临床试验疗效评估之统计基本观念.pdfVIP

臨療念 臨 ABSTRACT The approval and the market of a new drug in the claimed indication need sufficient evidence of efficacy and safety from clinical trials. In Taiwan as well as over the world, protocols of the clinical trials are required to be reviewed and approved by regulatory agency before its initiation. The essential role of statisticians in the regulatory review is well recognized for the reason that they lead the judgment of a scientifically meaningful data from the clinical trial. Henceforth, the critical role of clinical trials in the drug approval process coupled with the important role of statisticians in the regulatory review team necessitates a succinct document of statistical issues in regulatory review. In this article, it will first be introduced briefly three phases of clinical trials in drug development. Following for a scientifically meaningful clinical trial, issues that are often debated will be offered (and discussed) with regards to sample size, primary efficacy endpoint, non-inferiority/equivalence margin, data analysis, etc. (introduction) 臨臨 (phase I clinical trial)臨理療 (healthy adult) 量 (maximum tolerable dose, MTD), 量量(single dose) 量 (repeated dose) (absorption, distribution, metabolism, and elimination, ADME) 力(pharmacokinetics) (pharmacodynamics)臨臨 (phase II clinical trial)療 (efficacy)臨 識 (indication) 臨 量療 臨臨 (exploratory) 量療(dose-response reaction) 臨療 數量() 不療臨 紐(pivotal trial) 度切 臨臨 量 降量(MTD) 量累(dose escalation)量 ( 10%量LD101/10) 量 量(dose limited toxicity, DLT)不率 ( 1/3)量 臨數(不 20 ) 量療(descriptive statistics) 臨療 臨 數力 (power) 漏療數 易臨數 論 (primary endpoint) 療臨 (indication) 療療聯療 (surrogate endpoint) 流行療聯 若 率 說類(type