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Clinical trials can be separated into phase I (dose finding and safety), phase II (activity or early efficacy), phase III (efficacy compared with current standard of care) and occasionally phase IV (postmarketing studies). A new compound would usually have to go through phase I–III studies sequentially with all of the financial and regulatory hurdles this poses. A recent study has estimated that only 13.8% of compounds tested will be successful in achieving a marketing license.1
Adaptive designs are an extensive class of flexible tools which use accumulated data in the trial to make preplanned adaptations to the trial’s course. They can be used in all trial phases. The adaptations can include stopping an arm early for futility or safety, closing recruitment to an arm early if there is strong evidence of efficacy, changing of target sample size or allocation ratios. They are usually more efficient, informative and ethical than traditional fixed designs (where no interim adaptations are permitted). They can often offer savings in resources and even number of patients (figure 1).2
A novel paradigm for conducting adaptive trials, which allows several treatments to be assessed concurrently with preplanned interim adaptations, is the so-called multiarm, …
Contributors GCM wrote and revised the initial manuscript. CY wrote, reviewed and revised the manuscript prior to submission. Both authors approved the final manuscript.
Funding CY is funded by Cancer Research UK Grant No. C22436/A15958.
Competing interests None declared.
Patient consent for publication Not required.
Provenance and peer review Commissioned; externally peer reviewed.
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