Drugs for the Treatment of Partial Onset Seizures: Extrapolation of Efficacy from Adults to Pediatric Patients 1 Month of Age and Older July 2024

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/drugs-treatment-partial-onset-seizures-extrapolation-efficacy-adults-pediatric-patients-1-month-age?utm_medium=email&utm_source=govdelivery FDA Announces Availability of a Revised Final Guidance on Extrapolating Efficacy Data to Treat Partial Onset Seizures in Pediatric Patients On July 15, 2024, the FDA announced the availability of a revised final guidance for industry entitled Drugs for the Treatment of Partial Onset Seizures: Extrapolation of Efficacy from Adults to Pediatric Patients 1 Month of Age and Older, replacing the 2019 final guidance which discussed extrapolation to pediatric patients 4 years of age and older. This revised final guidance provides recommendations to sponsors on the clinical development of drugs for the treatment of partial onset seizures (POS) in pediatric patients. Specifically, this guidance addresses FDA’s current thinking regarding clinical development programs that can support extrapolation of the efficacy of drugs approved for the treatment of POS in adults to pediatric patients 1 month of age and older. This guidance discusses formulation development, efficacy and safety considerations, and clinical pharmacology and dosing considerations. This guidance does not address the development of drugs to treat other types of seizures. Historically, efficacy for the treatment of POS in pediatric patients was established by performing one or more adequate and well-controlled clinical studies in pediatric patients. However, efficacy can be extrapolated from adults to pediatric patients when it is reasonable to assume that children, compared with adults, have a similar progression of disease, similar response of disease to treatment, and similar exposure-response relationship. Initial systematic and quantitative analyses conducted by FDA, using data from clinical studies of drugs with a variety of putative mechanisms of action approved for the treatment of POS in both adults and pediatric patients, have shown that the relationship between exposure and response (reduction in seizure frequency) is similar in adults and pediatric patients 4 years of age and older. Subsequent evaluation by FDA determined that POS are readily identifiable in pediatric patients down to 1 month of age and are phenomenologically and pathophysiologically very similar to those in older patients. These analyses and observations have allowed FDA to conclude that the efficacy of drugs approved for the treatment of POS can be extrapolated from adults to pediatric patients 1 month of age and older.