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From 2012 to 2023, DEECs provided pivotal trial evidence that supported the FDA approval for 46 indications across 36 targeted anticancer drugs. Dose escalation uses 3 + 3, Bayesian optimal interval design, or continual reassessment method to explore the optimal dose level, whereas expansion cohorts directly incorporate dose-escalation cohort at the recommended phase II dose level. Expansion cohorts adopt flexible designs such as basket, umbrella, and platform trials. In addition, each expansion cohort in DEEC often uses adaptive approaches, such as Simon's two-stage design. To avoid the bias of end point assessment, conducting DEEC trials requires end point adjudication, often by an independent review committee. The design, conduct, and analysis of DEEC trials each have distinct characteristics. However, these characteristics were often overlooked in DEEC reporting. We reviewed the structural domains and items in trial design and conduct and discussed the strengths and limitations of DEEC studies, aiming to enhance the utilization of this trial design to generate higher-quality clinical evidence and ultimately contribute to better outcomes for patients with cancer.<\/jats:p>","DOI":"10.1200\/po-25-00253","type":"journal-article","created":{"date-parts":[[2025,8,1]],"date-time":"2025-08-01T19:59:09Z","timestamp":1754078349000},"update-policy":"https:\/\/doi.org\/10.1200\/crossmark","source":"Crossref","is-referenced-by-count":4,"title":["Using Dose-Escalation and -Expansion Cohort Study as Pivotal Trial for Targeted Anticancer Drug Approval"],"prefix":"10.1200","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4396-3870","authenticated-orcid":true,"given":"Yafang","family":"Huang","sequence":"first","affiliation":[{"name":"School of General Practice and Continuing Education, Capital Medical University, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ting","family":"Zhu","sequence":"additional","affiliation":[{"name":"School of General Practice and Continuing Education, Capital Medical University, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinjia","family":"Zhong","sequence":"additional","affiliation":[{"name":"School of General Practice and Continuing Education, Capital Medical University, Beijing, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinqiu","family":"Yuan","sequence":"additional","affiliation":[{"name":"Clinical Research Center, The Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"233","reference":[{"key":"e_1_3_6_2_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMp2208954"},{"key":"e_1_3_6_3_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamainternmed.2019.0583"},{"key":"e_1_3_6_4_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaoncol.2022.5985"},{"key":"e_1_3_6_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2023.03.006"},{"key":"e_1_3_6_6_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00109-017-1612-7"},{"key":"e_1_3_6_7_2","doi-asserted-by":"crossref","first-page":"e068177","DOI":"10.1136\/bmj-2021-068177","article-title":"Early phase clinical trials extension to guidelines for the content of statistical analysis plans","volume":"376","author":"Homer V","year":"2022","unstructured":"Homer V, Yap C, Bond S, et al: Early phase clinical trials extension to guidelines for the content of statistical analysis plans. 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