Data and Safety Monitoring
2
Peter C. O'Brien,
Peter C. O'Brien
National Institutes of Health, Bethesda, MD, USA
Search for more papers by this authorPeter C. O'Brien,
Peter C. O'Brien
National Institutes of Health, Bethesda, MD, USA
Search for more papers by this authorAbstract
We consider the role of a Data and Safety Monitoring Board (DSMB) in overseeing the conduct of a randomized clinical trial. This includes insuring both the scientific integrity of the study and patient safety, monitoring the occurrence of adverse events and assessing efficacy. Formal stopping rules are particularly useful in clinical trials for monitoring efficacy, with a view towards terminating the trial early if the evidence for or against efficacy becomes overwhelming prior to the scheduled completion of the trial. The most commonly used approaches in formal stopping rules are discussed in some detail. These include the Pocock and O'Brien–Fleming boundaries and the spending function approach developed by Lan and DeMets. Discussion of stochastic curtailment, point and interval estimation associated with early termination, multiple endpoints, and a brief overview of decision theoretic methods (including Bayesian methods) is provided.
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