PMID- 28872409
OWN - NLM
STAT- MEDLINE
DCOM- 20190726
LR  - 20190726
IS  - 1520-5711 (Electronic)
IS  - 1054-3406 (Linking)
VI  - 28
IP  - 3
DP  - 2018
TI  - Considerations for pediatric trial designs and analyses.
PG  - 413-436
LID - 10.1080/10543406.2017.1372771 [doi]
AB  - There are challenges in designing pediatric trials arising from special ethical 
      issues and the relatively small accessible patient population. The application of 
      conventional phase 3 trial designs to pediatrics is not realistic in some 
      therapeutic areas. To address this issue, we propose various approaches for 
      designing pediatric trials that incorporate data available from adult studies 
      using James-Stein shrinkage estimation, empirical shrinkage estimation, and 
      Bayesian methods. We also apply the concept of consistency used in multi-regional 
      trials to pediatric trials. The performance of these methods is assessed through 
      representative scenarios and an example using actual Type 2 diabetes mellitus 
      (T2DM) trials.
FAU - Cho, Meehyung
AU  - Cho M
AD  - a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
FAU - Qiu, Zhiying
AU  - Qiu Z
AD  - a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
FAU - Ye, Jenny
AU  - Ye J
AD  - a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
FAU - Quan, Hui
AU  - Quan H
AD  - a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
FAU - Zhao, Peng-Liang
AU  - Zhao PL
AD  - a Biostatistics and Programming , Sanofi US , Bridgewater , NJ , USA.
LA  - eng
PT  - Journal Article
DEP - 20171006
PL  - England
TA  - J Biopharm Stat
JT  - Journal of biopharmaceutical statistics
JID - 9200436
MH  - Child
MH  - Clinical Trials as Topic/ethics/*standards
MH  - Diabetes Mellitus, Type 2/diagnosis/epidemiology/*therapy
MH  - Humans
MH  - Pediatrics/ethics/*standards
MH  - Practice Guidelines as Topic/*standards
OTO - NOTNLM
OT  - Bayesian method
OT  - James-Stein shrinkage estimation
OT  - empirical shrinkage estimation
OT  - pediatric
OT  - sample size
OT  - weighted combination
EDAT- 2017/09/06 06:00
MHDA- 2019/07/28 06:00
CRDT- 2017/09/06 06:00
PHST- 2017/09/06 06:00 [pubmed]
PHST- 2019/07/28 06:00 [medline]
PHST- 2017/09/06 06:00 [entrez]
AID - 10.1080/10543406.2017.1372771 [doi]
PST - ppublish
SO  - J Biopharm Stat. 2018;28(3):413-436. doi: 10.1080/10543406.2017.1372771. Epub 
      2017 Oct 6.