PMID- 36905971
OWN - NLM
STAT- MEDLINE
DCOM- 20230526
LR  - 20230603
IS  - 1878-5921 (Electronic)
IS  - 0895-4356 (Linking)
VI  - 157
DP  - 2023 May
TI  - Early phase clinical trial played a critical role in the Food and Drug 
      Administration-approved indications for targeted anticancer drugs: 
      a cross-sectional study from 2012 to 2021.
PG  - 74-82
LID - S0895-4356(23)00049-5 [pii]
LID - 10.1016/j.jclinepi.2023.03.006 [doi]
AB  - OBJECTIVES: To characterize the indications approved by the US Food and Drug 
      Administration (FDA) on the basis of early phase clinical trials (EPCTs) and 
      compared with that of phase three randomized controlled trials. STUDY DESIGN AND 
      SETTING: We collected the publicly available FDA documents of targeted anticancer 
      drugs approved between January 2012 and December 2021. RESULTS: We identified 95 
      targeted anticancer drugs with 188 indications approved by the FDA. One hundred 
      and twelve (59.6%) indications were approved on the basis of EPCTs, with a 
      significant increase of 22.2% per year. Of 112 EPCTs, 32 (28.6%) were 
      dose-expansion cohort trials and 75 (67.0%) were single-arm phase 2 trials, 
      respectively, with a significant increase of 29.7% and 18.7% per year. Compared 
      with indications approved on the basis of phase three randomized controlled 
      trials, the indications approved on the basis of EPCTs had significantly higher 
      odds in receiving accelerated approval and lower odds in the number of entered 
      patients of pivotal trials. CONCLUSIONS: Dose-expansion cohort trials and 
      single-arm phase 2 trials played a critical role in EPCTs. EPCT was a major trial 
      type in providing evidences for the FDA approvals of targeted anticancer drugs.
CI  - Copyright (c) 2023 The Author(s). Published by Elsevier Inc. All rights reserved.
FAU - Huang, Yafang
AU  - Huang Y
AD  - School of General Practice and Continuing Education, Capital Medical University, 
      Beijing, China. Electronic address: yafang@ccmu.edu.cn.
FAU - Xiong, Weiyi
AU  - Xiong W
AD  - Department II of Pharmaceuticals Surveillance and Evaluation, National Center for 
      ADR monitoring, Beijing, China.
FAU - Zhao, Jingwei
AU  - Zhao J
AD  - School of General Practice and Continuing Education, Capital Medical University, 
      Beijing, China.
FAU - Li, Wentao
AU  - Li W
AD  - School of General Practice and Continuing Education, Capital Medical University, 
      Beijing, China.
FAU - Ma, Li
AU  - Ma L
AD  - Department of General Practice, Beijing Tiantan Hospital, Capital Medical 
      University, Beijing, China.
FAU - Wu, Hao
AU  - Wu H
AD  - School of General Practice and Continuing Education, Capital Medical University, 
      Beijing, China. Electronic address: wushunzhe@ccmu.edu.cn.
LA  - eng
PT  - Clinical Trial, Phase I
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20230310
PL  - United States
TA  - J Clin Epidemiol
JT  - Journal of clinical epidemiology
JID - 8801383
RN  - 0 (Antineoplastic Agents)
SB  - IM
MH  - United States
MH  - Humans
MH  - Cross-Sectional Studies
MH  - United States Food and Drug Administration
MH  - *Drug Approval
MH  - *Antineoplastic Agents/therapeutic use
OTO - NOTNLM
OT  - Dose-expansion cohort
OT  - Early phase clinical trial
OT  - FDA approved indications
OT  - Pivotal trial
OT  - Single-arm trial
OT  - Targeted anticancer drugs
EDAT- 2023/03/12 06:00
MHDA- 2023/05/26 06:42
CRDT- 2023/03/11 19:27
PHST- 2022/10/18 00:00 [received]
PHST- 2023/01/29 00:00 [revised]
PHST- 2023/03/06 00:00 [accepted]
PHST- 2023/05/26 06:42 [medline]
PHST- 2023/03/12 06:00 [pubmed]
PHST- 2023/03/11 19:27 [entrez]
AID - S0895-4356(23)00049-5 [pii]
AID - 10.1016/j.jclinepi.2023.03.006 [doi]
PST - ppublish
SO  - J Clin Epidemiol. 2023 May;157:74-82. doi: 10.1016/j.jclinepi.2023.03.006. Epub 
      2023 Mar 10.