Richard L. Schilsky, MD
Innovative clinical trials designed to exploit the explosion of data on the drivers of cancer are demonstrating value as tools to explore potential signals of efficacy across tumor types, according to oncology experts. These studies have become a feature of the drug discovery landscape in oncology and are likely to remain part of the fabric of clinical research in the genomic era.
Trials like TAPUR are basket-umbrella hybrids, testing a number of therapies in multiple diseases and assigning patients to cohorts according to their genomic alterations. If a study is open ended and regularly adds new therapies and patients, it is often called a platform trial. Master protocol-type trials may be adaptive, using Bayesian statistical methods to optimally adjust dosing and enrollment as the trial proceeds.
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Although investigators have used elements of these trial designs in the past, in the era of genomic medicine, such studies are particularly well suited to serving patients who have rare alterations and sorting out which agents are active in which disease variants, experts say.
“We’re now slicing up the molecular pie into many thin slices because we’re using a precision medicine approach. Finding these molecularly selected patients can be tough, so we need a good number of centers to actually find these patients, and an efficient way of doing that would be to do molecularly driven basket and umbrella studies where each arm represents a molecular subtype,” said Timothy Yap, MBBS, PhD, MRCP, medical director of the Institute for Applied Cancer Science at The University of Texas MD Anderson Cancer Center in Houston.
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