scholarly journals A simple solution to the inadequacy of asymptotic likelihood-based inference for response-adaptive clinical trials

2021 ◽  
Author(s):  
Alessandro Baldi Antognini ◽  
Marco Novelli ◽  
Maroussa Zagoraiou
Keyword(s):  
Clinical Trials ◽  
Primary Outcome ◽  
Wald Test ◽  
Simple Solution ◽  
Nuisance Parameters ◽  
Simulation Studies ◽  
Fundamental Properties ◽  
Adaptive Clinical Trials ◽  
Treatment Comparisons ◽  
Practical Recommendations

AbstractThe present paper discusses drawbacks and limitations of likelihood-based inference in sequential clinical trials for treatment comparisons managed via Response-Adaptive Randomization. Taking into account the most common statistical models for the primary outcome—namely binary, Poisson, exponential and normal data—we derive the conditions under which (i) the classical confidence intervals degenerate and (ii) the Wald test becomes inconsistent and strongly affected by the nuisance parameters, also displaying a non monotonic power. To overcome these drawbacks, we provide a very simple solution that could preserve the fundamental properties of likelihood-based inference. Several illustrative examples and simulation studies are presented in order to confirm the relevance of our results and provide some practical recommendations.

scholarly journals Critical concepts in adaptive clinical trials

2018 ◽  
Vol Volume 10 ◽  
pp. 343-351 ◽  
Author(s):  
Jay JH Park ◽  
Kristian Thorlund ◽  
Edward J Mills
Keyword(s):  
Clinical Trials ◽  
Adaptive Clinical Trials

Trial Termination and Drug Misclassification in Sequential Adaptive Clinical Trials

2018 ◽  
Vol 10 (3) ◽  
pp. 354-377 ◽  
Author(s):  
Alba C. Rojas-Cordova ◽  
Niyousha Hosseinichimeh
Keyword(s):  
Clinical Trials ◽  
Adaptive Clinical Trials
Sign in / Sign up

Export Citation Format

Share Document