Abstract:
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Advances in pan-omic technologies and systems biology have led to novel therapies in the era of precision medicine. After describing the challenges in designing randomized controlled trials to evaluate these new treatments, such as recruiting patients with rare genetic subtypes of a disease and the eventual unaffordability of the treatment, we give a brief review of recent developments in master protocols for umbrella, basket, and platform trials to address these challenges. We then introduce innovative adaptive designs of basket trials, which we illustrate with a master protocol of a clinical trial to evaluate the effect of a CK2 inhibitor on four baskets of cancers involved in the hedgehog pathway for pediatric patients.
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