Platform trial designs in drug development (2019) Pdf Free Download

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Platform trials, including umbrella and basket trials, test multiple therapies in one indication, one therapy for multiple indications, or both. As readers will learn in this volume, these novel clinical trial designs have the potential to dramatically increase the cost-effectiveness of drug development, leading to more life-altering medicines for people suffering from serious illnesses. Further, these medicines may be available at lower cost. Necessity is indeed the mother of invention in this instance, as the increasing cost of drug development is becoming unsustainable. Two particular problem areas are rare diseases, in which it may be quite difficult to accrue sufficient patients for traditional trial designs, and oncology, where increasing molecular understanding is creating small biomarker-defined subgroups from which it is also a challenge to accrue, and where the large number of such subgroups and the corresponding larger numbers of experimental therapies create a combinatorial explosion of reasonable testable clinical trial hypotheses.
Our intention was to recruit the key leaders and innovators in this domain, to give them free rein to discuss what they felt to be most important with minimal editorial constraints, and to cover the topic from multiple perspectives, including some that are unconventional. We have indeed been fortunate to collect thoughts from many of those who broke new ground to form and advance this field, and from a variety of creative thinkers viewing these trial designs from perspectives as diverse as quantum computing, patient’s rights to information, and international health.


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