Cost-utility and value of information analysis of tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma in the Irish healthcare setting

References

• Chaganti S, Illidge T, Barrington S, et al. Guidelines for the management of diffuse large B-cell lymphoma. Br J Haematol. 2016;174(1):43–12. DOI:10.1111/bjh.14136
   PubMed Web of Science ®Google Scholar
• Friedberg JW, Fisher RI. Diffuse large B-cell lymphoma. Hematol Oncol Clin North Am. 2008;22(5): 941–952. ix.
   PubMed Web of Science ®Google Scholar
• National Cancer Registry Ireland (NCRI). Number of cases diagnosed, incidence rates, projected case counts and survival for diffuse large B-cell lymphoma. 2019.
   Google Scholar
• Feugier P, Van Hoof A, Sebban C, et al. Long-term results of the R-CHOP study in the treatment of elderly patients with diffuse large B-cell lymphoma: a study by the groupe d’Etude des lymphomes de l’adulte. J Clin Oncol. 2005;23(18):4117–4126. DOI:10.1200/JCO.2005.09.131
   PubMed Web of Science ®Google Scholar
• Den Neste E V, Schmitz N, Mounier N, et al. Outcome of patients with relapsed diffuse large B-cell lymphoma who fail second-line salvage regimens in the International CORAL study. Bone Marrow Transplant. 2016;51(1):51–57. DOI:10.1038/bmt.2015.213
   PubMed Web of Science ®Google Scholar
• Den Neste E V, Schmitz N, Mounier N, et al. Outcomes of diffuse large B-cell lymphoma patients relapsing after autologous stem cell transplantation: an analysis of patients included in the CORAL study. Bone Marrow Transplant. 2017;52(2):216–221. DOI:10.1038/bmt.2016.213
   PubMed Web of Science ®Google Scholar
• Crump M, Neelapu SS, Farooq U, et al. Outcomes in refractory diffuse large B-cell lymphoma: results from the international SCHOLAR-1 study. Blood. 2017;130(16):1800–1808. DOI:10.1182/blood-2017-03-769620
   PubMed Web of Science ®Google Scholar
• Kymriah 1.2x106 - 6x108 cells dispersion for infusion: summary of product characteristics 2021. Available from: https://www.medicines.ie/medicines/kymriah-1-2-x-106-6-x-108-cells-dispersion-for-infusion-34945/spc.
   Google Scholar
• Sterner RC, Sterner RM. CAR-T cell therapy: current limitations and potential strategies. Blood Cancer J. 2021;11(4):69.
   PubMed Web of Science ®Google Scholar
• Wakase S, Teshima T, Zhang J, et al. Cost effectiveness analysis of tisagenlecleucel for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma in Japan. Transplant Cell Ther. 2021;27(6):506.e1–506.e10. DOI:10.1016/j.jtct.2021.03.005
   PubMedGoogle Scholar
• European Medicines Agency (EMA). Kymriah: EPAR -public assessment report. 2018.
   Google Scholar
• Institute for Clinical and Economic Review (ICER). Chimeric antigen receptor T-cell therapy for B-cell cancers: effectiveness and value. 2018.
   Google Scholar
• Schuster SJ, Bishop MR, Tam C, et al. Sustained disease control for adult patients with relapsed or refractory diffuse large B-cell lymphoma: an updated analysis of juliet, a global pivotal phase 2 trial of tisagenlecleucel. Blood. 2018;132(Supplement 1):1684. DOI:10.1182/blood-2018-99-115252
   Google Scholar
• Maurer MJ, Ghesquières H, Jais J-P, et al. Event-free survival at 24 months is a robust end point for disease-related outcome in diffuse large B-cell lymphoma treated with immunochemotherapy. J Clin Oncol. 2014;32(10):1066–1073. DOI:10.1200/JCO.2013.51.5866
   PubMed Web of Science ®Google Scholar
• Howlader N, Mariotto AB, Besson C, et al. Cancer-specific mortality, cure fraction, and noncancer causes of death among diffuse large B-cell lymphoma patients in the immunochemotherapy era. Cancer. 2017;123(17):3326–3334. DOI:10.1002/cncr.30739
   PubMed Web of Science ®Google Scholar
• Jakobsen LH, Bøgsted M, Brown PN, et al. Minimal loss of lifetime for patients with diffuse large B-cell lymphoma in remission and event free 24 months after treatment: a danish population-based study. J Clin Oncol. 2017;35(7):778–784. DOI:10.1200/JCO.2016.70.0765
   PubMed Web of Science ®Google Scholar
• Health Information and Quality Authority (HIQA). Guidelines for the Economic Evaluation of Health Technologies in Ireland. Dublin: HIQA: 2020.
   Google Scholar
• Schuster SJ, Bishop MR, Tam CS, et al. Tisagenlecleucel in adult relapsed or refractory diffuse large B-cell lymphoma. N Engl J Med. 2018;380(1):45–56. DOI:10.1056/NEJMoa1804980
   PubMed Web of Science ®Google Scholar
• Liu R, Oluwole OO, Diakite I, et al. Cost effectiveness of axicabtagene ciloleucel versus tisagenlecleucel for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy in the United States. J Med Econ. 2021;24(1):458–468.
   PubMed Web of Science ®Google Scholar
• The Canadian Agency for Drugs and Technologies in Health (CADTH). Tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma. Economic Review Report. Ottawa: CADTH; 2019.
   Google Scholar
• National Centre for Pharmacoeconomics (NCPE). Tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy: summary. 2019.
   Google Scholar
• National Centre for Pharmacoeconomics (NCPE). Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL) after two or more lines of systemic therapy: technical summary. 2019.
   Google Scholar
• National Cancer Control Programme (NCCP). Rituximab, Gemcitabine, Dexamethasone and CISplatin ((R)-GDP) Therapy. 2019.
   Google Scholar
• Guyot P, Ades AE, MJNM O, et al. Enhanced secondary analysis of survival data: reconstructing the data from published Kaplan-Meier survival curves. BMC Med Res Methodol. 2012;12(1):9.
   PubMed Web of Science ®Google Scholar
• Digitizelt Digitizer S. 2020 [cited 2020 Apr 04]. Available from: https://www.digitizeit.xyz/.
   Google Scholar
• Latimer N. NICE DSU Technical Support Document 14: Undertaking survival analysis for economic evaluations alongside clinical trials - extrapolation with patient-level data. University of Sheffield: Decision Support Unit; 2011. Available from: http://nicedsu.org.uk/.
   Google Scholar
• Hettle R, Corbett M, Hinde S, et al. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal. Health Technol Assess. 2017;21(7):1–204. DOI:10.3310/hta21070
   PubMed Web of Science ®Google Scholar
• Jaeger U, Bishop MR, Salles G, et al. Myc expression and tumor-infiltrating T cells are associated with response in patients (pts) with relapsed/refractory diffuse large B-Cell lymphoma (r/r DLBCL) treated with tisagenlecleucel in the juliet trial. Blood. 2020;136(Supplement 1):48–49. DOI:10.1182/blood-2020-137045
   Google Scholar
• Norwegian Medicines Agency (NoMA). Single technology assessment: tisagenlecleucel (Kymriah) for the treatment of second or later relapsed/refractory diffuse large B-cell lymphoma (DLBCL). 2019.
   Google Scholar
• Gisselbrecht C, Schmitz N, Mounier N, et al. Rituximab maintenance therapy after autologous stem-cell transplantation in patients with relapsed CD20+ diffuse large B-cell lymphoma: final analysis of the collaborative trial in relapsed aggressive lymphoma. J Clin Oncol. 2012;30(36):4462–4469. DOI:10.1200/JCO.2012.41.9416
   PubMed Web of Science ®Google Scholar
• Gisselbrecht C, Glass B, Mounier N, et al. Salvage regimens with autologous transplantation for relapsed large B-cell lymphoma in the rituximab era. J Clin Oncol. 2010;28(27):4184–4190. DOI:10.1200/JCO.2010.28.1618
   PubMed Web of Science ®Google Scholar
• Maziarz RT, Waller EK, Jaeger U, et al. Patient-reported long-term quality of life after tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma. Blood Adv. 2020;4(4):629–637. DOI:10.1182/bloodadvances.2019001026
   PubMed Web of Science ®Google Scholar
• Guadagnolo BA, Punglia RS, Kuntz KM, et al. Cost-effectiveness analysis of computerized tomography in the routine follow-up of patients after primary treatment for Hodgkin’s disease. J Clin Oncol. 2006;24(25):4116–4122.
   PubMed Web of Science ®Google Scholar
• Corbett M, Duarte A, Walker S, et al. Tisagenlecleucel for treating relapsed or refractory diffuse large B-cell lymphoma: a single technology appraisal. University of York: University of York Technology Assessment Group; 2018.
   Google Scholar
• Lloyd A, Nafees B, Narewska J, et al. Health state utilities for metastatic breast cancer. Br j cancer. 2006;95(6):683–690.
   PubMed Web of Science ®Google Scholar
• Healthcare Pricing Office (HPO). ABF 2020 admitted patient price list: dRG prices for inpatients and daycases 2020. 2020.
   Google Scholar
• Thielen FW, van Dongen-Leunis A, Arons AMM, et al. Cost-effectiveness of anti-CD19 chimeric antigen receptor T-cell therapy in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia. A societal view. Eur J Haematol. 2020;105(2):203–215.
   PubMed Web of Science ®Google Scholar
• Ernst&Young LLP. Analysis of hospital activity and costs following allogeneic stem cell transplantation in England. London: 2021.
   Google Scholar
• Bourke S, Burns RM, Gaynor C. Challenges in generating costs and utilisation rates associated with castration-resistant prostate cancer. J Mark Access Health Policy. 2014;2(1):10.3402/jmahp.v2.24072.
   PubMedGoogle Scholar
• Ara R, Brazier JE. Populating an economic model with health state utility values: moving toward better practice. Value Health. 2010;13(5):509–518.
   PubMed Web of Science ®Google Scholar
• Central Statistics Office (CSO). Consumer price index 2020. Available from: https://data.cso.ie/.
   Google Scholar
• Organisation for Economic Co-operation and Development (OECD). Purchasing power parities (PPP) 2020. Available from: https://data.oecd.org/conversion/purchasing-power-parities-ppp.htm.
   Google Scholar
• Tilly H, Gomes da Silva M, Vitolo U, et al. Diffuse large B-cell lymphoma (DLBCL): eSMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2015;26(Suppl 5):v116–25. DOI:10.1093/annonc/mdv304
   PubMed Web of Science ®Google Scholar
• Ibrahim Y-A, Christian C, Peter B, et al. Management of adults and children undergoing chimeric antigen receptor T-cell therapy: best practice recommendations of the european society for blood and marrow transplantation (EBMT) and the joint accreditation committee of ISCT and EBMT (JACIE). Haematologica. 2020;105(2):297–316. DOI:10.3324/haematol.2019.229781
   PubMed Web of Science ®Google Scholar
• Crump M, Kuruvilla J, Couban S, et al. Randomized comparison of gemcitabine, dexamethasone, and cisplatin versus dexamethasone, cytarabine, and cisplatin chemotherapy before autologous stem-cell transplantation for relapsed and refractory aggressive lymphomas: nCIC-CTG LY.12. J Clin Oncol. 2014;32(31):3490–3496. DOI:10.1200/JCO.2013.53.9593
   PubMed Web of Science ®Google Scholar
• National Centre for Pharmacoeconomics (NCPE). About us. Health Technology Assessment. 2021 [cited 2022 Mar 03]. Available from: https://www.ncpe.ie/about/.
   Google Scholar
• McCullagh L, Barry M. The pharmacoeconomic evaluation process in Ireland. Pharmacoeconomics. 2016;34(12):1267–1276.
   PubMed Web of Science ®Google Scholar
• Brennan A, Kharroubi S, O’Hagan A, et al. Calculating partial expected value of perfect information via monte carlo sampling algorithms. Med Decis Making. 2007;27(4):448–470.
   PubMed Web of Science ®Google Scholar
• Wood S Mgcv: mixed GAM computation vehicle with automatic smoothness estimation. 2021 [cited 2021 Nov 05]. Available from: https://cran.r-project.org/web/packages/mgcv/index.html.
   Google Scholar
• Strong M. R code for partial EVPI section of public health, school of health and related research. University of Sheffield; [cited 2021 Sept 28]. Available from: https://www.sheffield.ac.uk/scharr/people/staff/mark-strong.
   Google Scholar
• National Institute for Health and Care Excellence (NICE). CHTE methods review: discounting - task and finish group report. 2020.
   Google Scholar
• Gravelle H, Smith D. Discounting for health effects in cost-benefit and cost-effectiveness analysis. Health Econ. 2001;10(7):587–599.
   PubMed Web of Science ®Google Scholar
• Norwegian Medicines Agency (NoMA). Single technology assessment: axicabtagene ciloleucel (Yescarta) for the treatment of second or later relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL). 2018.
   Google Scholar
• Lin VW, Blaylock B, Epstein J, et al. Systematic literature review of health-related quality of life among aggressive non-Hodgkin lymphoma survivors. Curr Med Res Opin. 2018;34(8):1529–1535.
   PubMed Web of Science ®Google Scholar
• Lin V, Jiang Y, Chuang L, et al. Health utilities for patients with relapsed or refractory large B-cell lymphoma (R/R-LBCL): ad hoc analysis from an axicabtagene ciloleucel (axi-cel) safety managenebt study. Bone Marrow Transplant. 2018;53:878–887.
   Web of Science ®Google Scholar
• Qi CZ, Bollu V, Yang H, et al. Cost-effectiveness analysis of tisagenlecleucel for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma in the United States. Clin Ther. 2021;43(8):1300–1319.e8.
   PubMed Web of Science ®Google Scholar
• Moradi-Lakeh M, Yaghoubi M, Seitz P, et al. Cost-effectiveness of tisagenlecleucel in paediatric acute lymphoblastic leukaemia (pALL) and adult diffuse large B-cell lymphoma (DLBCL) in Switzerland. Adv Ther. 2021;38(6):3427–3443.
   PubMed Web of Science ®Google Scholar
• Rutherford SC, Leonard JP. Lymphoma “benchmark” or “bench-smudge”? Blood. 2017;130(16):1778–1779.
   PubMed Web of Science ®Google Scholar
• Sermer D, Batlevi C, Palomba ML, et al. Outcomes in patients with DLBCL treated with commercial CAR T cells compared with alternate therapies. Blood Adv. 2020;4(19):4669–4678. DOI:10.1182/bloodadvances.2020002118
   PubMed Web of Science ®Google Scholar
• Cher BP, Gan KY, Aziz MIA, et al. Cost utility analysis of tisagenlecleucel vs salvage chemotherapy in the treatment of relapsed/refractory diffuse large B-cell lymphoma from Singapore’s healthcare system perspective. J Med Econ. 2020;23(11):1321–1329. DOI:10.1080/13696998.2020.1808981
   PubMed Web of Science ®Google Scholar
• Rahman R, Ventz S, McDunn J, et al. Leveraging external data in the design and analysis of clinical trials in neuro-oncology. Lancet Oncol. 2021;22(10):e456–65. DOI:10.1016/S1470-2045(21)00488-5
   PubMed Web of Science ®Google Scholar
• Philips Z, Claxton K, Palmer S. The half-life of truth: what are appropriate time horizons for research decisions? Med Decis Making. 2008;28(3):287–299.
   PubMed Web of Science ®Google Scholar