{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,16]],"date-time":"2026-05-16T06:55:46Z","timestamp":1778914546518,"version":"3.51.4"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,1,20]],"date-time":"2021-01-20T00:00:00Z","timestamp":1611100800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100002702","name":"Aalborg Universitet","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002702","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009708","name":"Novo Nordisk Fonden","doi-asserted-by":"publisher","award":["NNF15SA0018404"],"award-info":[{"award-number":["NNF15SA0018404"]}],"id":[{"id":"10.13039\/501100009708","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2021,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Purpose<\/jats:title><jats:p>Personalized therapies are leading to an increasing number of marketing authorizations based on single\u2010arm trials, which increases the demand for better post\u2010authorization monitoring strategies. The aim of the present study was to estimate the power over time as data accrue in population\u2010based registries for detecting deviations from the expected efficacy\/safety of chimeric antigen receptor T cell (CAR\u2010T) therapy approved for relapsed\/refractory large B\u2010cell lymphoma (RR\u2010LBCL).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The number of real\u2010world RR\u2010LBCL patients was projected over time in a general population of 5, 15, and 25 million citizens using lymphoma registry data. For each scenario, we computed the power over time for detecting significant deviations in efficacy (1\u2010year overall survival [1yOS]) when comparing to historical controls (SCHOLAR\u20101 study; 1yOS, 28%) and RR\u2010LBCL patients treated with CAR\u2010T cell therapy in a single\u2010arm trial (ZUMA\u20101; 1yOS, 59%) as well as deviations in selected adverse events (grade \u22653 aphasia) from the ZUMA\u20101 trial. We assumed a 10% absolute deviation in 1yOS (efficacy) and a relative increase of 50% in grade \u22653 aphasia (safety).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Assuming a general population of 5, 15, and 25 million, the accrual time needed to achieve 80% power for detecting a significant increase over the 1yOS reported in SCHOLAR\u20101 was 9, 4, and 3\u2009years, respectively, while 80% power for detecting a significant decrease in 1yOS compared to ZUMA\u20101 required 10.5, 4.5, and 3\u2009years of data accrual, respectively. However, corresponding estimates for aphasia were &gt;20, 8, and 5\u2009years, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Projections of the statistical power for detecting important deviations in efficacy\/safety from that reported in pivotal clinical trials(s) provide critical information about the expected performance of post\u2010authorization monitoring programs.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.5195","type":"journal-article","created":{"date-parts":[[2021,1,15]],"date-time":"2021-01-15T07:20:27Z","timestamp":1610695227000},"page":"514-519","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Detecting deviations from the efficacy and safety results of single\u2010arm trials using real\u2010world data: The case of a <scp>CAR\u2010T<\/scp> cell therapy in B\u2010cell lymphoma"],"prefix":"10.1002","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6575-7686","authenticated-orcid":false,"given":"Lasse Hjort","family":"Jakobsen","sequence":"first","affiliation":[{"name":"Department of Hematology Clinical Cancer Research Center, Aalborg University Hospital  Denmark"},{"name":"Department of Clinical Medicine Aalborg University  Aalborg Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9903-5817","authenticated-orcid":false,"given":"Torbj\u00f6rn","family":"Callr\u00e9us","sequence":"additional","affiliation":[{"name":"Pharmacovigilance Research Center, Department of Drug Design and Pharmacology University of Copenhagen  Copenhagen Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0874-4744","authenticated-orcid":false,"given":"Maurizio","family":"Sessa","sequence":"additional","affiliation":[{"name":"Pharmacovigilance Research Center, Department of Drug Design and Pharmacology University of Copenhagen  Copenhagen Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4509-6707","authenticated-orcid":false,"given":"Mats","family":"Jerkeman","sequence":"additional","affiliation":[{"name":"Department of Oncology Lund University  Lund Sweden"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7029-2860","authenticated-orcid":false,"given":"Morten","family":"Andersen","sequence":"additional","affiliation":[{"name":"Pharmacovigilance Research Center, Department of Drug Design and Pharmacology University of Copenhagen  Copenhagen Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4406-380X","authenticated-orcid":false,"given":"Tarec Christoffer","family":"El\u2010Galaly","sequence":"additional","affiliation":[{"name":"Department of Hematology Clinical Cancer Research Center, Aalborg University Hospital  Denmark"},{"name":"Department of Clinical Medicine Aalborg University  Aalborg Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,1,20]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2018.09.003"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1080\/10428190701809156"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.1586"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.1638"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1707447"},{"key":"e_1_2_7_7_1","unstructured":"Yescarta.EPAR \u2010 Product Information. 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