神経疾患治療薬の世界的な開発動向と日本の創薬の現状

抄録

Neurological and muscular diseases are often difficult to treat, particularly those caused by genetic abnormalities. However, the advent of new therapeutic modalities, such as antisense oligonucleotide drugs and gene therapies, has significantly expanded treatment possibilities. In general, clinical trials for neurological diseases are time–consuming, with a median development period of 87.1 months, longer than other therapeutic areas. A significant issue discussed is the “drug loss” problem, where medications approved overseas remain undeveloped in Japan. This issue, exacerbated by the rise of bioventures in foreign markets, creates disparities in drug availability between Japan and other countries. The need for international cooperation and the evolution of clinical trial methodologies to address these challenges should be discussed, with a focus on improving Japan's drug development capability. Key strategies include promoting collaboration between academia and industry, utilizing real–world data, and fostering international clinical trial networks.