Accelerating drug development for amyotrophic lateral sclerosis: construction and application of a disease course model using historical placebo group data.

Ruifen CaiJuan YangLijuan WuYixiao LiuXinrui WangQingshan ZhengZewei Li

Published in: Orphanet journal of rare diseases (2024)

The established disease course model of the historical placebo group is valuable in the decision-making process for the clinical development of ALS drugs. It serves not only as an external control to evaluate the efficacy of the tested drug in single-arm trials but also as prior information that aids in accurately estimating the posterior distribution of the disease course in the placebo group during small-sample clinical trials.

Keyphrases

amyotrophic lateral sclerosis
decision making
emergency department
big data
antiretroviral therapy
health information
deep learning
placebo controlled
data analysis