Developing antiepileptic drugs in children: balancing protection and access.
This chapter addresses the issue of the balance between protection of children submitted to research and children's access to new treatments, focusing on the field of antiepileptic drugs. Due to the lack of interest pharmaceutical companies have for such an unrewarding market, ethical and legal problems, difficulties recruiting patients, and in Europe insufficient public resources, many infants and children with epilepsy are still exposed to off-label drugs. The incentives and regulations specifically directed toward research in children in the United States and EU provided the first step to change this condition. The challenge is to perform trials determined by the needs of children and to obtain benefits in the same order of magnitude as for adults but with reduced risks. In order to optimize the development of new AEDs in pediatrics, the new European guidelines (2010) recommend the identification of adult clinical trial results that can be extrapolated to the pediatric population (i.e., those in partial epilepsies) and the use of innovative strategies that help limit the number of pediatric patients enrolled in trials (i.e., those with epileptic encephalopathies). A key step will be to develop international networks of pediatric epilepsy centers with the shared purpose of optimizing development and execution of clinical trials.