二元反應臨床試驗的二階段設計之實務考量

Practical Considerations on Two-Stage Designs for Binary Outcome in Clinical Trials

林盛峰(Sheng-Fong Lin)

指導教授 ： 歐士田

國立臺北大學/商學院/統計學系/碩士(2008年)

摘要

本篇論文主要是去比較各種不同的第二期臨床試驗中的二階段設計的方法、設計、解決的問題與實際上的使用現況等等，在此介紹的方法反應結果都是二元的，還有在論文中的二階段設計，可以分為兩種，首先是把第二期的前期臨床試驗與第二期的後期臨床試驗
分別視為二階段設計的第一階段與第二階段，再者是在第二期的前期臨床試驗就使用二階段設計。由於Simon的二階段設計是最廣為使用在第二期癌症臨床試驗上，還有調整設計的發展越來越廣，因此特別著重在Simon與調整設計（adaptive design）的探討與比較，
而且是以樣本數為主要考量作為比較各種不同的第二期臨床試驗中的二階段設計，希望在倫理與成本之間取得平衡點，進一步讓法規單位與廠商所接受，並提供使用者在第二期臨床試驗中的二階段設計實務上所遇到的困難的參考文章。

關鍵字

二階段設計 ； 調整設計 ； 樣本數 ； 第二期臨床試驗

並列摘要

The objective of the study is to review a variety of two-stage designs with respect to methods, solutions and uses in practice, etc. The outcomes in all the reviewed methods are binary. Two-stage designs have applied in two situations. First, phase IIA and phase IIB are thought of as stage 1 and stage 2 in two-stage designs respectively. In addition, two-stage designs are used in phase IIA such as Simon two-stage design. Recently, Simon two-stage design is the most popular and well-known in phase II cancer clinical trials. Moreover, an extension of two-stage designs, called adaptive design, is developed. In the clinical research, sample size is the main consideration for comparing a variety of two-stage designs in phase II clinical trials. We hope to strike a balance between ethics and cost, and provide the recommedations for regulations and pharmaceutical companies. All in all, the study is to provide practicers with some useful suggestions when practicers are in a delimma as to which two-stage designs they should choose.

並列關鍵字

Two-Stage Design ； Adaptive Two-Stage Design ； Sample Size ； Phase II Clinical Trial

參考文獻

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