Adaptive Designs for Clinical Trials

Bhatt DL, Mehta C.
Adaptive Designs for Clinical Trials. N Engl J Med 2016;375:65-74.

The following are key points to remember from a review article on the changing face of clinical trials about adaptive designs for clinical trials:

  1. Randomized clinical trials have served as the standard for clinical research and have contributed significantly to advances in patient care. However, randomized clinical trials need a large sample size and long study duration, and may lack power to evaluate efficacy overall or in important subgroups, and have high cost.
  2. Adaptive trial design has been proposed as a means to increase the efficiency of randomized clinical trials, potentially benefiting trial participants and future patients while reducing costs and enhancing the likelihood of finding a true benefit, if one exists, of the therapy being studied.
  3. Adaptive designs are applicable to both exploratory and confirmatory clinical trials.
  4. Adaptive designs for exploratory clinical trials deal mainly with finding safe and effective doses or with dose–response modeling.
  5. In confirmatory trials, the adaptive nomenclature refers to making prospectively planned changes to the future course of an ongoing trial on the basis of an analysis of accumulating data from the trial itself, in a fully blinded or unblinded manner, without undermining the statistical validity of the conclusions.
  6. Wider use of adaptive trial designs may accelerate the discovery process, especially if coupled with other evolving trial concepts, such as large simple trials, but will require further dissemination and acceptance of the sometimes complex statistical methods for this design.

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