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Methods to improve the efficiency of confirmatory clinical trials

This thesis demonstrates how innovative clinical trial methodology can foster pharmaceutical development. pharmaceutical development is increasingly costly and ineffective. Most time and money is directed towards late-stage clinical trials that aim to confirm the safety and efficacy of the investigated medicine. The methods discussed in this thesis help to optimize the efficiency and success-rates of these trials, which is crucial to ensure the continued arrival of new and affordable therapies. Clinical Trial Simulation (CTS) was presented as an effective technique to simulate the conduct of a trial in order to identify shortcomings in the study protocol before the trial is started. Various adaptive study designs (i.e. designs that allow for modifications of the protocol while the trial is on-going) were shown to have major potential to increase trial efficiency (i.e. reduce sample size requirements). The methods that were discussed in this thesis were shown to be statistically sound and potentially (much) more efficient than conventional approaches. Pharmaceutical developers are therefore encouraged to apply them.

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