{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T05:42:51Z","timestamp":1779169371035,"version":"3.51.4"},"reference-count":53,"publisher":"Elsevier BV","issue":"7","license":[{"start":{"date-parts":[[2024,7,1]],"date-time":"2024-07-01T00:00:00Z","timestamp":1719792000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,7,1]],"date-time":"2024-07-01T00:00:00Z","timestamp":1719792000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,7,1]],"date-time":"2025-07-01T00:00:00Z","timestamp":1751328000000},"content-version":"vor","delay-in-days":365,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","valueinhealthjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Value in Health"],"published-print":{"date-parts":[[2024,7]]},"DOI":"10.1016\/j.jval.2024.03.010","type":"journal-article","created":{"date-parts":[[2024,3,26]],"date-time":"2024-03-26T17:10:46Z","timestamp":1711473046000},"page":"926-935","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":8,"title":["Tackling Challenges in Assessing the Economic Value of Tumor-Agnostic Therapies: A Cost-Effectiveness Analysis of Pembrolizumab as a Case Study"],"prefix":"10.1016","volume":"27","author":[{"given":"Yilin","family":"Chen","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter","family":"Martin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lurdes Y.T.","family":"Inoue","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anirban","family":"Basu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Josh J.","family":"Carlson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"9","key":"10.1016\/j.jval.2024.03.010_bib1","doi-asserted-by":"crossref","first-page":"975","DOI":"10.1200\/JCO.2014.59.8433","article-title":"Basket trials and the evolution of clinical trial design in an era of genomic medicine","volume":"33","author":"Redig","year":"2015","journal-title":"J\u00a0Clin Oncol"},{"issue":"36","key":"10.1016\/j.jval.2024.03.010_bib3","doi-asserted-by":"crossref","first-page":"3472","DOI":"10.1200\/JCO.18.02320","article-title":"Basket cases: how real-world testing for drugs Approved based on basket trials might lead to false diagnoses, patient risks, and Squandered resources","volume":"37","author":"Ramsey","year":"2019","journal-title":"J\u00a0Clin Oncol Off J Am Soc Clin Oncol"},{"key":"10.1016\/j.jval.2024.03.010_bib4","doi-asserted-by":"crossref","first-page":"S469","DOI":"10.1016\/j.jval.2020.08.399","article-title":"PCN262 understanding the challenges in the evaluation of tumour-agnostic therapies: barriers posed by the current trial designs and future trends","volume":"23","author":"Delimpaltadakis","year":"2020","journal-title":"Value Health"},{"key":"10.1016\/j.jval.2024.03.010_bib6","series-title":"Preparing health systems for tumour-agnostic treatment. AT Kearney Analysis","author":"Thomas","year":"2019"},{"issue":"3","key":"10.1016\/j.jval.2024.03.010_bib7","doi-asserted-by":"crossref","first-page":"583","DOI":"10.3390\/cancers13030583","article-title":"Bringing onco-innovation to Europe\u2019s healthcare systems: the potential of biomarker testing, real world evidence, tumour agnostic therapies to empower personalised medicine","volume":"13","author":"Horgan","year":"2021","journal-title":"Cancers"},{"issue":"6","key":"10.1016\/j.jval.2024.03.010_bib8","doi-asserted-by":"crossref","first-page":"1002","DOI":"10.1016\/j.jval.2021.11.1354","article-title":"Comparison of alternative methods to assess the cost-effectiveness of tumor-agnostic therapies: a triangulation approach using larotrectinib as a case study","volume":"25","author":"Briggs","year":"2022","journal-title":"Value Health"},{"issue":"6","key":"10.1016\/j.jval.2024.03.010_bib9","doi-asserted-by":"crossref","DOI":"10.1136\/bmjopen-2016-011666","article-title":"Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999-2014","volume":"6","author":"Hatswell","year":"2016","journal-title":"BMJ Open"},{"key":"10.1016\/j.jval.2024.03.010_bib10","doi-asserted-by":"crossref","first-page":"457","DOI":"10.2147\/CLEP.S242097","article-title":"Synthetic and external controls in clinical trials\u2013a primer for researchers","volume":"12","author":"Thorlund","year":"2020","journal-title":"Clin Epidemiol"},{"key":"10.1016\/j.jval.2024.03.010_bib11","doi-asserted-by":"crossref","first-page":"1135","DOI":"10.2147\/CLEP.S373291","article-title":"Oncology drug effectiveness from electronic health record data calibrated against RCT evidence: the PARSIFAL trial emulation","volume":"14","author":"Merola","year":"2022","journal-title":"Clin Epidemiol"},{"issue":"6","key":"10.1016\/j.jval.2024.03.010_bib12","doi-asserted-by":"crossref","first-page":"544","DOI":"10.1016\/j.annemergmed.2010.01.002","article-title":"The importance of \u201cshrinkage\u201d in subgroup analyses","volume":"55","author":"Lipsky","year":"2010","journal-title":"Ann Emerg Med"},{"issue":"9","key":"10.1016\/j.jval.2024.03.010_bib13","doi-asserted-by":"crossref","first-page":"929","DOI":"10.1016\/j.annonc.2022.05.519","article-title":"Pembrolizumab in microsatellite instability high or mismatch repair deficient cancers: updated analysis from the phase II KEYNOTE-158 study","volume":"33","author":"Maio","year":"2022","journal-title":"Ann Oncol"},{"issue":"1","key":"10.1016\/j.jval.2024.03.010_bib14","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1200\/JCO.19.02105","article-title":"Efficacy of pembrolizumab in patients with noncolorectal high microsatellite instability\/mismatch repair\u2013deficient cancer: results from the phase II KEYNOTE-158 study","volume":"38","author":"Marabelle","year":"2020","journal-title":"J\u00a0Clin Oncol"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib15","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1177\/0272989X211029556","article-title":"Improving the estimation of subgroup effects for clinical trial participants with multimorbidity by incorporating drug class-level information in Bayesian hierarchical models: a simulation study","volume":"42","author":"Hannigan","year":"2022","journal-title":"Med Decis Mak"},{"issue":"6","key":"10.1016\/j.jval.2024.03.010_bib16","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1161\/CIRCOUTCOMES.111.960724","article-title":"Bayesian hierarchical modeling and the integration of heterogeneous information on the effectiveness of cardiovascular therapies","volume":"4","author":"Kwok","year":"2011","journal-title":"Circ Cardiovasc Qual Outcomes"},{"issue":"19","key":"10.1016\/j.jval.2024.03.010_bib17","doi-asserted-by":"crossref","first-page":"3148","DOI":"10.1200\/JCO.2008.20.5054","article-title":"Phase II multicenter trial of imatinib in 10 histologic subtypes of sarcoma using a bayesian hierarchical statistical model","volume":"27","author":"Chugh","year":"2009","journal-title":"J\u00a0Clin Oncol"},{"issue":"5","key":"10.1016\/j.jval.2024.03.010_bib18","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1007\/s40258-022-00740-1","article-title":"Economic evaluation of a tumour-agnostic therapy: Dutch economic value of Larotrectinib in TRK fusion-positive cancers","volume":"20","author":"Michels","year":"2022","journal-title":"Appl Health Econ Health Policy"},{"issue":"9","key":"10.1016\/j.jval.2024.03.010_bib19","doi-asserted-by":"crossref","first-page":"796","DOI":"10.1056\/NEJMp1405158","article-title":"Updating cost-effectiveness\u2014the curious resilience of the $50,000-per-QALY threshold","volume":"371","author":"Neumann","year":"2014","journal-title":"N\u00a0Engl J Med"},{"issue":"1","key":"10.1016\/j.jval.2024.03.010_bib20","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1016\/j.jval.2021.11.1351","article-title":"Consolidated health economic evaluation reporting standards 2022 (CHEERS 2022) statement: updated reporting guidance for health economic evaluations","volume":"25","author":"Husereau","year":"2022","journal-title":"Value Health J Int Soc Pharmacoecon Outcomes Res"},{"issue":"1","key":"10.1016\/j.jval.2024.03.010_bib21","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1200\/JCO.19.02107","article-title":"Phase II open-label study of pembrolizumab in treatment-refractory, microsatellite instability\u2013high\/mismatch repair\u2013deficient metastatic colorectal cancer: KEYNOTE-164","volume":"38","author":"Le","year":"2020","journal-title":"J\u00a0Clin Oncol"},{"issue":"7","key":"10.1016\/j.jval.2024.03.010_bib22","doi-asserted-by":"crossref","first-page":"752","DOI":"10.1200\/JCO.21.01874","article-title":"Pembrolizumab in patients with microsatellite instability\u2013high advanced endometrial cancer: results from the KEYNOTE-158 study","volume":"40","author":"O\u2019Malley","year":"2022","journal-title":"J\u00a0Clin Oncol"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib23","doi-asserted-by":"crossref","first-page":"147","DOI":"10.36469\/9896","article-title":"A\u00a0criterion-based approach for the systematic and transparent extrapolation of clinical trial survival data","volume":"2","author":"Haines","year":"2015","journal-title":"J\u00a0Health Econ Outcomes Res"},{"key":"10.1016\/j.jval.2024.03.010_bib24","doi-asserted-by":"crossref","unstructured":"Chen Y, Carlson JJ, Montano-Campos F, Basu A, Inoue LY. Tumor-specific decisions using tumor-agnostic evidence from basket trials: a bayesian hierarchical approach. medRxiv. Published September 21,\u00a02023.","DOI":"10.1101\/2023.09.19.23295807"},{"key":"10.1016\/j.jval.2024.03.010_bib25","series-title":"United States Life Tables","author":"Arias","year":"2020"},{"issue":"15","key":"10.1016\/j.jval.2024.03.010_bib27","doi-asserted-by":"crossref","first-page":"1865","DOI":"10.2217\/fon-2020-1041","article-title":"Defining treatment regimens and lines of therapy using real-world data in oncology","volume":"17","author":"Hess","year":"2021","journal-title":"Future Oncol"},{"issue":"11","key":"10.1016\/j.jval.2024.03.010_bib28","doi-asserted-by":"crossref","first-page":"1935","DOI":"10.1080\/03007995.2022.2112872","article-title":"Real-world utilization and outcomes of systemic therapy among patients with advanced or recurrent endometrial cancer in the United States","volume":"38","author":"Liu","year":"2022","journal-title":"Curr Med Res Opin"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib29","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1097\/MPA.0000000000001479","article-title":"Real-world dosing patterns and outcomes of patients with metastatic pancreatic cancer treated with a liposomal irinotecan regimen in the United States","volume":"49","author":"Barzi","year":"2020","journal-title":"Pancreas"},{"issue":"4","key":"10.1016\/j.jval.2024.03.010_bib30","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/j.clgc.2019.12.019","article-title":"Treatment patterns and outcomes in patients with metastatic castration-resistant prostate cancer in a real-world clinical practice setting in the United States","volume":"18","author":"George","year":"2020","journal-title":"Clin Genitourin Cancer"},{"issue":"8","key":"10.1016\/j.jval.2024.03.010_bib31","doi-asserted-by":"crossref","first-page":"e58","DOI":"10.1097\/MLR.0b013e31828fad9f","article-title":"Algorithm for identifying chemotherapy\/biological regimens for metastatic colon cancer in SEER-Medicare","volume":"53","author":"Bikov","year":"2015","journal-title":"Med Care"},{"issue":"7","key":"10.1016\/j.jval.2024.03.010_bib32","doi-asserted-by":"crossref","first-page":"893","DOI":"10.1177\/0272989X221096070","article-title":"Approaches to selecting \u201ctime zero\u201d in external control arms with multiple potential entry points: a simulation study of 8 approaches","volume":"42","author":"Hatswell","year":"2022","journal-title":"Med Decis Mak"},{"key":"10.1016\/j.jval.2024.03.010_bib33","author":"Book"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib35","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1093\/jnci\/djq495","article-title":"Projections of the cost of cancer care in the United States: 2010-2020","volume":"103","author":"Mariotto","year":"2011","journal-title":"J\u00a0Natl Cancer Inst"},{"issue":"6","key":"10.1016\/j.jval.2024.03.010_bib36","doi-asserted-by":"crossref","first-page":"1294","DOI":"10.1002\/(SICI)1097-0142(20000315)88:6<1294::AID-CNCR4>3.0.CO;2-M","article-title":"Quality of life in survivors of colorectal carcinoma","volume":"88","author":"Ramsey","year":"2000","journal-title":"Cancer"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib37","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1016\/j.jval.2012.11.001","article-title":"Cost-effectiveness of cetuximab, cetuximab plus irinotecan, and panitumumab for third and further lines of treatment for KRAS wild-type patients with metastatic colorectal cancer","volume":"16","author":"Hoyle","year":"2013","journal-title":"Value Health J Int Soc Pharmacoecon Outcomes Res"},{"issue":"9","key":"10.1016\/j.jval.2024.03.010_bib38","doi-asserted-by":"crossref","first-page":"1273","DOI":"10.1038\/bjc.2011.390","article-title":"Cost-effectiveness analysis of trastuzumab to treat HER2-positive advanced gastric cancer based on the randomised ToGA trial","volume":"105","author":"Shiroiwa","year":"2011","journal-title":"Br J Cancer"},{"issue":"1","key":"10.1016\/j.jval.2024.03.010_bib39","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1007\/s12029-016-9902-9","article-title":"Health-related quality of life in patients with metastatic pancreatic cancer","volume":"48","author":"Picozzi","year":"2017","journal-title":"J\u00a0Gastrointest Cancer"},{"issue":"4","key":"10.1016\/j.jval.2024.03.010_bib40","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1016\/j.jval.2013.01.011","article-title":"Cost-effectiveness of trabectedin plus pegylated liposomal doxorubicin for the treatment of women with relapsed platinum-sensitive ovarian cancer in the UK: analysis based on the final survival data of the OVA-301 trial","volume":"16","author":"Fisher","year":"2013","journal-title":"Value Health J Int Soc Pharmacoecon Outcomes Res"},{"issue":"4","key":"10.1016\/j.jval.2024.03.010_bib44","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1007\/s40273-013-0033-x","article-title":"A\u00a0systematic review of utility values for chemotherapy-related adverse events","volume":"31","author":"Shabaruddin","year":"2013","journal-title":"Pharmacoeconomics"},{"issue":"7","key":"10.1016\/j.jval.2024.03.010_bib41","first-page":"e667","article-title":"Health state utilities for patients with brain metastases","volume":"8","author":"Lester-Coll","year":"2016","journal-title":"Cureus"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib42","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1002\/cncr.31795","article-title":"Cost-effectiveness of immune checkpoint inhibitors for microsatellite instability-high\/mismatch repair-deficient metastatic colorectal cancer","volume":"125","author":"Chu","year":"2019","journal-title":"Cancer"},{"issue":"1","key":"10.1016\/j.jval.2024.03.010_bib47","doi-asserted-by":"crossref","first-page":"7","DOI":"10.3322\/caac.21708","article-title":"Cancer statistics, 2022","volume":"72","author":"Siegel","year":"2022","journal-title":"CA Cancer J Clin"},{"issue":"10","key":"10.1016\/j.jval.2024.03.010_bib50","doi-asserted-by":"crossref","first-page":"e430","DOI":"10.1016\/S1470-2045(21)00337-5","article-title":"Basket clinical trial design for targeted therapies for cancer: a French National Authority for Health statement\u00a0for health technology assessment","volume":"22","author":"Lenglin\u00e9","year":"2021","journal-title":"Lancet Oncol"},{"key":"10.1016\/j.jval.2024.03.010_bib51","doi-asserted-by":"crossref","first-page":"l6435","DOI":"10.1136\/bmj.l6435","article-title":"How should we assess the clinical and cost effectiveness of histology independent cancer drugs?","volume":"368","author":"Cooper","year":"2020","journal-title":"BMJ"},{"issue":"10","key":"10.1016\/j.jval.2024.03.010_bib52","doi-asserted-by":"crossref","first-page":"1063","DOI":"10.1007\/s40273-017-0535-z","article-title":"Performance-based risk-sharing arrangements: an updated international review","volume":"35","author":"Carlson","year":"2017","journal-title":"Pharmacoeconomics"},{"issue":"10","key":"10.1016\/j.jval.2024.03.010_bib53","first-page":"1010","article-title":"Using performance-based risk-sharing arrangements to address uncertainty in indication-based pricing","volume":"23","author":"Yeung","year":"2017","journal-title":"J\u00a0Manag Care Spec Pharm"},{"issue":"7","key":"10.1016\/j.jval.2024.03.010_bib54","doi-asserted-by":"crossref","first-page":"1022","DOI":"10.1016\/j.jval.2023.02.004","article-title":"Designing a value-based formulary for a commercial health plan: a simulated case study of diabetes medications","volume":"26","author":"Chen","year":"2023","journal-title":"Value Health"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib55","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1177\/0272989X20980327","article-title":"Exploring heterogeneity in histology-independent technologies and the implications for cost-effectiveness","volume":"41","author":"Murphy","year":"2021","journal-title":"Med Decis Mak"},{"issue":"2","key":"10.1016\/j.jval.2024.03.010_bib56","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1016\/j.jval.2022.07.009","article-title":"A\u00a0guide to selecting flexible survival models to inform economic evaluations of cancer immunotherapies","volume":"26","author":"Palmer","year":"2023","journal-title":"Value Health J Int Soc Pharmacoecon Outcomes Res"},{"issue":"10","key":"10.1016\/j.jval.2024.03.010_bib57","doi-asserted-by":"crossref","first-page":"924","DOI":"10.18632\/oncotarget.27518","article-title":"Prognostic and predictive factors in pancreatic cancer","volume":"11","author":"Dell\u2019Aquila","year":"2020","journal-title":"Oncotarget"},{"issue":"12","key":"10.1016\/j.jval.2024.03.010_bib58","first-page":"1394","article-title":"Prognostic value of microsatellite instability and p53 expression in metastatic colorectal cancer treated with oxaliplatin and fluoropyrimidine-based chemotherapy","volume":"52","author":"N\u00f6pel-D\u00fcnnebacke","year":"2014","journal-title":"Z\u00a0Gastroenterol"},{"issue":"5","key":"10.1016\/j.jval.2024.03.010_bib59","first-page":"1615","article-title":"Microsatellite instability does not predict the efficacy of chemotherapy in metastatic colorectal cancer. A systematic review and meta-analysis","volume":"29","author":"Des Guetz","year":"2009","journal-title":"Anticancer Res"},{"issue":"4","key":"10.1016\/j.jval.2024.03.010_bib60","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1002\/ijc.29449","article-title":"The benefit of microsatellite instability is attenuated by chemotherapy in stage II and stage III gastric cancer: results from a large cohort with subgroup analyses","volume":"137","author":"Kim","year":"2015","journal-title":"Int J Cancer"},{"key":"10.1016\/j.jval.2024.03.010_bib61","doi-asserted-by":"crossref","first-page":"156","DOI":"10.1186\/s12885-015-1093-4","article-title":"Systematic review of the predictive effect of MSI status in colorectal cancer patients undergoing 5FU-based chemotherapy","volume":"15","author":"Webber","year":"2015","journal-title":"BMC Cancer"},{"issue":"387","key":"10.1016\/j.jval.2024.03.010_bib62","doi-asserted-by":"crossref","first-page":"516","DOI":"10.1080\/01621459.1984.10478078","article-title":"Reducing bias in observational studies using subclassification on the propensity score","volume":"79","author":"Rosenbaum","year":"1984","journal-title":"J\u00a0Am Stat Assoc"}],"container-title":["Value in Health"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1098301524001256?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1098301524001256?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,7,1]],"date-time":"2025-07-01T12:05:29Z","timestamp":1751371529000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1098301524001256"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7]]},"references-count":53,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2024,7]]}},"alternative-id":["S1098301524001256"],"URL":"https:\/\/doi.org\/10.1016\/j.jval.2024.03.010","relation":{},"ISSN":["1098-3015"],"issn-type":[{"value":"1098-3015","type":"print"}],"subject":[],"published":{"date-parts":[[2024,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Tackling Challenges in Assessing the Economic Value of Tumor-Agnostic Therapies: A Cost-Effectiveness Analysis of Pembrolizumab as a Case Study","name":"articletitle","label":"Article Title"},{"value":"Value in Health","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jval.2024.03.010","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}