{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T21:41:54Z","timestamp":1790718114062,"version":"4.1.0"},"reference-count":38,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2023,2,24]],"date-time":"2023-02-24T00:00:00Z","timestamp":1677196800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004361","name":"Krebsliga Schweiz","doi-asserted-by":"publisher","award":["KFS\u20104842\u201008\u20102019"],"award-info":[{"award-number":["KFS\u20104842\u201008\u20102019"]}],"id":[{"id":"10.13039\/501100004361","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Intl Journal of Cancer"],"published-print":{"date-parts":[[2023,6,15]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Concerns have been raised that regulatory programs to accelerate approval of cancer drugs in cancer may increase uncertainty about benefits and harms for survival and quality of life (QoL). We analyzed all pivotal clinical trials and all non\u2010pivotal randomized controlled trials (RCTs) for all cancer drugs approved for the first time by the FDA between 2000 and 2020. We report regulatory and trial characteristics. Effects on overall survival (OS), progression\u2010free survival and tumor response were summarized in meta\u2010analyses. Effects on QoL were qualitatively summarized. Between 2000 and 2020, the FDA approved 145 novel cancer drugs for 156 indications based on 190 clinical trials. Half of indications (49%) were approved without RCT evidence; 82% had a single clinical trial only. OS was primary endpoint in 14% of trials and QoL data were available from 25%. The median OS benefit was 2.55\u2009months (IQR, 1.33\u20104.28) with a mean hazard ratio for OS of 0.75 (95%CI, 0.72\u20100.79,\n                    <jats:italic>I<\/jats:italic>\n                    <jats:sup>2<\/jats:sup>\n                    \u00a0=\u200942). Improvement for QoL was reported for 7 (4%) of 156 indications. Over time, priority review was used increasingly and the mean number of trials per indication decreased from 1.45 to 1.12. More trials reported results on QoL (19% in 2000\u20102005; 41% in 2016\u20102020). For 21\u2009years, novel cancer drugs have typically been approved based on one single, often uncontrolled, clinical trial, measuring surrogate endpoints. This leaves cancer patients without solid evidence that novel drugs improve their survival or QoL and there is no indication towards improvement.\n                  <\/jats:p>","DOI":"10.1002\/ijc.34473","type":"journal-article","created":{"date-parts":[[2023,2,13]],"date-time":"2023-02-13T09:05:40Z","timestamp":1676279140000},"page":"2474-2484","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":50,"title":["The evidence base of\n                    <scp>US<\/scp>\n                    Food and Drug Administration approvals of novel cancer therapies from 2000 to 2020"],"prefix":"10.1002","volume":"152","author":[{"given":"Viktoria","family":"Gloy","sequence":"first","affiliation":[{"name":"Department of Clinical Research University of Basel and University Hospital Basel  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9568-8164","authenticated-orcid":false,"given":"Andreas M.","family":"Schmitt","sequence":"additional","affiliation":[{"name":"Department of Clinical Research University of Basel and University Hospital Basel  Basel Switzerland"},{"name":"Department of Medical Oncology University Hospital Basel  Basel Switzerland"},{"name":"Department of Medical Oncology The Royal Marsden Hospital NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascal","family":"D\u00fcblin","sequence":"additional","affiliation":[{"name":"Department of Clinical Research University of Basel and University Hospital Basel  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian","family":"Hirt","sequence":"additional","affiliation":[{"name":"Department of Clinical Research University of Basel and University Hospital Basel  Basel Switzerland"},{"name":"International Graduate Academy, Institute of Health and Nursing Science, Medical Faculty Martin Luther University Halle\u2010Wittenberg  Halle (Saale) Germany"},{"name":"Institute of Nursing Science, Department of Health Eastern Switzerland University of Applied Sciences  St. Gallen Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cathrin","family":"Axfors","sequence":"additional","affiliation":[{"name":"Department for Women's and Children's Health Uppsala University  Uppsala Sweden"},{"name":"Meta\u2010Research Innovation Center at Stanford (METRICS) Stanford University  Stanford California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanna","family":"Kuk","sequence":"additional","affiliation":[{"name":"Department of Medicine University of Ottawa  Ottawa Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiago V.","family":"Pereira","sequence":"additional","affiliation":[{"name":"Applied Health Research Centre (AHRC) Li Ka Shing Knowledge Institute of St. Michael's Hospital University of Toronto Canada"},{"name":"Department of Health Sciences University of Leicester  Leicester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clara","family":"Locher","sequence":"additional","affiliation":[{"name":"Univ Rennes, CHU Rennes, Inserm, Centre d'investigation clinique de Rennes (CIC1414), service de pharmacologie clinique, Institut de recherche en sant\u00e9, environnement et travail (Irset)  Rennes France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Caquelin","sequence":"additional","affiliation":[{"name":"Univ Rennes, CHU Rennes, Inserm, Centre d'investigation clinique de Rennes (CIC1414), service de pharmacologie clinique, Institut de recherche en sant\u00e9, environnement et travail (Irset)  Rennes France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Walter\u2010Claudi","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology University Hospital Basel  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8952-7639","authenticated-orcid":false,"given":"Mark P.","family":"Lythgoe","sequence":"additional","affiliation":[{"name":"Department of Surgery &amp; 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