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Abstract:
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The traditional randomized clinical trial turned medical research into a real science. But it is not well suited to the modern era of many therapies and diseases increasingly recognized to be heterogeneous. New designs consider many therapies (platform trials) and many disease subtypes (basket trials). These designs are being used or are being introduced into many cancers, Alzheimer’s, Ebola, sepsis, and many other diseases. The FDA has been instrumental in encouraging and promoting innovations in clinical trials design, for example, “Promoting the Use of Complex Innovative Designs in Clinical Trials". In my presentation I will describe platform trials in cancer that also include indication finding within particular diseases. These include I-SPY 2, a phase II trial in neoadjuvant breast cancer, and GBM AGILE and Precision Promise, seamless phase II/III trials in glioblastoma and pancreatic cancer. The latter two trials take a Bayesian approach and include many statistical innovations never before used in drug registration trials. These include utilizing nonconcurrently randomized controls and adaptive randomization. GBM AGILE is currently accruing patients and Precision Promise is scheduled to start in December 2020.
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