224
Views
14
CrossRef citations to date
0
Altmetric
Original Research

Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Relapsed or Refractory B-Cell Acute Lymphoblastic Leukaemia in Children and Young Adults in Spain

ORCID Icon, , , , &
Pages 253-264 | Published online: 15 May 2020

References

  • Coebergh JW, Reedijk AM, de Vries E, et al. Leukaemia incidence and survival in children and adolescents in Europe during 1978–1997. Report from the automated childhood cancer information system project. Eur J Cancer. 2006;42(13):2019–2036. doi:10.1016/j.ejca.2006.06.005
  • Wartenberg D, Groves FD, Adelman AS, editors. Acute lymphoblastic leukemia: epidemiology and etiology. In: Acute Leukemias. Berlin: Springer; 2008:77–93. doi:10.1007/978-3-540-72304-2_5.
  • Hunger SP, Mullighan CG. Acute lymphoblastic leukemia in children. Longo DL, ed. N Engl J Med. 2015;373(16):1541–1552. doi:10.1056/NEJMra1400972
  • Pui CH, Sandlund J, Pei D, et al. Improved outcome for children with acute lymphoblastic leukemia: results of total therapy study XIIIB at St Jude Children’s Research Hospital. Blood. 2004;104(9):2690–2696. doi:10.1182/blood-2004-04-1616
  • Nguyen K, Devidas M, Cheng SC, et al. Factors influencing survival after relapse from acute lymphoblastic leukemia: a children’s oncology group study. Leukemia. 2008;22(12):2142–2150. doi:10.1038/leu.2008.251
  • Reismüller B, Peters C, Dworzak MN, et al. Outcome of children and adolescents with a second or third relapse of acute lymphoblastic leukemia (ALL). J Pediatr Hematol Oncol. 2013;35(5):e200–e204. doi:10.1097/MPH.0b013e318290c3d6
  • Chessells JM, Veys P, Kempski H, et al. Long-term follow-up of relapsed childhood acute lymphoblastic leukaemia. Br J Haematol. 2003;123(3):396–405. doi:10.1046/j.1365-2141.2003.04584.x
  • Ceppi F, Duval M, Leclerc JM, et al. Improvement of the outcome of relapsed or refractory acute lymphoblastic leukemia in children using a risk-based treatment strategy. Mills K, ed. PLoS One. 2016;11(9):e0160310. doi:10.1371/journal.pone.0160310
  • Nietfeld JJ, Pasquini MC, Logan BR, Verter F, Horowitz MM. Lifetime probabilities of hematopoietic stem cell transplantation in the U.S. Biol Blood Marrow Transplant. 2008;14(3):316–322. doi:10.1016/j.bbmt.2007.12.493
  • Dini G, Zecca M, Balduzzi A, et al. No difference in outcome between children and adolescents transplanted for acute lymphoblastic leukemia in second remission. Blood. 2011;118(25):6683–6690. doi:10.1182/blood-2011-05-354233
  • Jacobsohn DA, Hewlett B, Ranalli M, Seshadri R, Duerst R, Kletzel M. Outcomes of unrelated cord blood transplants and allogeneic-related hematopoietic stem cell transplants in children with high-risk acute lymphocytic leukemia. Bone Marrow Transplant. 2004;34(10):901–907. doi:10.1038/sj.bmt.1704681
  • Mateos MK, O’Brien TA, Oswald C, et al. Transplant-related mortality following allogeneic hematopoeitic stem cell transplantation for pediatric acute lymphoblastic leukemia: 25-year retrospective review. Pediatr Blood Cancer. 2013;60(9):1520–1527. doi:10.1002/pbc.24559
  • Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with c-cell lymphoblastic leukemia. N Engl J Med. 2018;378(5):439–448. doi:10.1056/NEJMoa1709866
  • European Medicines Agency (EMA). Kymriah: cells dispersion for infusion [summary of product characteristics]. Available from: https://www.ema.europa.eu/en/documents/product-information/kymriah-epar-product-information_en.pdf. Accessed March 7, 2019.
  • Agencia Española de Medicamentos y Productos Sanitarios (AEMPS). Informe de Posicionamiento Terapéutico de Tisagenlecleucel (Kymriah®) [Therapeutic Positioning Report of Tisagenlecleucel]. Madrid; 2019. Available from: https://www.aemps.gob.es/medicamentosUsoHumano/informesPublicos/docs/IPT-tisagenlecleucel-kymriah-LAL-LCGB.pdf. Accessed March 4, 2019.
  • ClinicalTrials.gov. Determine efficacy and safety of CTL019 in pediatric patients with relapsed and refractory B-cell ALL (ELIANA). Available from: https://clinicaltrials.gov/ct2/show/NCT02435849. Accessed February 13, 2019.
  • ClinicalTrials.gov. Study of efficacy and safety of CTL019 in pediatric ALL patients. Available from: https://clinicaltrials.gov/ct2/show/NCT02228096. Accessed February 13, 2019.
  • ClinicalTrials.gov. Phase I/IIA study of CART19 cells for patients with chemotherapy resistant or refractory CD19+ leukemia and lymphoma (Pedi CART19). Available from: https://clinicaltrials.gov/ct2/show/NCT01626495. Accessed February 13, 2019.
  • European Medicines Agency (EMA). Kymriah (EPAR) [summary of product characteristics]. Available from: www.ema.europa.eu/en/documents/assessment-report/kymriah-epar-public-assessment-report_en.pdf. Accessed March 7, 2019.
  • López Bastida J, Oliva J, Antoñanzas F, et al. Propuesta de guía para la evaluación económica aplicada a las tecnologías sanitarias [A proposed guideline for economic evaluation of health technologies]. Gac Sanit. 2010;24(2):154–170. doi:10.1016/j.gaceta.2009.07.011
  • Ortega A, Marín R, Fraga MD, López-Briz E, Puigventós F. Guía de evaluación económica e impacto presupuestario en los informes de evaluación de medicamentos: guía práctica [Economic evaluation and budgetary impact guidelines for assessment reports on medicinal products]. Available from: https://gruposdetrabajo.sefh.es/genesis/genesis/Documents/GUIA_EE_IP_GENESIS-SEFH_19_01_2017.pdf. Accessed February 25, 2019.
  • Von Stackelberg A, Völzke E, Kühl JS, et al. Outcome of children and adolescents with relapsed acute lymphoblastic leukaemia and non-response to salvage protocol therapy: a retrospective analysis of the ALL-REZ BFM study group. Eur J Cancer. 2011;47(1):90–97. doi:10.1016/j.ejca.2010.09.020
  • MacArthur AC, Spinelli JJ, Rogers PC, Goddard KJ, Abanto ZU, McBride ML. Mortality among 5-year survivors of cancer diagnosed during childhood or adolescence in British Columbia, Canada. Pediatr Blood Cancer. 2007;48(4):460–467. doi:10.1002/pbc.20922
  • Instituto Nacional de Estadística (INE). Instituto Nacional de Estadística [homepage on the internet]. Available from: http://www.ine.es. Accessed February 25, 2019.
  • Kelly MJ, Pauker SG, Parsons SK. Using nonrandomized studies to inform complex clinical decisions: the thorny issue of cranial radiation therapy for T-cell acute lymphoblastic leukemia. Pediatr Blood Cancer. 2015;62(5):790–797. doi:10.1002/pbc.25451
  • Sung L, Buckstein R, Doyle JJ, Crump M, Detsky AS. Treatment options for patients with acute myeloid leukemia with a matched sibling donor: a decision analysis. Cancer. 2003;97(3):592–600. doi:10.1002/cncr.11098
  • Szende A, Janssen B, Cabases J, eds. Self-Reported Population Health: An International Perspective Based on EQ-5D. Dordrecht: Springer; 2014.
  • Jefatura del Estado (Spain). Real Decreto-ley (RDL) 8/2010, de 20 de mayo, por el que se adoptan medidas extraordinarias para la reducción del déficit público. Boletín Oficial del Estado. Núm. 126. Sec. I. Pág. 45070. Lunes 24 de mayo de 2010. [ Updated 2010]. Available from: https://www.boe.es/boe/dias/2010/05/24/pdfs/BOE-A-2010-8228.pdf. Accessed March 6, 2019.
  • CGCOF. Bot PLUS 2.0 [homepage on the Internet]. Base de datos del Consejo General de Colegios Oficiales de Farmacéuticos [General council of the association of official pharmacists database]. Available from: https://botplusweb.portalfarma.com/. Accessed March 19, 2019.
  • Nuño-Solinís R, Herrera Molina E, Librada Flores S, Orueta Mendía JF, Cabrera-León A. Actividad asistencial y costes en los últimos 3 meses de vida de pacientes fallecidos con cáncer en Euskadi [Care costs and activity in the last three months of life of cancer patients who died in the Basque Country (Spain)]. Gac Sanit. 2017;31(6):524–530. doi:10.1016/j.gaceta.2016.06.005
  • Ministerio de Sanidad, Servicios Sociales e Igualdad. Conjunto Mínimo Básico de Datos al alta hospitalaria [Basic minimum dataset of hospital discharge] [homepage on the internet]. Available from: http://pestadistico.inteligenciadegestion.msssi.es/publicosns. Accessed May 23, 2018.
  • Gisbert R, Brosa M eSalud: infomación económica del sector sanitario [webpage on the Internet] [eSalud: economic information of the health sector]. Barcelona: oblikue consulting, S.L, 2018. Available from: http://oblikue.com/bddcostes. Accessed July 22, 2019.
  • Raetz EA, Borowitz MJ, Devidas M, et al. Reinduction platform for children with first marrow relapse of acute lymphoblastic leukemia: a children’s oncology group study. J Clin Oncol. 2008;26(24):3971–3978. doi:10.1200/JCO.2008.16.1414
  • Maude SL, Grupp SA, Pulsipher MA, et al. Analysis of safety data from 2 multicenter trials of CTL019 in pediatric and young adult patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). European Hematology Association; 2017. Available at: https://library.ehaweb.org/eha/2017/22nd/181804/.
  • Ministerio de Sanidad Consumo y Bienestar Social. Nota informativa de la reunión de la comisión interministerial de precios de los medicamentos. [Informative note of the meeting from the Spanish interministerial medicinal products pricing committee]. Sesión 187. [ Updated 2018]. Available from: https://www.mscbs.gob.es/profesionales/farmacia/pdf/NOTA_INFORMATIVA_DE_LA_CIPM_187_web.pdf. Accessed February 24, 2020.
  • Sarkar RR, Gloude NJ, Schiff D, Murphy JD. Cost-effectiveness of chimeric antigen receptor T-cell therapy in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia. J Natl Cancer Inst. 2018;111(7):719–726. doi:10.1093/jnci/djy193
  • Whittington MD, McQueen RB, Ollendorf DA, et al. Long-term survival and value of chimeric antigen receptor T-cell therapy for pediatric patients with relapsed or refractory leukemia. JAMA Pediatr. 2018;172(12):1161–1168. doi:10.1001/jamapediatrics.2018.2530
  • Lin JK, Lerman BJ, Barnes JI, et al. Cost effectiveness of chimeric antigen receptor T-cell therapy in relapsed or refractory pediatric B-cell acute lymphoblastic leukemia. J Clin Oncol. 2018;In press. doi:10.1200/JCO.2018.79.0642
  • National Institute for Health and Care Excellence (NICE). Tisagenlecleucel for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years: technology appraisal guidance. Available from: https://www.nice.org.uk/guidance/ta554/resources/tisagenlecleucel-for-treating-relapsed-or-refractory-bcell-acute-lymphoblastic-leukaemia-in-people-aged-up-to-25-years-pdf-82607021872837. Accessed May 31, 2019.
  • Institute for Clinical and Economic Review (ICER). Chimeric antigen receptor T-cell therapy for B-cell cancers: effectiveness and value: final evidence report. Available from: https://icer-review.org/wp-content/uploads/2017/07/ICER_CAR_T_Final_Evidence_Report_032318.pdf. Accessed March 4, 2019.
  • Sacristán JA, Oliva J, Del Llano J, Prieto L, Pinto JL. ¿Qué es una tecnología sanitaria eficiente en España? [What is an efficient health technology in Spain?]. Gac Sanit. 2002;16(4):334–343. doi:10.1016/S0213-9111(02)71933-X
  • Agencia Española de Medicamentos y Productos Sanitarios (AEMPS). Informe de Posicionamiento Terapéutico de Blinatumomab (Blincyto®) [Therapeutic positioning report of blinatumomab]. Madrid; 2017. Available from: https://www.aemps.gob.es/medicamentosUsoHumano/informesPublicos/docs/IPT-blinatumomab-Blincyto-LAL.pdf. Accessed March 14, 2019.
  • Ministerio de Sanidad Consumo y Bienestar Social. Nota informativa de la reunión de la Comisión Interministerial de Precios de los Medicamentos [Informative note of the meeting from the Spanish interministerial medicinal products pricing committee]. Sesión 190. [ Updated 2019]. Available from: https://www.mscbs.gob.es/va/profesionales/farmacia/pdf/NOTA_INFORMATIVA_DE_LA_CIPM_190_web.pdf. Accessed June 11, 2019.
  • Food and Drug Administration (FDA). Highlights of prescribing information: BLINCYTO® (blinatumomab) for injection, for intravenous use Initial U.S: approval; 2014. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/125557s013lbl.pdf. Accessed March 7, 2019..
  • Brown PA, Ji L, Xu X, et al. A randomized phase 3 trial of blinatumomab vs. chemotherapy as post-reinduction therapy in High and Intermediate Risk (HR/IR) first relapse of B-Acute Lymphoblastic Leukemia (B-ALL) in Children and Adolescents/Young Adults (AYAs) demonstrates superior Eff. Blood. 2019;134(Supplement_2):LBA-1-LBA-1. doi:10.1182/blood-2019-132435
  • Von Stackelberg A, Locatelli F, Zugmaier G, et al. Phase I/Phase II study of blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia. J Clin Oncol. 2016;34(36):4381–4389. doi:10.1200/JCO.2016.67.3301
  • Locatelli F, Zugmaier G, Bader P, et al. Blinatumomab in pediatric patients with relapsed/refractory B-cell precursor and molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): updated analysis of 110 patients treated in an expanded access study (RIALTO). Blood. 2019;134(Supplement_1):1294. doi:10.1182/blood-2019-121615
  • Jeha S, Razzouk B, Rytting M, et al. Phase II study of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia. J Clin Oncol. 2009;27(26):4392–4397. doi:10.1200/JCO.2008.18.8706
  • Brosa M, García Del Muro X, Mora J, et al. Orphan drugs revisited: cost-effectiveness analysis of the addition of mifamurtide to the conventional treatment of osteosarcoma. Expert Rev Pharmacoecon Outcomes Res. 2015;15(2):331–340. doi:10.1586/14737167.2015.972378
  • Núñez M, Gil B, Sacristán J, Dilla T. Analysis of orphan drugs marketed in Spain during the period 2010–2015: epidemiological, clinical and economic characteristics. Heal Econ Outcome Res. 2017;3(3):1–10. doi:10.4172/2471-268X.1000140