Points to consider for clinical trials of neurodegerative diseases

Abstract

Clinical trials targeting neurodegenerative diseases have particular difficulties in planning and operation due to the complexity of clinical evaluation and the small numbers of patients. Information from clinical trials of pharmaceuticals approved for neurological diseases in Japan is useful. The information of clinical trials can be obtained from package inserts, review reports, and summary of application documents. One of important point of views is whether the test drug is a symptomatic drug or a disease modifying drug. As an example of symptomatic drugs developed in Japan, the review report of zonisamide for Parkinsonism of dementia with lewy body describes association between mechanism of action and endpoints. As an example of disease–modifying drugs developed in Japan, the review report of edaravone for amyotrophic lateral sclerosis describes the selection of endpoints and clinical trial design including treatment period. In addition, guidelines and guidance on clinical trials are useful information for understanding information on clinical trials for drugs approved for neurological diseases. In order to succeed in clinical trials for neurodegenerative diseases and create new evidence, it is important to plan and conduct clinical trials based on the characteristics of the target neurodegenerative diseases.