從統計角度來判斷藥物臨床試驗所具有之臨床價值

Statistical Judgement on the Clinical Value Resulted from the Clinical Trials

10.29807/JTITAS.200506.0004

歐士田(Shyh-Tyan Ou)

統計原則 ； 臨床試驗 ； 試驗偏差與變異 ； 藥物療效與安全性 ； Statistical principles ； Clinical trials ； Bias and variability of a clinical trial ； Drug efficacy and safety

智慧科技與應用統計學報

3卷1期（2005 / 06 / 01）

73 - 84

繁體中文

當一個活性成分經過臨床前試驗(pre-clinical trial)，包含藥理、毒理、藥動等方面，証實可能具有治療某種疾病之效果時，接下來會有各式各樣的人體臨床試驗被用來確定此活性成分在人體身上是否仍具有療效。臨床試驗結果的臨床價值大小，影響藥物發展的決策。然而，當研究者面對這些不同試驗設計所得結果時，都會產生一個共同困擾，即是到底哪些試驗結果不具有參考價值，哪些試驗結果十分具有參考價值。換言之，判定試驗結果之參考價值高低的準則有哪些？對醫藥發展而言，如何正確判斷藥物臨床試驗所具有之臨床價值，將會影響藥物研究之速度，因而可能造成藥物使用者之不便。本篇文章將針對此一主題，從統計之角度來看，提出結合相關的國際指導方針及個人在藥物臨床試驗之審查經驗的因應之道。文中內容主要包含藥物臨床試驗背景之簡介，臨床試驗計劃書的統計基本考量、樞紐試驗的準則及結論與建議。作者衷心希望本篇文章所提供之訊息，能夠真正幫助國內藥界人士，有效地判定臨床試驗之價值。 本篇文章之內容，純屬個人看法，不代表任何立場。

As a drug is effective for some specific disease, demonstrated by the pre-clinical trials for pharmacology, toxicology, pharmacokinetics and pharmacodynamics, the next step will be to show whether the drug with the active component is still effective on humans with the specific disease through various clinical trials. However, the researchers are frequently bothered to make judgement on the clinical value obtained from various clinical trials. In other words, what criteria do we use to judge the clinical value of a clinical trial? For the drug development, the progress of drug research and the convenience of drug users will be dependent on the accurate judgement on the clinical value of clinical trials. In this article, from the statistical point of view, the solution, obtained from the international guidance and the personal experience on the drug regulation, for the judgement of clinical value of clinical trials will be presented. The main content of this article includes the introduction of clinical trials, the statistical considerations on clinical protocols, and the criteria for a pivotal clinical study. The author truly hopes that this article is helpful for people who are interested on the drug research to know how to make an accurate judgement on the clinical value of a clinical trial. This report represents the view of personal, but such view should not be ascribed to the author's employing institution.