178: Evolving Approaches to Epilepsy Clinical Trial Design

obviously dr perry epilepsy trial design has you know evolved and changed a lot over the last decade or so so where do we think we are right now and what's been the biggest shift in how we approach these studies

You know, I mean, there's been a lot of things that have changed in trials.

I think one of the more recent things that we're seeing is the use of basket trial design.

So this concept that, you know, a lot of anti-seizure meds are just that, the kind of anti-seizure meds, they're not specific necessarily to any one etiology.

So instead of grouping, you know, a trial around a single syndrome, you could take several syndromes that are kind of similar, right? So take multiple developmental and epileptic encephalopathies.

And as long as the medicine doesn't have some specific precision reason to be better for one than the other, why not group them together and study them as a group? And then maybe within the group, in the trial, you'll see some do better than others.

But just a way to kind of really... examine more epilepsies at one time.

I think some other changes, you're starting to see designs that are trying to get away from placebo groups if we can, especially when we're dealing with these more severe epilepsies where there's considerable risks to placebo for long-term.

So think about designs that are like time to event trials, where at least if you're in the placebo group, you can get out of placebo as soon as the target event happens, or maybe studies where we're using natural history.

So instead of having a placebo group at all, just compare the outcomes of those in the trial to what the known natural history of the disease is.

Gosh, what else? We're seeing more N of 1 trials.

Right.

So more and more things being designed specifically for a patient and that being the one person that's in the trial that's getting the drug.

And then obviously, I am biased to say that the change I really like is that we're including more really, really young people in trial.

So those under the age of two that so rarely get opportunities to have drug trials designed for them.

Yeah.

And obviously, if we're able to make an impact, a significant impact at that early of a stage in an infant's life, then obviously we can have some substantial changes for the long term.

You know, we've obviously talked about kind of the design of trials and kind of the makeup of them, but I wanted to kind of shift focuses a little bit more on some of the endpoints.

It's kind of moved a little bit beyond seizure frequency as a primary endpoint in epilepsy trials.

So what other outcomes do you think deserve more attention that should kind of be propped up for primary or secondary endpoints in these trials?