Melding om prioritering. Oslo; 2016."},{"issue":"3\u20134","key":"623_CR10","doi-asserted-by":"publisher","first-page":"279","DOI":"10.18261\/issn.1504-3053-2018-03-04-08","volume":"35","author":"M Magelssen","year":"2018","unstructured":"Magelssen M, F\u00f8rde R. Prioritering i helsetjenesten \u2013 ni dilemmaer i kj\u00f8lvannet av Spinraza-saken. Nytt norsk tidsskrift. 2018;35(3\u20134):279\u201388.","journal-title":"Nytt norsk tidsskrift"},{"issue":"18","key":"623_CR11","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","volume":"377","author":"RS Finkel","year":"2017","unstructured":"Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723\u201332. https:\/\/doi.org\/10.1056\/NEJMoa1702752.","journal-title":"N Engl J Med"},{"issue":"7","key":"623_CR12","doi-asserted-by":"publisher","first-page":"625","DOI":"10.1056\/NEJMoa1710504","volume":"378","author":"E Mercuri","year":"2018","unstructured":"Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378(7):625\u201335.","journal-title":"N Engl J Med"},{"key":"623_CR13","unstructured":"Nye Metoder. Kriterier for behandling med nusinersen (Spinraza\u00ae) for barn (0\u201318 \u00e5r). 2018. https:\/\/nyemetoder.no\/Documents\/Beslutninger\/Kriterier%20for%20behandling%20med%20nusinersen%20Spinraza%20for%20barn%200-18%20\u00e5r.pdf. Accessed 5 Mar 2021."},{"issue":"2","key":"623_CR14","doi-asserted-by":"publisher","first-page":"105","DOI":"10.1016\/j.nedt.2003.10.001","volume":"24","author":"UH Graneheim","year":"2004","unstructured":"Graneheim UH, Lundman B. Qualitative content analysis in nursing research: concepts, procedures and measures to achieve trustworthiness. Nurse Educ Today. 2004;24(2):105\u201312.","journal-title":"Nurse Educ Today"},{"key":"623_CR15","doi-asserted-by":"publisher","first-page":"8","DOI":"10.1016\/j.npls.2016.01.001","volume":"2","author":"M Bengtsson","year":"2016","unstructured":"Bengtsson M. How to plan and perform a qualitative study using content analysis. NursingPlus Open. 2016;2:8\u201314.","journal-title":"NursingPlus Open"},{"issue":"11","key":"623_CR16","doi-asserted-by":"publisher","first-page":"842","DOI":"10.1016\/j.nmd.2019.09.007","volume":"29","author":"DC De Vivo","year":"2019","unstructured":"De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842\u201356.","journal-title":"Neuromuscul Disord"},{"issue":"9","key":"623_CR17","doi-asserted-by":"publisher","first-page":"756","DOI":"10.1016\/j.nmd.2020.07.005","volume":"30","author":"G Coratti","year":"2020","unstructured":"Coratti G, Pera MC, Lucibello S, Montes J, Pasternak A, Mayhew A, et al. Age and baseline values predict 12 and 24-month functional changes in type 2 SMA. Neuromuscul Disord. 2020;30(9):756\u201364.","journal-title":"Neuromuscul Disord"}],"container-title":["BMC Medical Ethics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s12910-021-00623-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s12910-021-00623-5\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s12910-021-00623-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,5,4]],"date-time":"2021-05-04T02:04:45Z","timestamp":1620093885000},"score":1,"resource":{"primary":{"URL":"https:\/\/bmcmedethics.biomedcentral.com\/articles\/10.1186\/s12910-021-00623-5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,5,4]]},"references-count":17,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2021,12]]}},"alternative-id":["623"],"URL":"https:\/\/doi.org\/10.1186\/s12910-021-00623-5","relation":{},"ISSN":["1472-6939"],"issn-type":[{"value":"1472-6939","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,5,4]]},"assertion":[{"value":"9 March 2021","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 April 2021","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 May 2021","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The study was evaluated and approved by the Data Protection Official at the Norwegian Centre for Research Data (ref. 320544); according to the Norwegian system, no further research ethics approval was required. Participants were informed about the project in writing and orally. Informed consent was obtained from all participants. The data do not contain information about individual patients. The research was carried out in\u00a0accordance\u00a0with all relevant guidelines and regulations.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"MM has received fees for lectures from Sanofi-Aventis and Roche. MR and SW are members of the national expert group on Spinraza treatment in spinal muscular atrophy (SMA) and work clinically with SMA patients. RF has no conflicts of interests.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"54"}}