Designing a new proof-of-principle trial for treatment of partial seizures to demonstrate efficacy with minimal sample size and duration—A case study

Highlights • We modeled an ideal study design for an epilepsy proof-of-principle (PoP) trial. • The ideal PoP study gives a strong efficacy signal with minimum treatment exposure. • Modeling was based on pooled data from 4 pregabalin add-on focal epilepsy trials. • The model showed: PoP trial with 4 weeks baseline, 3 weeks treatment is sufficient. • Sufficient sample size was 40–50 per group (for a highly efficacious drug).