Introduction
According to the news (ALS薬、国内初の治験GL策定へ 和泉研究班、最新手法で開発促進・ラグ解消 | 日刊薬業 – 医薬品産業の総合情報サイト (jiho.jp)) released by NIKKAN YAKUGYO as of 10/05/2024, a research team led by Professor Yuinobu Izumi at Tokushima University is set to develop Japan’s first clinical trial guidelines for Amyotrophic Lateral Sclerosis (ALS) treatments this spring. The aim is to integrate the latest methodologies to expedite new drug development and reduce drug lag and loss, with a completion target for the fiscal year 2026.
Urgent Need for New ALS Treatments
Japan has around 10,000 ALS patients, and existing treatments offer limited efficacy and only modest benefits in slowing disease progression. New treatments like Biogen’s Tofersen and Amylyx Pharmaceuticals’ AMX0035 have shown promise internationally, raising calls for their early approval in Japan.
Addressing Clinical Trial Challenges
Japan lacks standardized clinical trial guidelines, hindering efficient and timely drug evaluations. This new project will create guidelines inspired by Western models but adapted to Japanese clinical evidence. Recommendations will include innovative trial designs, such as platform and adaptive trials, and biomarkers for better patient selection and efficacy assessments.
Conclusion
By developing these guidelines, the team seeks to align Japanese ALS research with international standards, facilitating the rapid development of effective treatments for ALS patients.