Adaptive designs in clinical drug development--an Executive Summary of the PhRMA Working Group.
A PhRMA Working Group on adaptive clinical trial designs has been formed to investigate and facilitate opportunities for wider acceptance and usage of adaptive designs and related methodologies. A White Paper summarizing the findings of the group is in preparation; this article is an Executive Summary for that full White Paper, and summarizes the findings and recommendations of the group. Logistic, operational, procedural, and statistical challenges associated with adaptive designs are addressed. Three particular areas where it is felt that adaptive designs can be utilized beneficially are discussed: dose finding, seamless Phase II/III trials designs, and sample size reestimation.
Gallo
Paul
P
Novartis Pharmaceuticals, East Hanover, NJ 07936, USA. paul.gallo@novartis.com
Chuang-Stein
Christy
C
Dragalin
Vladimir
V
Gaydos
Brenda
B
Krams
Michael
M
Pinheiro
José
J
D035843:Biomedical Research; D026661:Clinical Trials Data Monitoring Committees; D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D003627:Data Interpretation, Statistical; D004305:Dose-Response Relationship, Drug; D004345:Drug Industry / Q000592:standards; D017408:Guidelines as Topic; D006801:Humans; D012107:Research Design; D018401:Sample Size; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards