[Adaptive studies to assess drug effectiveness]
Paolo Bruzzi1, Giuseppe Traversa2
1Dipartimento di epidemiologia clinica, IRCCS AOU San Martino - IST, Genova. bruzzipaolo49@gmail.com.
Abstract:
The clinical development of drugs is based on a consolidated model that adopts, as a reference, the conduct of consecutive dose-finding and activity studies (the phases), up to the classical randomized controlled trials (RCTs), which are strictly regulated by a study protocol. This model is likely to be too rigid, since it cannot incorporate the information that gradually becomes available. To overcome, at least partly, this limited flexibility, so-called adaptive designs have been proposed: in these designs, it is possible to introduce changes and also to modify relevant aspects of a single design on the basis of results/information obtained in the study itself. This article presents the main characteristics of these designs and discusses the precautions to be taken into account in the application in clinical studies.
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