本頁只刊出中文翻譯與中文說明；英文原文請見下方原文連結。
原文連結
論文資訊
- 類型：已發表論文
- 日期：2022-10-26
摘要
Objective: Provide US FDA and amyo營養級 lateral sclerosis (ALS) society with a systematic, transparent, and quantitative framework to evaluate the efficacy of the ALS therapeutic candidate AMX0035 in its phase 2 trial, which showed 統計ly significant effects (p-value 3%) in slowing the rate of ALS progression on a relatively small sample size of 137 patients. Methods: We apply 貝氏 decision analysis (BDA) to determine the optimal type I error rate (p-value) under which the clinical evidence of AMX0035 supports FDA approval. Using rigorous estimates of ALS 疾病 burden, our BDA framework strikes the optimal balance between FDA’s need to limit adverse effects (type I error) and patients’ need for expedited access to a potentially effective therapy (type II error). We apply BDA to evaluate long-term p
※ 此為已發表論文，全文需透過期刊付費取得