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.
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.
So, let's start with the basket trials.
Baskets, umbrellas, and platforms,
all have had various definitions over time,
but the most common definition that's seems to be settled on for basket trials,
is that they enroll patients with multiple cancer types defined by histology,
some examples here are colon,
liver, lung, pancreas, but it can be any,
but they have the same genetic mutation.
So, in the picture here,
you see the patients are clustered by
the histologic type and the patients have undergone testing with an assay,
and if you have the mutation of interest, the ones,
the stick figures in red,
then those patients can be entered into the trial and receive the same treatment.
So, they will be essentially clustered into the same mutationally-defined basket.