臨床試驗實務之設立規則與執行之研究

藥物開發過程需要很長的開發時間，在基礎研究上進行相當完整的機制研究與藥理毒理研究，而臨床試驗是藥物研發的最後一個最重要的階段。經由臨床人體試驗之結果，確認藥物療效與副作用，藥物才得以上市，造福患者與降低醫療資源耗費，及促進現代醫學發展。臨床試驗為在藥品或醫療器材於上市前所做的試驗評估，分為對照組與實驗組。對照組為已知藥品或安慰劑，實驗組為欲試驗之藥物或醫療材料，藉此比較欲試驗藥物對患者之安全性評估、醫療效果評估與不良反應事件評估，以作為藥品醫材上市前之用藥評估與依據。建立與設計良好的臨床試驗研究，對於整個臨床研究有相當大的助益。臨床試驗分為四期，其中第一期 (phase I) 及第二期 (phase II)，是為了取得更多試驗藥物之訊息，包含藥物安全劑量與可能發生之不良反應。獲得足夠之藥物之訊息後，在第三期 (Phase III) 真正測試藥物之目標療效，而第四期 (Phase IV) 則為針對已經上市之藥物，試著評估若長期使用該藥物是否會產生慢性副作用及確認其療效或與其他藥物之交互作用，所得到的資訊即為藥物的風險效益評估。每一期臨床試驗皆需要病人與醫院相關工作人員參與，所以亟需建立良好且完善的臨床試驗，才能為醫學發展帶來良好的助益。在此研究，利用簡單描述方式介紹臨床試驗，與提供真實案例，讓未參與臨床試驗或有興趣參與之人員對臨床試驗有更多的認識，亦冀希能幫助與鼓勵更多臨床試驗人員，能夠積極參與臨床試驗，與幫助釐清不了解的部分，增加臨床試驗相關工作與法規的知識，使臨床試驗工作能更加順利。並針對臨床試驗之現況不足及需改進的地方加以討論與建議，讓臨床試驗蓬勃發展，促使更多藥物研發成功及順利上市，幫助更多亟需新藥物治療之患者。

關鍵字

臨床試驗；醫療器材；臨床試驗法規

並列摘要

Clinical trial is a very important stage for drug discovery, it usually takes a long time to validate the effect of treatment on clinical. There are four phases in clinical trial, and it need the cooperation of doctors, study nurses, and case managers in every phase. Phase I clinical trial is usually a small trial, and only few cases are enrolled to test the safety of new drugs. In phase II study is usually larger than Phase I, maybe up to 100 cases in use to test the best dose for treatment, and the side effect it is. The Phase III is the most important stage of the clinical trial. It compares with current regular treatment and to know more about its side effects, new way for giving standard treatment, and better treatment in particular cancer or disease. New drugs can be commercial since finishing the Phase III clinical trial. Phase IV is to know more knowledge about side effects of licensed drugs, and its long term risks and benefits. Researchers on bench and medical personnel in clinical are not familiar with clinical trials. In order to provide more information about clinical trials and encourage more experts join clinical trials, I introduce what is clinical trials and provide the protocol and how to execution of clinical trials step by step in this study. From the phases of clinical trial introduction to the regulation of clinical trials, and how to apply the clinical trials process. And giving an example for application of the clinical trial approval to Taiwan Food and Drug Administration and the detail of essential documents filling up.