Clinical trial designs for rare diseases 2: trial protocols

Published on May 30, 2019   27 min

Other Talks in the Category: Methods

0:04
Let's switch gears and now talk about some innovative or newer clinical trial approaches that are beginning to be used. The master protocols which includes at the moment basket trials, umbrellas, and platforms.
0:20
So, a master protocol is generally described as a framework in which multiple parallel drugs substudies are operated under one over-arching protocol. The main goal of a master protocol is efficiency; efficiency in time, efficiency in the use of patients, lowering costs, shared information, and infrastructure. The aims, both now and aspirationally are, increased efficiencies for these targeted drug approaches, and we're moving ever more towards personalized medicines, and how are we going to be able to study what may be one or a very small numbers of patients in clinical trials. So, basket and umbrella trials are biomarker or precision medicine-based designs, and we'll go back to this in a minute. They are generally leveraging advances in molecular profiling and target identification. A platform is generally described as a framework with longevity, where the infrastructure lives on regardless of what treatment groups are in there. They're often adaptive designs, but they aren't required to be and we'll touch on this in a few minutes as well. So, currently, most of the experience with these is in cancer. Again, cancer is leading the way with precision medicine, but they aren't limited to it, and we'll go over some of these examples.
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Clinical trial designs for rare diseases 2: trial protocols

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