Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: applications and practical considerations.
Adaptive seamless phase II/III designs combine a phase II and a phase III study into one single confirmatory clinical trial. Several examples of such designs are presented, where the primary endpoint is binary, time-to-event or continuous. The interim adaptations considered include the selection of treatments and the selection of hypotheses related to a pre-specified subgroup of patients. Practical aspects concerning the planning and implementation of adaptive seamless confirmatory studies are also discussed.
Schmidli
Heinz
H
Novartis Pharma AG, Lichtstrasse 35, 4002 Basel, Switzerland.
Bretz
Frank
F
Racine
Amy
A
Maurer
Willi
W
D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data*; D017326:Clinical Trials, Phase III as Topic / Q000706:statistics & numerical data*; D023381:Endpoint Determination; D012107:Research Design / Q000706:statistics & numerical data*; D018401:Sample Size