{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T03:49:14Z","timestamp":1788839354233,"version":"build-2803163510"},"reference-count":49,"publisher":"Cambridge University Press (CUP)","issue":"1","license":[{"start":{"date-parts":[[2022,7,11]],"date-time":"2022-07-11T00:00:00Z","timestamp":1657497600000},"content-version":"unspecified","delay-in-days":191,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Int J Technol Assess Health Care"],"published-print":{"date-parts":[[2022]]},"abstract":"<jats:title>Abstract<\/jats:title>\n\t  <jats:sec id=\"S0266462322000356_sec1\">\n\t    <jats:title>Objectives<\/jats:title>\n\t    <jats:p>This study evaluates the cost-effectiveness of tisagenlecleucel (a CAR T-cell therapy), versus blinatumomab, for the treatment of pediatric and young adult patients with relapsed\/refractory acute lymphoblastic leukemia (R\/R ALL) in the Irish healthcare setting. The value of conducting further research, to investigate the value of uncertainty associated with the decision problem, is assessed by means of expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462322000356_sec2\" sec-type=\"methods\">\n\t    <jats:title>Methods<\/jats:title>\n\t    <jats:p>A three-state partitioned survival model was developed. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 60\u00a0months; general population mortality with a standardized mortality ratio was then applied. Estimated EVPI and EVPPI were scaled up to population according to the incidence of the decision.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462322000356_sec3\" sec-type=\"results\">\n\t    <jats:title>Results<\/jats:title>\n\t    <jats:p>At list prices, the incremental cost-effectiveness ratio was EUR 73,086 per quality-adjusted life year (QALY) (incremental costs EUR 156,928; incremental QALYs 2.15). The probability of cost-effectiveness, at the willingness-to-pay threshold of EUR 45,000 per QALY, was 16 percent. At this threshold, population EVPI was EUR 314,455; population EVPPI was below EUR 100,000 for each parameter category.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462322000356_sec4\" sec-type=\"conclusions\">\n\t    <jats:title>Conclusions<\/jats:title>\n\t    <jats:p>Tisagenlecleucel is not cost effective, versus blinatumomab, for the treatment of pediatric and young adult patients with R\/R ALL in Ireland (at list prices). Further research to decrease decision (parameter) uncertainty, at the defined willingness-to-pay threshold, may not be of value. However, there is a high degree of uncertainty underpinning the analysis, which may not be captured by EVPI analysis.<\/jats:p>\n\t  <\/jats:sec>","DOI":"10.1017\/s0266462322000356","type":"journal-article","created":{"date-parts":[[2022,7,11]],"date-time":"2022-07-11T08:23:12Z","timestamp":1657527792000},"source":"Crossref","is-referenced-by-count":13,"title":["Tisagenlecleucel for relapsed\/refractory acute lymphoblastic leukemia in the Irish healthcare setting: cost-effectiveness and value of information analysis"],"prefix":"10.1017","volume":"38","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6174-9718","authenticated-orcid":false,"given":"Niamh","family":"Carey","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joy","family":"Leahy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lea","family":"Trela-Larsen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"McCullagh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Barry","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"56","published-online":{"date-parts":[[2022,7,11]]},"reference":[{"key":"S0266462322000356_r44","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1177\/0272989X13505910","article-title":"Estimating multiparameter partial expected value of perfect information from a probabilistic sensitivity analysis sample:A nonparametric regression approach","volume":"34","author":"Strong","year":"2014","journal-title":"Med Decis Mak."},{"key":"S0266462322000356_r23","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.14965"},{"key":"S0266462322000356_r42","doi-asserted-by":"publisher","DOI":"10.1177\/0272989X07302555"},{"key":"S0266462322000356_r1","doi-asserted-by":"crossref","first-page":"e202","DOI":"10.1016\/S2352-3026(17)30052-2","article-title":") Worldwide comparison of survival from childhood leukaemia for 1995-2009, by subtype, age, and sex (CONCORD-2): A population-based study of individual data for 89 828 children from 198 registries in 53 countries","volume":"4","author":"Bonaventure","year":"2017","journal-title":"Lancet Haematol."},{"key":"S0266462322000356_r2","unstructured":"2. National Cancer Registry Ireland (NCRI) (2019) Number of cases diagnosed, incidence rates, projected case counts and survival for acute lymphoblastic leukaemia."},{"key":"S0266462322000356_r7","unstructured":"7. European Medicines Agency (EMA) (2020) Kymriah: EPAR public assessment report update."},{"key":"S0266462322000356_r34","unstructured":"34. Ernst & Young LLP (2021) Analysis of hospital activity and costs following allogeneic stem cell transplantation in England. London."},{"key":"S0266462322000356_r4","doi-asserted-by":"crossref","first-page":"648","DOI":"10.1200\/JCO.2009.22.2950","article-title":"Outcome of patients treated for relapsed or refractory acute lymphoblastic leukemia: A therapeutic advances in childhood leukemia consortium study","volume":"28","author":"Ko","year":"2010","journal-title":"J Clin Oncol."},{"key":"S0266462322000356_r20","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1186\/1471-2288-12-9","article-title":"Enhanced secondary analysis of survival data: Reconstructing the data from published Kaplan-Meier survival curves","volume":"12","author":"Guyot","year":"2012","journal-title":"BMC Med Res Methodol."},{"key":"S0266462322000356_r12","volume-title":"Guidelines for the economic evaluation of health technologies in Ireland","year":"2020"},{"key":"S0266462322000356_r26","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1177\/0272989X17732990","article-title":"A systematic review and meta-analysis of childhood health utilities","volume":"38","author":"Kwon","year":"2018","journal-title":"Med Decis Making."},{"key":"S0266462322000356_r16","unstructured":"16. 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