Proposal for a "phase II" multicenter trial model for preclinical new antiepilepsy therapy development

Terence J. O'Brien, Elinor Ben-Menachem, Edward H. Bertram, Stephen D. Collins, Merab Kokaia, Holger Lerche, Henrik Klitgaard, Kevin J. Staley, Elisabetta Vaudano, Matthew C. Walker, Michele Simonato

Research output: Contribution to journalArticleResearchpeer-review

12 Citations (Scopus)


There is a pressing need to address the current major gaps in epilepsy treatment, in particular drug-resistant epilepsy, antiepileptogenic therapies, and comorbidities. A major concern in the development of new therapies is that current preclinical testing is not sufficiently predictive for clinical efficacy. Methodologic limitations of current preclinical paradigms may partly account for this discrepancy. Here we propose and discuss a strategy for implementing a "phase II" multicenter preclinical drug trial model based on clinical phase II/III studies designed to generate more rigorous preclinical data for efficacy. The goal is to improve the evidence resulting from preclinical studies for investigational new drugs that have shown strong promise in initial preclinical "phase I" studies. This should reduce the risk for expensive clinical studies in epilepsy and therefore increase the appeal for funders (industry and government) to invest in their clinical development. Wiley Periodicals, Inc.

Original languageEnglish
Pages (from-to)70-74
Number of pages5
Issue numberSUPPL.4
Publication statusPublished - Aug 2013
Externally publishedYes


  • Antiepilepsy therapies
  • Multicenter
  • Preclinical development

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