Takeaways from the Epilepsy Foundation Pipeline Conference in Washington D.C.

Research evidence excerpt

Takeaways from the Epilepsy Foundation Pipeline Conference in Washington D.C.

anslate to

Accordingly, the key ways to address inefficiencies of study designs discussed included: 1) successful pivotal studies.

platform trial infrastructure to use a continuous placebo arm over time compared against

multiple different drugs in development; 2) re-thinking power calculations to reduce sample Regulatory risk. Even with guidance by

size; 3) implementing continuous blinded interim analyses to provide opportunities for regulators for late-stage clinical trials, success

studies to be stopped early for efficacy or futility; 4) adjusting potential cutoffs in eligibility on these clinical endpoints still may not fetch

recruitment criteria; 5) design adjustments to improve safety (e.g. lowering required seizure approval.

frequency, etc.); and 6) resolving statistical ties from seizure freedom to improve outcomes

measures. See pg. 4 Financing risk. Clinical development programs

2. Moreover, the conference featured a panel of experts (sponsors, sites/coordinators, could be delayed or halted due to an inability to

participants) to discuss considerations for designing clinical trial programs as it pertains raise sufficient capital.

to costs and toxicology models. From a cost perspective, the panel acknowledged that

different types of toxicology models are needed for different types of studies listed

in order from least to most expensive: 1) Ph1 FIH requires a single dose ~2-week

toxicology study; 2) Ph1/2 studies require a sub-chronic ~1-3 month model to cover

duration of exposure; 3) Ph3/chronic use requires chronic ~6-9-month models to evaluate

longer-term toxicology; and 4) final marketing approval needs a ~2-year carcinogenicity

toxicology model.