{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T19:58:59Z","timestamp":1792785539164,"version":"4.5.0"},"reference-count":49,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"AveXis, Inc"}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Journal of Market Access & Health Policy"],"published-print":{"date-parts":[[2019,1,1]]},"DOI":"10.1080\/20016689.2019.1601484","type":"journal-article","created":{"date-parts":[[2019,5,8]],"date-time":"2019-05-08T05:55:11Z","timestamp":1557294911000},"page":"1601484","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":84,"title":["Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients"],"prefix":"10.3390","volume":"7","author":[{"given":"Daniel C.","family":"Malone","sequence":"first","affiliation":[{"name":"College of Pharmacy, University of Arizona, Tucson, AZ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca","family":"Dean","sequence":"additional","affiliation":[{"name":"Precision Xtract, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ramesh","family":"Arjunji","sequence":"additional","affiliation":[{"name":"AveXis, Inc., Bannockburn, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivar","family":"Jensen","sequence":"additional","affiliation":[{"name":"Precision Xtract, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Phil","family":"Cyr","sequence":"additional","affiliation":[{"name":"Precision Xtract, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beckley","family":"Miller","sequence":"additional","affiliation":[{"name":"Precision Xtract, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benit","family":"Maru","sequence":"additional","affiliation":[{"name":"SSI Strategy, Parsippany, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas M.","family":"Sproule","sequence":"additional","affiliation":[{"name":"AveXis, Inc., Bannockburn, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas E.","family":"Feltner","sequence":"additional","affiliation":[{"name":"AveXis, Inc., Bannockburn, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omar","family":"Dabbous","sequence":"additional","affiliation":[{"name":"AveXis, Inc., Bannockburn, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2019,5,8]]},"reference":[{"key":"CIT0001","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24864"},{"key":"CIT0002","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0671-8"},{"key":"CIT0003","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.09.015"},{"key":"CIT0004","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"CIT0005","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"CIT0006","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/7.10.1531"},{"key":"CIT0007","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24497"},{"key":"CIT0008","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0724-z"},{"key":"CIT0009","doi-asserted-by":"publisher","DOI":"10.1586\/14737159.4.1.15"},{"key":"CIT0010","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"CIT0011","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"CIT0012","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"CIT0013","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"CIT0014","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-09-382317"},{"key":"CIT0015","volume-title":"22nd Annual SMA Researcher Meeting","author":"Mendell JR","year":"2018"},{"key":"CIT0016","volume-title":"American Academy of Neurology Annual Meeting","author":"Shell R"},{"key":"CIT0017","unstructured":"Avexis. The first SMA Gene Therapy in Human Trials 2018 [cited August 8, 2018]. Available from: https:\/\/www.avexis.com\/research-and-development."},{"key":"CIT0018","volume-title":"ISPOR 20th Annual European Congress","author":"Zuluaga-Sanchez S","year":"2017"},{"key":"CIT0019","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-016-0461-5"},{"key":"CIT0020","volume-title":"ISPOR 20TH Annual European Congress","author":"Lloyd AJ","year":"2017"},{"key":"CIT0021","volume-title":"ISPOR 20TH Annual European Congress","author":"Thompson R","year":"2017"},{"key":"CIT0022","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2016.04.011"},{"key":"CIT0023","doi-asserted-by":"publisher","DOI":"10.1002\/sim.7448"},{"key":"CIT0024","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-510X(96)00284-5"},{"key":"CIT0025","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25101"},{"key":"CIT0026","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-013-0123-9"},{"key":"CIT0027","doi-asserted-by":"publisher","DOI":"10.1186\/1471-2288-11-139"},{"key":"CIT0028","doi-asserted-by":"publisher","DOI":"10.1186\/1745-6215-8-16"},{"key":"CIT0029","doi-asserted-by":"publisher","DOI":"10.18637\/jss.v070.i08"},{"key":"CIT0030","doi-asserted-by":"crossref","DOI":"10.1093\/oso\/9780198526629.001.0001","volume-title":"Decision modelling for health economic evaluation","author":"Briggs AH","year":"2006"},{"key":"CIT0031","doi-asserted-by":"publisher","DOI":"10.1097\/PEP.0b013e3182351f04"},{"key":"CIT0032","volume-title":"Cure SMA Annual Meeting","author":"Shieh PB","year":"2017"},{"key":"CIT0033","volume-title":"A guide to Spinraza reimbursement: important information to help navigate the access and reimbursement process","author":"Biogen","year":"2016"},{"key":"CIT0034","author":"Garrison LP","journal-title":"J Manag Care Spec Pharm"},{"key":"CIT0035","doi-asserted-by":"publisher","DOI":"10.1586\/ecp.12.73"},{"key":"CIT0036","first-page":"31","author":"Dreitlin B","year":"2018","journal-title":"Value Outcomes Spotlight"},{"key":"CIT0037","doi-asserted-by":"publisher","DOI":"10.2217\/cer-2017-0068"},{"key":"CIT0038","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2013.08.834"},{"key":"CIT0039","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0695-0"},{"key":"CIT0040","unstructured":"New SPINRAZA\u00ae (nusinersen) data presented at annual congress of the world muscle society demonstrate benefits in treating presymptomatic infants with spinal muscular atrophy. Available from: http:\/\/investors.biogen.com\/news-releases\/news-release-details\/new-spinrazar-nusinersen-data-presented-annual-congress-world."},{"key":"CIT0041","unstructured":"FDA. CENTER FOR DRUG EVALUATION AND RESEARCH APPLICATION NUMBER:209531Orig1s000 MEDICAL REVIEW(S). [cited 2019 Mar 25]. Available from: https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531orig1s000medr.pdf"},{"key":"CIT0042","volume-title":"Neuromuscular Disorders of Infancy, Childhood and Adolescence: A clinician\u2018s approach","author":"Darras B","year":"2015","edition":"2"},{"key":"CIT0043","doi-asserted-by":"publisher","DOI":"10.1186\/s12891-015-0457-x"},{"issue":"2","key":"CIT0044","first-page":"186","volume":"25","author":"Kuester MK","year":"2019","journal-title":"J Manag Care Spec Pharm"},{"key":"CIT0045","unstructured":"Usdin S. Biocentury. Focusing on Cures. Available from: https:\/\/www.biocentury.com\/printpdf\/300659."},{"key":"CIT0046","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2012-2278"},{"key":"CIT0047","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-014-0153-y"},{"key":"CIT0048","doi-asserted-by":"publisher","DOI":"10.1111\/j.1460-9592.2009.03055.x"},{"key":"CIT0049","doi-asserted-by":"publisher","DOI":"10.1007\/s41669-018-0093-0"}],"container-title":["Journal of Market Access & Health Policy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/tandfonline.com\/doi\/pdf\/10.1080\/20016689.2019.1601484","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/20016689.2019.1601484","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,17]],"date-time":"2024-07-17T15:04:38Z","timestamp":1721228678000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/search?q=10.1080\/20016689.2019.1601484"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,1,1]]},"references-count":49,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2019,1,1]]}},"alternative-id":["10.1080\/20016689.2019.1601484"],"URL":"https:\/\/doi.org\/10.1080\/20016689.2019.1601484","relation":{},"ISSN":["2001-6689"],"issn-type":[{"value":"2001-6689","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,1,1]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=zjma20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=zjma20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2019-02-21","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-03-25","order":1,"name":"revised","label":"Revised","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-03-27","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-05-08","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}