Clinical trial and evaluation guidelines for neurological diseases : domestic and international perspectives

Abstract

Clinical trials for neurological diseases such as amyotrophic lateral sclerosis (ALS) face numerous challenges due to disease–specific characteristics and the significant burden on patients. In Japan and overseas, guidelines for clinical trial design and evaluation have been established for some neurological diseases, contributing to the implementation of high–quality trials. Recently, the utilization of real–world data (RWD) and decentralized clinical trials (DCT) has gained attention, offering the potential to reduce patient burden and improve trial efficiency. These efforts play a crucial role in advancing the development of new drugs for neurological diseases.