論申請新藥上市核准的有效性判斷──以美國法制為論述中心

Study on the Effectiveness Judgment of Approving the Application for New Drug: U.S. Legal System as the Center

關於藥品的安全性與有效性，係一項新藥／學名藥／生物產品申請上市核准的基本要件，俾以保障國民身體健康，並減少其財產支出。為此，各國立法及藥政機關莫不制訂嚴格的新藥上市核准要件規定及關於申請試驗用藥品臨床試驗及申請新藥（含學名藥）上市與FDA審查的嚴密規範。其中，關於藥品的有效性判斷部分，乃是整個藥事法領域最核心的基本問題。本文試著以美國法制為經，兼以ICH所公布的各項原則為緯，從規範面、產業面及科學面三個面向探討美國有關申請新藥上市核准有效性判斷之相關問題，並闡明藥品有效性的內涵。例如從產業面向探討製藥業者如何調控及設計其藥品將具有其宣稱的或所表示要具有的效果及其與藥品有效性的關係；從規範面向闡明證明藥品有效性的實質證據之內容、組成及數量，包括充分的且受良好控制的試驗，及隨機、盲性及對照試驗的黃金標準等；從科學面向探討如何證明藥品有效性的試驗設計（如顯著性試驗、優效性試驗、等效性試驗及非劣效性試驗等），及統計上顯著性、臨床上顯著性之意義、如何檢定或測量？如何成為藥品有效性之法定要求的要素？以及在其藥品行政與司法實務上適用之爭議等，以期較全面地理解藥品有效性之內涵，釐清該領域之有關疑點，充實我國藥事法領域的研究能量，並對後進者有所助益。

Regarding the safety and effectiveness of drugs, it is a basic requirement for the approval of a NDA/ANDA/BLA, beneficial to protect the health of the people and reduce their property expenses. To this end, all the national legislations and the authoritues of drug administrations in the world have stipulated strict legal requirements for the approval of new drug listings and strict law and specifications to regulate a IND/NDA/ANDA/BLA filed by a spnsor/applicant respectively and govern the FDA to review and approve. Among them, the judgment on the effectiveness of drug is the core problem in the whole field of pharmaceutical law. This article tries to explore the American legal system as the basis and the guidelines published by ICH as the latitude. From the normative, industrial and scientific aspects, it discusses the relevant issues concerning how to approve a NDA and ANDA in U.S.A., and clarifies the connotation of effectiveness of the drug. For example, from the industrial perspective, how the pharmaceutical industry regulates and designs the drug will have the effect it purports or is represented to have and its relationship with the effectiveness of the drug; from the normative aspect, this article will elucidate the content, composition and quantity of substantial evidence to prove the effectiveness of the drug, including adequate and well-controlled investigations/trials, and gold standards for randomized, blinded, and controlled trials; from the scientific aspect, this article will explore the design of trials of how to demonstrate the effectiveness of drugs (e.g., significance trial, superiority trial, equivalence trial, and non-inferiority trial etc.), and the meanings of the statistical significance, clinical significance, how to test or measure? how to become a element of legal requirement for the effectiveness of drug? and their applicable disputes in the drug administration and judicial practice, expecting more comprehensively to understand the connotation of effectiveness of the drug, clarify the relevant doubts in the field, and enrich the research energy in the field of pharmaceutical law in Taiwan (R.O.C.) and will help the latecomers.