Regulatory Aspects for Drug Development

Translating preclinical epilepsy therapy research into clinical therapeutic products requires an understanding of the regulatory requirements pertaining to drug development. In this chapter, we discuss regulatory considerations in the development of new molecular entities, or the repurposing of approved drugs for the treatment of epilepsy, as an investigator moves a potential drug therapy through discovery, preclinical and clinical development, and approval. We provide an overview of relevant guidances and harmonization by regulatory agencies, and the regulatory process and requirements for studying drugs in humans. Specifically, this chapter summarizes the process for submitting and maintaining an investigation new drug (IND) application, and provides an overview of the required IND content, including preclinical pharmacology and toxicology study summaries; chemistry, manufacturing, and controls information; previous human experience; and the clinical investigation plan. These components are discussed with an emphasis on preclinical testing specific for epilepsy. Last, the topics of repurposing an approved drug and obtaining orphan drug designation are highlighted as special considerations that may be important for investigator-initiated research in epilepsy.