Abstract
For the past 60 years, the gold standard for assessing a new treatment’s efficacy is the randomized phase III trial. When properly designed, conducted, and reported, these studies can provide level-1 evidence that a new treatment is, on average, more effective than standard treatment in a patient population. This trial design does have some limitations, however, and nonrandomized treatment comparisons can be used to provide level-2 evidence concerning treatment effects or even extend the interpretation of a randomized trial. Propensity score analysis is one analytic approach for comparing nonrandomized treatments. Alternative analytic approaches to comparing nonrandomized treatments are an active area of statistical research.
Due to the increasing complexity and cost of conducting randomized phase III trials, there has been a growing reliance on early phase trials to provide preliminary evidence of the new treatment’s activity or to refine the hypotheses for the phase III trial. Growing interest in targeted therapies has contributed to a shift away from single-arm trials toward randomized phase II trials. In an effort to shorten the time for drug development and reduce resource requirements, clinical trialists are reassessing the phase II/III trial design. Adaptive trial designs attempt to provide a more seamless approach to speeding up and increasing the efficiency of drug development.
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Brady, M.F., Gebski, V. (2014). Trial Design: Should Randomized Phase III Trials in Gynecological Cancers Be Abandoned?. In: Ledermann, J., Creutzberg, C., Quinn, M. (eds) Controversies in the Management of Gynecological Cancers. Springer, London. https://doi.org/10.1007/978-0-85729-910-9_25
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