Adaptive Designs and bias in treatment effects estimation
Fulvio Di Stefano
Adaptive Designs and bias in treatment effects estimation.
Rel. Mauro Gasparini, Gaelle Saint-Hilary. Politecnico di Torino, Corso di laurea magistrale in Ingegneria Matematica, 2020
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Abstract
Every year a huge number of new drugs are developed against different pathologies and tested to evaluate their efficacy and safety. In clinical trials, one of the main objectives is to identify the most effective dose among several ones to obtain the maximum efficacy from a treatment. In classical Randomized Controlled Trials (RCTs), this is performed by giving different doses and a reference treatment or a placebo to a certain population, and by estimating the treatment effects via Maximum-Likelihood Estimation in order to compare them. In recent years, Adaptive Designs (ADs) have been developed to enhance clinical development. One of the main advantage of this procedure consists in the possibility, at interim analyses during the trial, to stop the evaluation of certain treatments for lack of efficacy and to focus only on the best ones.
This results in improvements both in terms of resources and ethics, because it reduces the number of patients receiving non effective treatments
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