{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,30]],"date-time":"2026-01-30T04:31:28Z","timestamp":1769747488047,"version":"3.49.0"},"reference-count":31,"publisher":"The Journal of Health Economics and Outcomes Research","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JHEOR"],"abstract":"<jats:p>Introduction: Primary myelofibrosis (MF) is a rare hematologic disease belonging to the group of Philadelphia-negative chronic myeloproliferative neoplasms. Identification of the Janus Kinase (JAK) gene mutations inaugurated a new era in the targeted therapy of myeloproliferative diseases. Ruxolitinib is the first JAK1\/JAK2 inhibitor specifically approved for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis. The objective of this study was to assess the cost-effectiveness of ruxolitinib vs best available therapy (BAT) in MF patients in Spain.\n\nMethods: A decision-tree and Markov model were adapted to the Spanish setting to assess the cost-effectiveness of ruxolitinib vs.\u00a0BAT on a lifetime horizon (\u226415 years) from the societal perspective, while healthcare system perspective was included in the one-way sensitivity analysis. The population was assumed to be similar to that of the COMFORT-II clinical trial (CT), which was also the source of treatment efficacy data. BAT composition was derived from the same CT and validated with Spanish experts. Utilities were derived from the COMFORT-I CT. Costs included treatment, management, hospitalizations, emergency and outpatient visits, as well as adverse events and end-of-life costs. Additionally, costs associated to productivity loss were taken into account. Resource use was validated with experts and costs were extracted from Spanish sources. A probabilistic sensitivity analysis was also performed to evaluate the consistency of the results under the uncertainty or variability of the input data.\n\nResults: Patients on ruxolitinib accumulated 6.1 life years gained (LYGs), resulting in 73% extra life-years compared to patients treated with BAT (3.5LYs gained). Ruxolitinib provided 4.4 quality-adjusted life years (QALYs), with a 99% improvement compared to BAT (2.2 QALYs). This analysis gave an incremental cost of \u20ac47 199 per LYG and an incremental cost of \u20ac55 616 per QALY gained from the societal perspective.\n\nConclusions: Ruxolitinib would be cost-effective in Spain according to the end-of-life criteria defined by the NICE and commonly referred for Spain (cost-effectiveness threshold of \u20ac61 500\/QALY), in line with results published for other European countries.<\/jats:p>","DOI":"10.36469\/9808","type":"journal-article","created":{"date-parts":[[2019,10,16]],"date-time":"2019-10-16T20:58:49Z","timestamp":1571259529000},"page":"162-174","source":"Crossref","is-referenced-by-count":6,"title":["Cost-effectiveness of Ruxolitinib vs Best Available Therapy in the Treatment of Myelofibrosis in Spain"],"prefix":"10.36469","volume":"5","author":[{"given":"Mar\u00eda Teresa","family":"G\u00f3mez-Casares","sequence":"first","affiliation":[{"name":"Doctor Negr\u00edn University Hospital, Las Palmas de Gran Canaria, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Juan Carlos","family":"Hern\u00e1ndez-Boluda","sequence":"additional","affiliation":[{"name":"Clinic University Hospital, Valencia, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Antonio","family":"Jim\u00e9nez-Velasco","sequence":"additional","affiliation":[{"name":"Regional University Hospital, M\u00e1laga, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joaquin","family":"Mart\u00ednez-L\u00f3pez","sequence":"additional","affiliation":[{"name":"Hospital Universitario 12 de Octubre, Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mar\u00eda Giovanna","family":"Ferrario","sequence":"additional","affiliation":[{"name":"Outcomes\u201910, Castell\u00f3n de la Plana, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Irmina","family":"Gozalbo","sequence":"additional","affiliation":[{"name":"Outcomes\u201910, Castell\u00f3n de la Plana, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joana","family":"Gostkorzewicz","sequence":"additional","affiliation":[{"name":"Novartis Oncology, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rudi","family":"Subir\u00e1","sequence":"additional","affiliation":[{"name":"Novartis Oncology, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"22273","published-online":{"date-parts":[[2017,11,29]]},"reference":[{"key":"ref0","doi-asserted-by":"crossref","unstructured":"Gimenez E, Besses C, Boque C, et al. Indirect and non-medical economic burden, quality-of-life, and disabilities of the myelofibrosis disease in Spain. J Med Econ. 2014;17(6):435\u201341.","DOI":"10.3111\/13696998.2014.903257"},{"key":"ref1","doi-asserted-by":"publisher","unstructured":"Vandewalle B, Andreozzi V, Almeida J, F\u00e9lix J. Pharmacoeconomics of ruxolitinib therapy in patients with myelofibrosis. J Med Econ. 2016;19(4):424\u201331.","DOI":"10.3111\/13696998.2015.1133430"},{"key":"ref2","doi-asserted-by":"publisher","unstructured":"Cervantes  F,  Vannucchi  AM,  Kiladjian  JJ,  et  al.  Three-year  efficacy,  safety,  and  survival  findings  from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013;122(25):4047\u201353.","DOI":"10.1182\/blood-2016-11-750505"},{"key":"ref3","doi-asserted-by":"crossref","unstructured":"Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis : results of a median 3-year follow-up of COMFORT-I. 2015;100(4):479\u201388.","DOI":"10.3324\/haematol.2014.115840"},{"key":"ref4","doi-asserted-by":"publisher","unstructured":"Harrison C, Kiladjian J-J, Al-Ali HK, et al. JAK Inhibition with Ruxolitinib versus Best Available Therapy for Myelofibrosis. N Engl J Med. 2012;366(9):787\u201398.","DOI":"10.1056\/nejmoa1110556"},{"key":"ref5","doi-asserted-by":"publisher","unstructured":"Grupo Espa\u00f1ol de Enfermedades Mieloproliferativas Cr\u00f3nicas Filadelfia Negativas (GEMFIN). Manual de recomendaciones en neoplasias mieloproliferativas cr\u00f3nicas filadelfia negativas. http:\/\/www.sehh.es\/images\/stories\/recursos\/2014\/documentos\/guias\/GUIA_GEMFIN.pdf. Accessed June 2017.","DOI":"10.24245\/rhematol.v20i4.3139"},{"key":"ref6","doi-asserted-by":"publisher","unstructured":"Reilly JT, Mcmullin MF, Beer PA, et al. Use of JAK inhibitors in the management of myelofibrosis: A revision of  the British Committee for Standards in Haematology Guidelines for Investigation and Management of  Myelofibrosis 2012. Br J Haematol. 2014;167(3):418\u201320.","DOI":"10.1111\/bjh.12985"},{"key":"ref7","doi-asserted-by":"publisher","unstructured":"Mesa R, Jamieson C, Bhatia R, et al. Myeloproliferative Neoplasms, Version 2.2017, NCCN Clinical Practice Guidelines in Oncology. Natl Compr Canc Netw. 2016;14(12):1572\u2013611.","DOI":"10.6004\/jnccn.2017.0083"},{"key":"ref8","doi-asserted-by":"publisher","unstructured":"Mehta  J,  Wang  H,  Fryzek  JP,  Iqbal  SU,  Mesa  R.  Health  resource  utilization  and  cost  associated  with myeloproliferative neoplasms in a large United States health plan. Leuk Lymphoma. 2014;55(10):2368\u201374.","DOI":"10.3109\/10428194.2013.879127"},{"key":"ref9","doi-asserted-by":"publisher","unstructured":"Harrison CN, Vannucchi AM, Kiladjian J-J, et al. Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia. 2016;30(8):1701.","DOI":"10.1038\/leu.2016.148"},{"key":"ref10","doi-asserted-by":"publisher","unstructured":"Wade R, Rose M, Neilson AR, et al. Ruxolitinib for the Treatment of Myelofibrosis: A NICE Single Technology Appraisal. Pharmacoeconomics. 2013;31(10):841\u201352.","DOI":"10.1007\/s40273-013-0083-0"},{"key":"ref11","doi-asserted-by":"publisher","unstructured":"Puig-Junoy J, Oliva-Moreno J, Trapero-Bertr\u00e1n M, Abell\u00e1n-Perpi\u00f1\u00e1n JM, Brosa M SC de la S (CatSalut). Gu\u00eda  y  recomendaciones  para  la  realizaci\u00f3n  y  presentaci\u00f3n  de  evaluaciones  econ\u00f3micas  y  an\u00e1lisis  de impacto presupuestario de medicamentos en el \u00e1mbito del CatSalut. Generalitat de Catalunya. Departament de  Salut.  Servei  Catal\u00e0  de  la  Salut.  Barcelona;  2014.  http:\/\/catsalut.gencat.cat\/web\/.content\/minisite\/catsalut\/proveidors_professionals\/medicaments_farmacia\/farmaeconomica\/caeip\/documents\/gaeip_publica_castellano_octubre2014_catsalut.pdf. Accessed December 2016.","DOI":"10.1016\/s0025-7753(11)70030-6"},{"key":"ref12","doi-asserted-by":"publisher","unstructured":"Mukuria C, Rowen D, Brazier JE, Young TA, Nafees B. Deriving a Preference-Based Measure for Myelofibrosis from the EORTC QLQ-C30 and the MF-SAF. Value Health. 2015;18(6):846\u201355.","DOI":"10.1016\/j.jval.2015.07.004"},{"key":"ref13","unstructured":"Consejo General de Colegios Oficiales de Farmac\u00e9uticos. Bot Plus Web. https:\/\/botplusweb.portalfarma.com\/. Accessed February 2017."},{"key":"ref14","unstructured":"JAKAVI. Summary of product characteristics. http:\/\/www.ema.europa.eu\/docs\/en_GB\/document_library\/EPAR_-_Product_Information\/human\/002464\/WC500133223.pdf. Accessed February 2017."},{"key":"ref15","unstructured":"Ministerio de Sanidad Servicios Sociales e Igualdad. Listado de medicamentos afectados por las deducciones del  real  decreto-ley  8\/2010  -Noviembre-2016.  https:\/\/www.msssi.gob.es\/profesionales\/farmacia\/pdf\/DeduccionesNoviembre2016.pdf. Accessed December 2016."},{"key":"ref16","unstructured":"Oblikue  Consulting.  eSalud  -  Informaci\u00f3n  econ\u00f3mica  del  sector  sanitario.  http:\/\/esalud.oblikue.com\/. Accessed February 2017."},{"key":"ref17","unstructured":"Haematological  Malignancy  Research  Network.  Clinical  management,  resource  utilisation  and outcome  in  primary  and  secondary  myelofibrosis.  2012.  p.  1\u201349.  https:\/\/www.hmrn.org\/Download.aspx?target=document&id=3. Accessed December 2016"},{"key":"ref18","doi-asserted-by":"publisher","unstructured":"Harrison CN, Kiladjian J, Al-Ali HK, et al. Results of a randomized study of the JAK inhibitor ruxolitinib (INC424) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) or post-essential thrombocythemia-myelofibrosis (PET-MF). J Clin Oncol. 2011;29(18 suppl):LBA6501-LBA6501.","DOI":"10.1200\/jco.2011.29.18_suppl.lba6501"},{"key":"ref19","unstructured":"Instituto Nacional de Estad\u00edstica (INE). Encuesta de Poblaci\u00f3n Activa (EPA). 2014. http:\/\/www.ine.es\/dyngs\/INEbase\/es\/operacion.htm?c=Estadistica_C&cid=1254736176918&menu=resultados&secc= 1254736195128&idp=1254735976595. Accessed December 2016"},{"key":"ref20","doi-asserted-by":"publisher","unstructured":"Mesa R, Miller CB, Thyne M, et al. Myeloproliferative neoplasms (MPNs) have a significant impact on patients\u2019 overall health and productivity: the MPN Landmark survey. BMC Cancer. 2016;16(1):167.","DOI":"10.1186\/s12885-016-2208-2"},{"key":"ref21","unstructured":"National Institute for Health and Clare Excellence. Update Report On The Application Of The \u201cEnd -Of- Life\u201d Supplementary Advice In Health Technology Appraisals. http:\/\/www.nice.org.uk\/media\/E4A\/79\/SupplementaryAdviceTACEoL.pdf. Accessed June 2017. 2009."},{"key":"ref22","doi-asserted-by":"publisher","unstructured":"Hahl J, Kurki S, Miettinen T, Snicker K. Cost-effectiveness of ruxolitinib for the treatment of myelofibrosis in Finland economic evaluation based on Finnish Auria Biobank data on health care resource utilization. Value Health. 2015;8:3.","DOI":"10.1016\/j.jval.2015.09.2446"},{"key":"ref23","unstructured":"Evidence Review Group Report. Ruxolitinib for the treatment of myelofibrosis: A Single Technology Appraisal.   2012.   https:\/\/www.nice.org.uk\/guidance\/ta289\/documents\/myelofibrosis-splenomegaly-symptoms-ruxolitinib-evidence-review-group-report2. Accessed January 2017."},{"key":"ref24","unstructured":"The World Bank. World Bank national accounts data, and OECD National Accounts data files. http:\/\/data.worldbank.org\/indicator\/NY.GDP.PCAP.CN?locations=FI. Accessed July 2017."},{"key":"ref25","doi-asserted-by":"publisher","unstructured":"Marseille E, Larson B, Kazi DS, Kahn JG, Rosen S. Thresholds for the cost-effectiveness of interventions: alternative approaches. Bull World Health Organ. 2015;93(2):118\u201324.","DOI":"10.2471\/blt.14.138206"},{"key":"ref26","unstructured":"National Institute for Health and Care Excellence (NICE). Ruxolitinib for treating disease-related splenomegaly or symptoms in adults with myelofibrosis. https:\/\/www.nice.org.uk\/guidance\/ta386 Published 23 March 2016. Accessed July 2017."},{"key":"ref27","unstructured":"Zagadailov E, Fine M, Shields A. Patient-reported outcomes are changing the landscape in oncology care: Challenges and opportunities for payers. Am Heal Drug Benefits. 2013;6(5)."},{"key":"ref28","doi-asserted-by":"publisher","unstructured":"Mesa RA, Shields A, Hare T, et al. Progressive burden of myelofibrosis in untreated patients: Assessment of patient-reported outcomes in patients randomized to placebo in the COMFORT-I study. Leuk  Res. 2013;37:911\u20136.","DOI":"10.1016\/j.leukres.2013.04.017"},{"key":"ref29","doi-asserted-by":"publisher","unstructured":"Crespo C, Moreno E, Sierra J, Serip S, Rubio M. Cost-effectiveness analysis of azacitidine in the treatment of high-risk myelodysplastic syndromes in Spain. Health Econ Rev. 2013;3(1):28.","DOI":"10.1186\/2191-1991-3-28"},{"key":"ref30","doi-asserted-by":"publisher","unstructured":"G\u00f3mez-Batiste X, Tuca A, Corrales E, et al. Resource consumption and costs of palliative care services in Spain: A Multicenter Prospective Study. 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