{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T05:14:13Z","timestamp":1789708453230,"version":"4.0.1"},"reference-count":76,"publisher":"Springer Science and Business Media LLC","issue":"2","license":[{"start":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T00:00:00Z","timestamp":1727395200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T00:00:00Z","timestamp":1727395200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"name":"Siriraj Research Developmental Fund, Faculty of Medicine Siriraj Hospital, Mahidol University","award":["R016532007"],"award-info":[{"award-number":["R016532007"]}]},{"DOI":"10.13039\/501100004156","name":"Mahidol University","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100004156","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Appl Health Econ Health Policy"],"published-print":{"date-parts":[[2025,3]]},"DOI":"10.1007\/s40258-024-00915-y","type":"journal-article","created":{"date-parts":[[2024,9,27]],"date-time":"2024-09-27T17:02:17Z","timestamp":1727456537000},"page":"277-290","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis"],"prefix":"10.1007","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6430-0630","authenticated-orcid":false,"given":"Sarayuth","family":"Khuntha","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3356-7890","authenticated-orcid":false,"given":"Juthamas","family":"Prawjaeng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5130-7545","authenticated-orcid":false,"given":"Kunnatee","family":"Ponragdee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4557-0387","authenticated-orcid":false,"given":"Oranee","family":"Sanmaneechai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5311-7657","authenticated-orcid":false,"given":"Varalak","family":"Srinonprasert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9584-0293","authenticated-orcid":false,"given":"Pattara","family":"Leelahavarong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,9,27]]},"reference":[{"issue":"5","key":"915_CR1","doi-asserted-by":"publisher","first-page":"1348","DOI":"10.1212\/con.0000000000000918","volume":"26","author":"JR Nance","year":"2020","unstructured":"Nance JR. Spinal muscular atrophy. Continuum (Minneap Minn). 2020;26(5):1348\u201368. https:\/\/doi.org\/10.1212\/con.0000000000000918.","journal-title":"Continuum (Minneap Minn)."},{"issue":"2","key":"915_CR2","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1002\/mus.24497","volume":"51","author":"WD Arnold","year":"2015","unstructured":"Arnold WD, Kassar D, Kissel JT. Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle Nerve. 2015;51(2):157\u201367. https:\/\/doi.org\/10.1002\/mus.24497.","journal-title":"Muscle Nerve"},{"key":"915_CR3","first-page":"117","volume-title":"Neuromuscular disorders of infancy, childhood, and adolescence","author":"BT Darras","year":"2015","unstructured":"Darras BT, Markowitz JA, Monani UR, De Vivo DC. Chapter 8\u2014spinal muscular atrophies. In: Darras BT, Jones HR, Ryan MM, De Vivo DC, editors. Neuromuscular disorders of infancy, childhood, and adolescence. 2nd ed. San Diego: Academic Press; 2015. p. 117\u201345.","edition":"2"},{"issue":"8","key":"915_CR4","doi-asserted-by":"publisher","first-page":"1027","DOI":"10.1177\/0883073807305788","volume":"22","author":"CH Wang","year":"2007","unstructured":"Wang CH, Finkel RS, Bertini ES, Schroth M, Simonds A, Wong B, et al. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027\u201349. https:\/\/doi.org\/10.1177\/0883073807305788.","journal-title":"J Child Neurol"},{"issue":"5","key":"915_CR5","doi-asserted-by":"publisher","first-page":"518","DOI":"10.1001\/archneur.1995.00540290108025","volume":"52","author":"K Zerres","year":"1995","unstructured":"Zerres K, Rudnik-Sch\u00f6neborn S. Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications. Arch Neurol. 1995;52(5):518\u201323. https:\/\/doi.org\/10.1001\/archneur.1995.00540290108025.","journal-title":"Arch Neurol"},{"issue":"11","key":"915_CR6","doi-asserted-by":"publisher","first-page":"965","DOI":"10.3345\/kjp.2010.53.11.965","volume":"53","author":"HB Park","year":"2010","unstructured":"Park HB, Lee SM, Lee JS, Park MS, Park KI, Namgung R, et al. Survival analysis of spinal muscular atrophy type I. Korean J Pediatr. 2010;53(11):965\u201370. https:\/\/doi.org\/10.3345\/kjp.2010.53.11.965.","journal-title":"Korean J Pediatr"},{"issue":"9","key":"915_CR7","doi-asserted-by":"publisher","first-page":"810","DOI":"10.1212\/wnl.0000000000000741","volume":"83","author":"RS Finkel","year":"2014","unstructured":"Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810\u20137. https:\/\/doi.org\/10.1212\/wnl.0000000000000741.","journal-title":"Neurology"},{"issue":"1","key":"915_CR8","doi-asserted-by":"publisher","first-page":"67","DOI":"10.1016\/s0022-510x(96)00284-5","volume":"146","author":"K Zerres","year":"1997","unstructured":"Zerres K, Rudnik-Sch\u00f6neborn S, Forrest E, Lusakowska A, Borkowska J, Hausmanowa-Petrusewicz I. A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients. J Neurol Sci. 1997;146(1):67\u201372. https:\/\/doi.org\/10.1016\/s0022-510x(96)00284-5.","journal-title":"J Neurol Sci"},{"issue":"1","key":"915_CR9","doi-asserted-by":"publisher","first-page":"155","DOI":"10.1016\/j.jpeds.2012.05.067","volume":"162","author":"MA Farrar","year":"2013","unstructured":"Farrar MA, Vucic S, Johnston HM, du Sart D, Kiernan MC. Pathophysiological insights derived by natural history and motor function of spinal muscular atrophy. J Pediatr. 2013;162(1):155\u20139. https:\/\/doi.org\/10.1016\/j.jpeds.2012.05.067.","journal-title":"J Pediatr"},{"issue":"4","key":"915_CR10","doi-asserted-by":"publisher","first-page":"831","DOI":"10.1016\/j.ncl.2015.07.004","volume":"33","author":"SJ Kolb","year":"2015","unstructured":"Kolb SJ, Kissel JT. Spinal muscular atrophy. Neurol Clin. 2015;33(4):831\u201346. https:\/\/doi.org\/10.1016\/j.ncl.2015.07.004.","journal-title":"Neurol Clin"},{"issue":"6","key":"915_CR11","doi-asserted-by":"publisher","first-page":"71","DOI":"10.1186\/1750-1172-6-71","volume":"2","author":"A D'Amico","year":"2011","unstructured":"D\u2019Amico A, Mercuri E, Tiziano FD, Bertini E. Spinal muscular atrophy. Orphanet J Rare Dis. 2011;2(6):71. https:\/\/doi.org\/10.1186\/1750-1172-6-71.","journal-title":"Orphanet J Rare Dis"},{"issue":"1","key":"915_CR12","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.pediatrneurol.2011.09.001","volume":"46","author":"JA Markowitz","year":"2012","unstructured":"Markowitz JA, Singh P, Darras BT. Spinal muscular atrophy: a clinical and research update. Pediatr Neurol. 2012;46(1):1\u201312. https:\/\/doi.org\/10.1016\/j.pediatrneurol.2011.09.001.","journal-title":"Pediatr Neurol"},{"issue":"1","key":"915_CR13","doi-asserted-by":"publisher","first-page":"124","DOI":"10.1186\/s13023-017-0671-8","volume":"12","author":"IEC Verhaart","year":"2017","unstructured":"Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy\u2014a literature review. Orphanet J Rare Dis. 2017;12(1):124. https:\/\/doi.org\/10.1186\/s13023-017-0671-8.","journal-title":"Orphanet J Rare Dis"},{"issue":"12","key":"915_CR14","doi-asserted-by":"publisher","first-page":"1015","DOI":"10.1038\/sj.ejhg.5201288","volume":"12","author":"S Ogino","year":"2004","unstructured":"Ogino S, Wilson RB, Gold B. New insights on the evolution of the SMN1 and SMN2 region: simulation and meta-analysis for allele and haplotype frequency calculations. Eur J Hum Genet. 2004;12(12):1015\u201323. https:\/\/doi.org\/10.1038\/sj.ejhg.5201288.","journal-title":"Eur J Hum Genet"},{"key":"915_CR15","unstructured":"Spinal Muscular Atrophy Foundation. SMA Overview. https:\/\/smafoundation.org\/about-sma\/materials\/. Accessed 20 Feb 2023."},{"key":"915_CR16","doi-asserted-by":"publisher","unstructured":"Chaisomchit S, Thongnoppakhun W, Pho-iam T, Limwongse C, Likasitwattanakul S, Chareonsiriwatana W, et al. Investigation of carrier frequency for spinal muscular atrophy (SMA) in Thailand using dried blood spot (DBS) specimens. Genom Genet. 2016;9(2&3):78-84. https:\/\/doi.org\/10.14456\/gag.2016.11.","DOI":"10.14456\/gag.2016.11"},{"issue":"3","key":"915_CR17","doi-asserted-by":"publisher","first-page":"291","DOI":"10.1016\/j.pedneo.2022.01.002","volume":"63","author":"M Aksaralikitsunti","year":"2022","unstructured":"Aksaralikitsunti M, Sanmaneechai O. Health-related quality of life in Thai children with spinal muscular atrophy. Pediatr Neonatol. 2022;63(3):291\u20137. https:\/\/doi.org\/10.1016\/j.pedneo.2022.01.002.","journal-title":"Pediatr Neonatol"},{"issue":"3","key":"915_CR18","doi-asserted-by":"publisher","first-page":"197","DOI":"10.1016\/j.nmd.2017.11.004","volume":"28","author":"RS Finkel","year":"2018","unstructured":"Finkel RS, Mercuri E, Meyer OH, Simonds AK, Schroth MK, Graham RJ, et al. Diagnosis and management of spinal muscular atrophy: Part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018;28(3):197\u2013207. https:\/\/doi.org\/10.1016\/j.nmd.2017.11.004.","journal-title":"Neuromuscul Disord"},{"issue":"2","key":"915_CR19","doi-asserted-by":"publisher","first-page":"103","DOI":"10.1016\/j.nmd.2017.11.005","volume":"28","author":"E Mercuri","year":"2018","unstructured":"Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, et al. Diagnosis and management of spinal muscular atrophy: Part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28(2):103\u201315. https:\/\/doi.org\/10.1016\/j.nmd.2017.11.005.","journal-title":"Neuromuscul Disord"},{"key":"915_CR20","unstructured":"Clinical practice guideline for diagnosis and treatments for spinal muscular atrophy (SMA) in Thailand; 2023."},{"issue":"10063","key":"915_CR21","doi-asserted-by":"publisher","first-page":"3017","DOI":"10.1016\/s0140-6736(16)31408-8","volume":"388","author":"RS Finkel","year":"2016","unstructured":"Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017\u201326. https:\/\/doi.org\/10.1016\/s0140-6736(16)31408-8.","journal-title":"Lancet"},{"issue":"18","key":"915_CR22","doi-asserted-by":"publisher","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","volume":"377","author":"JR Mendell","year":"2017","unstructured":"Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713\u201322. https:\/\/doi.org\/10.1056\/NEJMoa1706198.","journal-title":"N Engl J Med"},{"issue":"4","key":"915_CR23","doi-asserted-by":"publisher","first-page":"284","DOI":"10.1016\/S1474-4422(21)00001-6","volume":"20","author":"JW Day","year":"2021","unstructured":"Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(4):284\u201393. https:\/\/doi.org\/10.1016\/S1474-4422(21)00001-6.","journal-title":"Lancet Neurol"},{"issue":"10","key":"915_CR24","doi-asserted-by":"publisher","first-page":"832","DOI":"10.1016\/S1474-4422(21)00251-9","volume":"20","author":"E Mercuri","year":"2021","unstructured":"Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(10):832\u201341. https:\/\/doi.org\/10.1016\/S1474-4422(21)00251-9.","journal-title":"Lancet Neurol"},{"issue":"10","key":"915_CR25","doi-asserted-by":"publisher","first-page":"915","DOI":"10.1056\/NEJMoa2009965","volume":"384","author":"G Baranello","year":"2021","unstructured":"Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384(10):915\u201323. https:\/\/doi.org\/10.1056\/NEJMoa2009965.","journal-title":"N Engl J Med"},{"issue":"5","key":"915_CR26","doi-asserted-by":"publisher","first-page":"427","DOI":"10.1056\/NEJMoa2102047","volume":"385","author":"BT Darras","year":"2021","unstructured":"Darras BT, Masson R, Mazurkiewicz-Be\u0142dzi\u0144ska M, Rose K, Xiong H, Zanoteli E, et al. Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls. N Engl J Med. 2021;385(5):427\u201335. https:\/\/doi.org\/10.1056\/NEJMoa2102047.","journal-title":"N Engl J Med"},{"key":"915_CR27","unstructured":"Day JW, Annoussamy M, Baranello G, Boespflug-Tanguy O, Borell S, Goemans N, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with Type 2 or 3 spinal muscular atrophy (SMA). CURE-SMA 2020."},{"issue":"7","key":"915_CR28","doi-asserted-by":"publisher","first-page":"1945","DOI":"10.1111\/ene.15499","volume":"30","author":"E Mercuri","year":"2023","unstructured":"Mercuri E, Baranello G, Boespflug-Tanguy O, De Waele L, Goemans N, Kirschner J, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: a randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Eur J Neurol. 2023;30(7):1945\u201356. https:\/\/doi.org\/10.1111\/ene.15499.","journal-title":"Eur J Neurol"},{"key":"915_CR29","unstructured":"Canadian Agency for Drugs and Technologies in Health. Pharmacoeconomic Review Report: Nusinersen (Spinraza): (Biogen Canada Inc.): Indication: Treatment of patients with 5q SMA. Ottawa; 2018."},{"issue":"1","key":"915_CR30","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1186\/s12962-020-00234-8","volume":"18","author":"P Thokala","year":"2020","unstructured":"Thokala P, Stevenson M, Kumar VM, Ren S, Ellis AG, Chapman RH. Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US. Cost Eff Resour Alloc. 2020;18(1):41. https:\/\/doi.org\/10.1186\/s12962-020-00234-8.","journal-title":"Cost Eff Resour Alloc."},{"key":"915_CR31","unstructured":"National Institute for Health and Care Excellence. Single technology appraisal: Nusinersen for treating spinal muscular atrophy [ID1069]: Document B: Company evidence submission. London; 2018."},{"issue":"6","key":"915_CR32","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. https:\/\/doi.org\/10.1007\/s40273-019-00769-6.","journal-title":"Pharmacoeconomics"},{"key":"915_CR33","unstructured":"Institute for Clinical and Economic Review. Spinraza\u00ae and Zolgensma\u00ae for spinal muscular atrophy: effectiveness and value: final evidence report. Boston; 2019."},{"key":"915_CR34","doi-asserted-by":"publisher","unstructured":"Dean R, Jensen I, Cyr P, Miller B, Maru B, Sproule DM, et al. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma\u00ae) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). J Mark Access Health Policy. 2021;9(1):1889841. https:\/\/doi.org\/10.1080\/20016689.2021.1889841.","DOI":"10.1080\/20016689.2021.1889841"},{"issue":"1","key":"915_CR35","doi-asserted-by":"publisher","first-page":"1601484","DOI":"10.1080\/20016689.2019.1601484","volume":"7","author":"DC Malone","year":"2019","unstructured":"Malone DC, Dean R, Arjunji R, Jensen I, Cyr P, Miller B, et al. Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. J Mark Access Health Policy. 2019;7(1):1601484. https:\/\/doi.org\/10.1080\/20016689.2019.1601484.","journal-title":"J Mark Access Health Policy."},{"issue":"7","key":"915_CR36","doi-asserted-by":"publisher","first-page":"823","DOI":"10.1080\/14712598.2020.1772747","volume":"20","author":"M Connock","year":"2020","unstructured":"Connock M, Andronis L, Auguste P, Dussart C, Armoiry X. Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent \u2018value for money\u2019 for the NHS? A rapid inquiry into suggestions that it may be cost-effective. Expert Opin Biol Ther. 2020;20(7):823\u20137. https:\/\/doi.org\/10.1080\/14712598.2020.1772747.","journal-title":"Expert Opin Biol Ther"},{"issue":"6","key":"915_CR37","doi-asserted-by":"publisher","first-page":"759","DOI":"10.1016\/j.jval.2020.09.021","volume":"24","author":"TF Broekhoff","year":"2021","unstructured":"Broekhoff TF, Sweegers CCG, Krijkamp EM, Mantel-Teeuwisse AK, Leufkens HGM, Goettsch WG, et al. Early cost-effectiveness of onasemnogene abeparvovec-xioi (zolgensma) and nusinersen (spinraza) treatment for spinal muscular atrophy I in The Netherlands with relapse scenarios. Value Health. 2021;24(6):759\u201369. https:\/\/doi.org\/10.1016\/j.jval.2020.09.021.","journal-title":"Value Health."},{"issue":"12","key":"915_CR38","doi-asserted-by":"publisher","first-page":"6544","DOI":"10.1007\/s00415-022-11319-0","volume":"269","author":"T Wang","year":"2022","unstructured":"Wang T, Scuffham P, Byrnes J, Downes M. Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type I in Australia. J Neurol. 2022;269(12):6544\u201354. https:\/\/doi.org\/10.1007\/s00415-022-11319-0.","journal-title":"J Neurol"},{"key":"915_CR39","doi-asserted-by":"publisher","first-page":"108","DOI":"10.1016\/j.vhri.2023.11.004","volume":"40","author":"BD Fernandes","year":"2024","unstructured":"Fernandes BD, D\u2019Athayde Rodrigues F, Cardoso Cirilo HN, Borges SS, Krug BC, Probst LF, et al. Cost-effectiveness of onasemnogene abeparvovec compared with nusinersen and risdiplam in patients with spinal muscular atrophy type 1 in Brazil: Custo-Efetividade do Onasemnogeno Abeparvoveque (AVXS-101) em Compara\u00e7\u00e3o ao Nusinersena e Risdiplam em Pacientes com Atrofia Muscular Espinhal Tipo 1 no Brasil. Value Health Reg Issues. 2024;40:108\u201317. https:\/\/doi.org\/10.1016\/j.vhri.2023.11.004.","journal-title":"Value Health Reg Issues."},{"key":"915_CR40","unstructured":"Pannarunothai S, Pilasant S, Kingkaew P, Saengsri W. Guideline for health technology assessment in thailand updated edition: 2019. Nonthaburi: Health Systems Research Institute; 2021."},{"key":"915_CR41","unstructured":"Ministry of Commerce. General consumer price index of country. http:\/\/www.price.moc.go.th\/price\/cpi\/index_new.asp. Accessed 1 Jul 2023."},{"issue":"6","key":"915_CR42","doi-asserted-by":"publisher","first-page":"467","DOI":"10.1017\/s0266462319000321","volume":"35","author":"P Leelahavarong","year":"2019","unstructured":"Leelahavarong P, Doungthipsirikul S, Kumluang S, Poonchai A, Kittiratchakool N, Chinnacom D, et al. Health technology assessment in Thailand: institutionalization and contribution to healthcare decision making: review of literature. Int J Technol Assess Health Care. 2019;35(6):467\u201373. https:\/\/doi.org\/10.1017\/s0266462319000321.","journal-title":"Int J Technol Assess Health Care"},{"key":"915_CR43","unstructured":"Bank of Thailand. Daily Foreign Exchange Rates. 2023; https:\/\/www.bot.or.th\/en\/statistics\/exchange-rate.html. Accessed 26 Feb 2024."},{"issue":"1","key":"915_CR44","doi-asserted-by":"publisher","first-page":"10","DOI":"10.1016\/j.jval.2021.10.008","volume":"25","author":"D Husereau","year":"2022","unstructured":"Husereau D, Drummond M, Augustovski F, de Bekker-Grob E, Briggs AH, Carswell C, et al. Consolidated health economic evaluation reporting standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR CHEERS II good practices task force. Value Health. 2022;25(1):10\u201331. https:\/\/doi.org\/10.1016\/j.jval.2021.10.008.","journal-title":"Value Health."},{"issue":"4","key":"915_CR45","doi-asserted-by":"publisher","first-page":"322","DOI":"10.1097\/PEP.0b013e3182351f04","volume":"23","author":"AM Glanzman","year":"2011","unstructured":"Glanzman AM, McDermott MP, Montes J, Martens WB, Flickinger J, Riley S, et al. Validation of the Children\u2019s Hospital of Philadelphia infant test of neuromuscular disorders (CHOP INTEND). Pediatr Phys Ther. 2011;23(4):322\u20136. https:\/\/doi.org\/10.1097\/PEP.0b013e3182351f04.","journal-title":"Pediatr Phys Ther"},{"issue":"6","key":"915_CR46","doi-asserted-by":"publisher","first-page":"883","DOI":"10.1002\/ana.25101","volume":"82","author":"SJ Kolb","year":"2017","unstructured":"Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, et al. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol. 2017;82(6):883\u201391. https:\/\/doi.org\/10.1002\/ana.25101.","journal-title":"Ann Neurol"},{"key":"915_CR47","doi-asserted-by":"publisher","DOI":"10.1093\/oso\/9780198526629.001.0001","volume-title":"Decision modelling for health economic evaluation","author":"A Briggs","year":"2006","unstructured":"Briggs A, Sculpher M, Claxton K. Decision modelling for health economic evaluation. Oxford: Oxford University Press; 2006."},{"key":"915_CR48","unstructured":"World Health Organization. Life tables by country: Thailand. https:\/\/apps.who.int\/gho\/data\/?theme=main&vid=61640. Accessed 1 Jul 2023."},{"key":"915_CR49","unstructured":"Finance Department. Faculty of Medicine Siriraj Hospital. Charge to Cost Ratio. 2023; https:\/\/www.si.mahidol.ac.th\/th\/division\/finance\/. Accessed 1 Jul 2023."},{"issue":"5","key":"915_CR50","doi-asserted-by":"publisher","first-page":"597","DOI":"10.1177\/0272989x211001859","volume":"41","author":"T Shiroiwa","year":"2021","unstructured":"Shiroiwa T, Ikeda S, Noto S, Fukuda T, Stolk E. Valuation survey of EQ-5D-Y based on the international common protocol: development of a value set in Japan. Med Decis Making. 2021;41(5):597\u2013606. https:\/\/doi.org\/10.1177\/0272989x211001859.","journal-title":"Med Decis Making"},{"key":"915_CR51","doi-asserted-by":"publisher","first-page":"1054931","DOI":"10.3389\/fpubh.2022.1054931","volume":"10","author":"J Hu","year":"2022","unstructured":"Hu J, Zhu L, Bao H, Liu Y, Xing H, Kang Q, et al. Utility estimations of different health states of patients with type I, II, and III spinal muscular atrophy in China: a mixed approach study with patient and proxy-reported data. Front Public Health. 2022;10:1054931. https:\/\/doi.org\/10.3389\/fpubh.2022.1054931.","journal-title":"Front Public Health"},{"key":"915_CR52","volume-title":"Methods for the economic evaluation of health care programmes","author":"M Drummond","year":"2015","unstructured":"Drummond M, Sculpher M, Claxton K, Stoddart G, Torrance G. Methods for the economic evaluation of health care programmes. 4th ed. Oxford: Oxford University Press; 2015.","edition":"4"},{"issue":"7","key":"915_CR53","doi-asserted-by":"publisher","first-page":"A556","DOI":"10.1016\/j.jval.2014.08.1831","volume":"17","author":"P Vemer","year":"2014","unstructured":"Vemer P, Corro Ramos I, Van Voorn G, Al MJ, Feenstra TL. Advishe: a new tool to report validation of health-economic decision models. Value Health. 2014;17(7):A556\u20137. https:\/\/doi.org\/10.1016\/j.jval.2014.08.1831.","journal-title":"Value Health."},{"issue":"4","key":"915_CR54","doi-asserted-by":"publisher","first-page":"349","DOI":"10.1007\/s40273-015-0327-2","volume":"34","author":"P Vemer","year":"2016","unstructured":"Vemer P, Corro Ramos I, van Voorn GA, Al MJ, Feenstra TL. AdViSHE: a validation-assessment tool of health-economic models for decision makers and model users. Pharmacoeconomics. 2016;34(4):349\u201361. https:\/\/doi.org\/10.1007\/s40273-015-0327-2.","journal-title":"Pharmacoeconomics"},{"key":"915_CR55","volume-title":"Health technology assessment of gene therapies: are our methods fit for purpose?","author":"S Besley","year":"2022","unstructured":"Besley S, Henderson N, Towse A, Cole A. Health technology assessment of gene therapies: are our methods fit for purpose? London: Office of Health Economics; 2022."},{"key":"915_CR56","unstructured":"Zorginstituut Nederland. Guideline for economic evaluations in healthcare; 2016."},{"key":"915_CR57","unstructured":"Brazil. Ministry of Health. Secretariat of Science, Technology and Strategic Inputs. Department of Science and Technology. Methodological guideline: economic evaluation of health technologies. 2nd ed. 2014; https:\/\/rebrats.saude.gov.br\/diretrizes-metodologicas. Accessed 19 Mar 2024."},{"issue":"7","key":"915_CR58","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. https:\/\/doi.org\/10.1056\/NEJMoa1710504.","journal-title":"N Engl J Med"},{"issue":"18","key":"915_CR59","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":"9","key":"915_CR60","doi-asserted-by":"publisher","first-page":"A725","DOI":"10.1016\/j.jval.2017.08.1962","volume":"20","author":"R Thompson","year":"2017","unstructured":"Thompson R, Vaidya S, Teynor M. The utility of different approachs to developing health utilities data in childhood rare diseases\u2014a case study in spinal muscular atrophy (SMA). Value Health. 2017;20(9):A725\u20136. https:\/\/doi.org\/10.1016\/j.jval.2017.08.1962.","journal-title":"Value Health."},{"key":"915_CR61","volume-title":"Nusinersen for treating spinal muscular atrophy: a single technology appraisal","author":"P Tappenden","year":"2018","unstructured":"Tappenden P, Hamilton J, Kaltenthaler E, Hock E, Rawdin A, Mukuria C, et al. Nusinersen for treating spinal muscular atrophy: a single technology appraisal. Sheffield: School of Health and Related Research (ScHARR), The University of Sheffield; 2018."},{"key":"915_CR62","unstructured":"Zorginstituut Nederland. Pakketadvies sluisgeneesmiddel nusinersen (Spinraza\u00ae) voor de behandeling van spinale musculaire atrofie (SMA). https:\/\/www.zorginstituutnederland.nl\/publicaties\/adviezen\/2018\/02\/07\/pakketadvies-nusinersen-spinraza-voor-de-behandeling-van-spinale-musculaire-atrofie-sma. Accessed 1 Jul 2023."},{"issue":"1","key":"915_CR63","doi-asserted-by":"publisher","first-page":"e1","DOI":"10.1212\/wnl.0000000000009715","volume":"95","author":"GM Chambers","year":"2020","unstructured":"Chambers GM, Settumba SN, Carey KA, Cairns A, Menezes MP, Ryan M, et al. Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy. Neurology. 2020;95(1):e1\u201310. https:\/\/doi.org\/10.1212\/wnl.0000000000009715.","journal-title":"Neurology"},{"issue":"5","key":"915_CR64","doi-asserted-by":"publisher","first-page":"e548","DOI":"10.1542\/peds.2004-0668","volume":"114","author":"BH Chung","year":"2004","unstructured":"Chung BH, Wong VC, Ip P. Spinal muscular atrophy: survival pattern and functional status. Pediatrics. 2004;114(5):e548\u201353. https:\/\/doi.org\/10.1542\/peds.2004-0668.","journal-title":"Pediatrics"},{"key":"915_CR65","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1016\/B978-0-12-803685-3.00001-X","volume-title":"Spinal muscular atrophy","author":"M Oskoui","year":"2017","unstructured":"Oskoui M, Darras BT, De Vivo DC. Chapter 1\u2014spinal muscular atrophy: 125 years later and on the verge of a cure. In: Sumner CJ, Paushkin S, Ko C-P, editors. Spinal muscular atrophy. New York: Academic Press; 2017. p. 3\u201319."},{"issue":"1","key":"915_CR66","doi-asserted-by":"publisher","first-page":"138","DOI":"10.1186\/s40545-023-00643-z","volume":"16","author":"D Butani","year":"2023","unstructured":"Butani D, Faradiba D, Dabak SV, Isaranuwatchai W, Huang-Ku E, Pachanee K, et al. Expanding access to high-cost medicines under the Universal Health Coverage scheme in Thailand: review of current practices and recommendations. J Pharm Policy Pract. 2023;16(1):138. https:\/\/doi.org\/10.1186\/s40545-023-00643-z.","journal-title":"J Pharm Policy Pract."},{"issue":"1","key":"915_CR67","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1186\/s13023-022-02283-z","volume":"17","author":"MJ Postma","year":"2022","unstructured":"Postma MJ, Noone D, Rozenbaum MH, Carter JA, Botteman MF, Fenwick E, et al. Assessing the value of orphan drugs using conventional cost-effectiveness analysis: is it fit for purpose? Orphanet J Rare Dis. 2022;17(1):157. https:\/\/doi.org\/10.1186\/s13023-022-02283-z.","journal-title":"Orphanet J Rare Dis"},{"issue":"2","key":"915_CR68","doi-asserted-by":"publisher","DOI":"10.1016\/s2352-3026(24)00011-5","volume":"11","author":"The Lancet Haematology","year":"2024","unstructured":"The Lancet Haematology. Improving equity for people living with rare diseases. Lancet Haematol. 2024;11(2): e85. https:\/\/doi.org\/10.1016\/s2352-3026(24)00011-5.","journal-title":"Lancet Haematol."},{"key":"915_CR69","doi-asserted-by":"publisher","DOI":"10.3390\/ijerph20064732","author":"T Adachi","year":"2023","unstructured":"Adachi T, El-Hattab AW, Jain R, Nogales Crespo KA, Quirland Lazo CI, Scarpa M, et al. Enhancing equitable access to rare disease diagnosis and treatment around the world: a review of evidence, policies, and challenges. Int J Environ Res Public Health. 2023. https:\/\/doi.org\/10.3390\/ijerph20064732.","journal-title":"Int J Environ Res Public Health"},{"issue":"1","key":"915_CR70","doi-asserted-by":"publisher","first-page":"484","DOI":"10.1186\/s12913-023-09494-5","volume":"23","author":"M Drummond","year":"2023","unstructured":"Drummond M, Ciani O, Fornaro G, Jommi C, Dietrich ES, Espin J, et al. How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy? BMC Health Serv Res. 2023;23(1):484. https:\/\/doi.org\/10.1186\/s12913-023-09494-5.","journal-title":"BMC Health Serv Res"},{"issue":"1","key":"915_CR71","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1017\/s0266462310001297","volume":"27","author":"M Klemp","year":"2011","unstructured":"Klemp M, Fr\u00f8nsdal KB, Facey K. What principles should govern the use of managed entry agreements? Int J Technol Assess Health Care. 2011;27(1):77\u201383. https:\/\/doi.org\/10.1017\/s0266462310001297.","journal-title":"Int J Technol Assess Health Care"},{"issue":"5","key":"915_CR72","doi-asserted-by":"publisher","first-page":"593","DOI":"10.1016\/j.jval.2019.01.001","volume":"22","author":"H Kim","year":"2019","unstructured":"Kim H, Goodall S, Liew D. Health technology assessment challenges in oncology: 20 years of value in health. Value Health. 2019;22(5):593\u2013600. https:\/\/doi.org\/10.1016\/j.jval.2019.01.001.","journal-title":"Value Health."},{"issue":"4","key":"915_CR73","doi-asserted-by":"publisher","first-page":"425","DOI":"10.1017\/s0266462307070651","volume":"23","author":"J Hutton","year":"2007","unstructured":"Hutton J, Trueman P, Henshall C. Coverage with evidence development: an examination of conceptual and policy issues. Int J Technol Assess Health Care. 2007;23(4):425\u201332. https:\/\/doi.org\/10.1017\/s0266462307070651.","journal-title":"Int J Technol Assess Health Care"},{"issue":"1","key":"915_CR74","doi-asserted-by":"publisher","first-page":"47","DOI":"10.1186\/s13023-024-03041-z","volume":"19","author":"VW Dayer","year":"2024","unstructured":"Dayer VW, Drummond MF, Dabbous O, Toumi M, Neumann P, Tunis S, et al. Real-world evidence for coverage determination of treatments for rare diseases. Orphanet J Rare Dis. 2024;19(1):47. https:\/\/doi.org\/10.1186\/s13023-024-03041-z.","journal-title":"Orphanet J Rare Dis"},{"issue":"5","key":"915_CR75","doi-asserted-by":"publisher","first-page":"483","DOI":"10.1080\/14737167.2023.2197214","volume":"23","author":"M Toumi","year":"2023","unstructured":"Toumi M, Dabbous O, Aball\u00e9a S, Drummond MF, von der Schulenburg JG, Malone DC, et al. Recommendations for economic evaluations of cell and gene therapies: a systematic literature review with critical appraisal. Expert Rev Pharmacoecon Outcomes Res. 2023;23(5):483\u201397. https:\/\/doi.org\/10.1080\/14737167.2023.2197214.","journal-title":"Expert Rev Pharmacoecon Outcomes Res"},{"issue":"6","key":"915_CR76","doi-asserted-by":"publisher","first-page":"661","DOI":"10.1016\/j.jval.2019.03.012","volume":"22","author":"MF Drummond","year":"2019","unstructured":"Drummond MF, Neumann PJ, Sullivan SD, Fricke F-U, Tunis S, Dabbous O, et al. Analytic considerations in applying a general economic evaluation reference case to gene therapy. Value Health. 2019;22(6):661\u20138. https:\/\/doi.org\/10.1016\/j.jval.2019.03.012.","journal-title":"Value Health."}],"container-title":["Applied Health Economics and Health Policy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40258-024-00915-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40258-024-00915-y\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40258-024-00915-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,13]],"date-time":"2025-02-13T16:07:48Z","timestamp":1739462868000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40258-024-00915-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9,27]]},"references-count":76,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,3]]}},"alternative-id":["915"],"URL":"https:\/\/doi.org\/10.1007\/s40258-024-00915-y","relation":{},"ISSN":["1175-5652","1179-1896"],"issn-type":[{"value":"1175-5652","type":"print"},{"value":"1179-1896","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,9,27]]},"assertion":[{"value":"20 August 2024","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 September 2024","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"S.K. and P.L. conducted the main analysis and interpretation of the data, drafted the manuscript, and took responsibility for the integrity of the data and the accuracy of the data analysis. P.L. and S.K. conceived and designed the study. Data collection and analysis were performed by J.P. and K.P. Moreover,\u00a0O.S. and V.S. provided clinical opinions and policy implications. All authors read and approved the final manuscript.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Author Contributions"}},{"value":"Open access funding provided by Mahidol University. This study was supported by the Siriraj Research Developmental Fund, Faculty of Medicine Siriraj Hospital, Mahidol University [grant no. (IO) R016532007]. This manuscript represents the views of the authors. The funding sources had no role in study design, data collection and analysis, the decision to publish, or the preparation of the manuscript.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Financial support"}},{"value":"The authors declare that they have no conflicts of interest.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Potential conflicts of interest"}},{"value":"This study was conducted in accordance with the Declaration of Helsinki. The protocol was approved by the ethics committee of the Siriraj Hospital institutional review board [certificate of Approval (CoA) no. Si 802\/2021].","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics statement"}},{"value":"Written informed consent was provided by study participants and all participating caregivers of participating children.","order":6,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"The study participants and the participating caregivers of participating children consented to their data being used for publication.","order":7,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Data used in this study were retrieved from both published and unpublished literature; data and sources of data are provided in Appendix 2: Model input parameters. The datasets used and\/or analyzed during the current study are available from the corresponding author upon reasonable request.","order":8,"name":"Ethics","group":{"name":"EthicsHeading","label":"Data availability"}},{"value":"The model used in this study was provided to the journal\u2019s peer reviewers for their reference when reviewing the manuscript. The details of the model are available from the corresponding author upon reasonable request.","order":9,"name":"Ethics","group":{"name":"EthicsHeading","label":"Code availability"}}]}}