{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T12:05:54Z","timestamp":1753877154863,"version":"3.41.2"},"reference-count":16,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2025,2,13]],"date-time":"2025-02-13T00:00:00Z","timestamp":1739404800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,3,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Background<\/jats:title>\n                  <jats:p>It is important to understand the relationship between drug efficacy measured in randomized clinical trials (RCTs) and real-world drug effectiveness. We estimate how RCT overall survival (OS) and RCT radiographic progression-free survival (rPFS) benefits predict the association between treatments and real-world OS gains for metastatic castration-resistant prostate cancer (mCRPC) drugs.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>Using the National Cancer Institute list of approved cancer drugs and the National Comprehensive Cancer Network Treatment Guidelines, we identified all pharmaceutical therapies for mCRPC approved between 2010 and 2019. We obtained RCT OS and rPFS hazard ratios from the pivotal trials used for Food and Drug Administration (FDA) approval, and we estimated real-world OS hazard ratios using the Optum Clinformatics Extended DataMart Databases. We modeled real-world OS hazard ratios as a function of both RCT OS and RCT rPFS hazard ratios using Cox proportional hazards regressions, adjusted for year of diagnosis, age, race, and Elixhauser Comorbidity Index.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>When we did not account for nonrandom real-world selection of patients into receiving a newly approved therapy (ie, \u201ctreatment selection bias\u201d), real-world OS gains were 15% lower than associated RCT OS and RCT rPFS benefits. However, after accounting for treatment selection bias in real-world settings, real-world OS gains were almost 28% greater than RCT OS and RCT rPFS benefits. Association between treatment and OS gains increased the longer a new therapy was on the market.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusions<\/jats:title>\n                  <jats:p>After adjusting for treatment selection bias, RCT OS and RCT rPFS estimates serve as useful, or even conservative, predictors of RW OS gains.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1093\/jncics\/pkaf018","type":"journal-article","created":{"date-parts":[[2025,2,13]],"date-time":"2025-02-13T16:06:13Z","timestamp":1739462773000},"source":"Crossref","is-referenced-by-count":0,"title":["Do trial benefits predict real-world gains in metastatic castration resistant prostate cancer"],"prefix":"10.1093","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1538-0013","authenticated-orcid":false,"given":"Sarah","family":"Axeen","sequence":"first","affiliation":[{"name":"Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California , Los Angeles, CA 90089,","place":["United States"]},{"name":"Keck School of Medicine, University of Southern California , Los Angeles, CA 90089,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7908-2664","authenticated-orcid":false,"given":"Alice J","family":"Chen","sequence":"additional","affiliation":[{"name":"Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California , Los Angeles, CA 90089,","place":["United States"]},{"name":"Sol Price School of Public Policy, University of Southern California , Los Angeles, CA 90089,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5934-8042","authenticated-orcid":false,"given":"Darius N","family":"Lakdawalla","sequence":"additional","affiliation":[{"name":"Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California , Los Angeles, CA 90089,","place":["United States"]},{"name":"Sol Price School of Public Policy, University of Southern California , Los Angeles, CA 90089,","place":["United States"]},{"name":"Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California , Los Angeles, CA 90089,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neal","family":"Masia","sequence":"additional","affiliation":[{"name":"Columbia Business School, Columbia University , New York City, NY 10027,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alexander","family":"Niyazov","sequence":"additional","affiliation":[{"name":"Pfizer, Inc. , Collegeville, PA 19426,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4553-0094","authenticated-orcid":false,"given":"Bhakti","family":"Arondekar","sequence":"additional","affiliation":[{"name":"Pfizer, Inc. , Collegeville, PA 19426,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8104-6419","authenticated-orcid":false,"given":"Stephen J","family":"Freedland","sequence":"additional","affiliation":[{"name":"Department of Urology, Cedars Sinai Medical Center , Los Angeles, CA 90048,","place":["United States"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2025,2,13]]},"reference":[{"key":"2025030423473184700_pkaf018-B1","doi-asserted-by":"crossref","first-page":"1562861","DOI":"10.1080\/20016689.2018.1562861","article-title":"Perspective to mitigate payer uncertainty in health technology assessment of novel oncology drugs","volume":"7","author":"Sola-Morales","year":"2019","journal-title":"J Mark Access Policy"},{"key":"2025030423473184700_pkaf018-B2","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1016\/S0140-6736(04)17670-8","article-title":"External validity of randomised controlled trials: \u201cto whom do the results of this trial apply?","volume":"365","author":"Rothwell","year":"2005","journal-title":"Lancet"},{"key":"2025030423473184700_pkaf018-B3","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1162\/REST_a_00670","article-title":"The local influence of pioneer investigators on technology adoption: evidence from new cancer drugs","volume":"100","author":"Agha","year":"2018","journal-title":"Rev Econ Stat"},{"key":"2025030423473184700_pkaf018-B4","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1093\/fampra\/16.4.407","article-title":"Diffusion of new drugs in Danish general practice","volume":"16","author":"Steffensen","year":"1999","journal-title":"Fam Pract"},{"key":"2025030423473184700_pkaf018-B5","doi-asserted-by":"crossref","first-page":"469","DOI":"10.1186\/1472-6963-14-469","article-title":"Factors affecting the uptake of new medicines: a systematic literature review","volume":"14","author":"Lubloy","year":"2014","journal-title":"BMC Health 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castration-resistant prostate cancer treated with cabazitaxel: comparison with the randomized clinical study CARD","volume":"26","author":"de Wit","year":"2023","journal-title":"Prostate Cancer Prostatic Dis"},{"key":"2025030423473184700_pkaf018-B9","doi-asserted-by":"crossref","first-page":"1717","DOI":"10.1002\/cncr.32697","article-title":"Assessing the efficacy-effectiveness gap for cancer therapies: a comparison of overall survival and toxicity between clinical trial and population-based, real-world data for contemporary parenteral cancer therapeutics","volume":"126","author":"Phillips","year":"2020","journal-title":"Cancer"},{"key":"2025030423473184700_pkaf018-B10","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.jval.2017.04.003","article-title":"Predicting real-world effectiveness of cancer therapies using overall survival and progression-free survival from clinical trials: empirical evidence for the ASCO value 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Health"}],"container-title":["JNCI Cancer 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