ALS治療薬臨床評価ガイドライン作成の目的と概要

抄録

Many clinical trials for amyotrophic lateral sclerosis (ALS) drugs are being conducted both in Japan and overseas in hopes of finding further benefits. There are currently no guidelines for clinical evaluation of ALS in Japan when conducting clinical trials. As a result, new clinical evaluation systems, such as platform trials and the use of biomarkers, have not been fully evaluated and utilized. To improve this situation, we will be working on the “Study on the Creation of Clinical Evaluation Guidelines for Drugs for Amyotrophic Lateral Sclerosis” from April 2024, commissioned by the Japan Agency for Medical Research and Development (AMED). The purpose of this study is to create clinical evaluation guidelines that correspond to those overseas, such as those of the European Medicines Agency and the US Food and Drug Administration. In order to propose an international standard that incorporates cutting–edge methods for clinical evaluation of ALS drugs and leads the world, an English version will be created in addition to the Japanese version.

By publishing this guideline, it is expected that the situation of ALS clinical trials in Japan will be greatly improved and will further promote international ALS drug development.

In addition, this study will also work on international harmonization of ALSFRS–R standard operating procedures (SOP). The ALSFRS–R also has a problem with variation in assessments between assessors, which may lead to failures in ALS clinical trials. To improve this situation, we plan to exchange ideas with the Northeast ALS Consortium (NEALS), the European network to cure ALS (ENCALS), and the Treatment Research Initiative to Cure ALS (TRIALS) to promote harmonized SOP.