論未經核准之試驗用藥品的擴大使用法制──以美國法制為論述核心

The Legislation of Expanded Access to the Unapproved Investigational New Drugs—US Legal System as the Core

「擴大使用」，亦稱「體恤使用」，係指罹患嚴重的或危及生命的疾病之病患或末期病患，在無可比較的或可替代的治療選項時，得獲准使用有希望治療其疾病且尚未獲准上市之試驗用藥品者。美國自一九六二年所確立之藥品管制法制，要求申請人必須以實質證據證明其藥品的安全性與有效性，始能獲准上市。FDA據此建立了嚴格的臨床試驗標準，進行充分的且受良好控制的試驗，以隨機分配、雙盲及對照試驗之方式，藉由科學與醫學的共同判斷，以檢驗與評價該試驗用藥品的安全性與有效性，並確保公眾的健康與用藥安全。然而，一項新藥從開發至被獲准上市，平均需耗時十至十五年，此使每年數十萬名乃至百萬名罹患嚴重的、危及生命的疾病病患及末期病患，蒙受痛苦卻無藥可救的困境，並在絕望中死去。為回應上開病患的特殊需要，國會及FDA不得不創設例外的試驗用新藥的擴大使用法制，以緩和嚴格的藥品管制法制。甚至病患聯盟仍進一步向聯邦法院提起訴訟，各州接著從事試驗權立法，以保障上開病患獲得使用已通過第一期臨床試驗的試驗用藥品之權利。本文所探討之未經核准的試驗用藥品之擴大使用法制，係以美國法制為論述核心，簡述美國聯邦藥品管制的立法史、新藥申請程序概觀、詳論試驗用藥品的擴大使用法制的演進、標準與類型，及其實務面的各方利益分析與運作的缺失、病患與州對於試驗用藥品擴大使用法制的挑戰等。本文囿於篇幅，僅以「藥品」為論述範圍，醫療器材及生物產品暫略。

“Expanded access”, also known as “compassionate use”, refers to a patient suffering from a serious or threatening life disease or a terminal patient would access to a promising investigational new drug (IND) which has not yet been approved to market, to treat or cure his/her disease when there is no comparable or alternative option of treatment. The legislation on drug regulation established by the United States in 1962, requires the applicant should submit the substantial evidence to demonstrate the safety and effectiveness of the drug before the approval of drug marketing. The FDA has established a strict standard of the clinical trial, conducted adequate and well-controlled investigations, in a maner of randomized, double-blinded, placebo-controlled trial, and by scientific and medical judgment to verify and evaluate the safety and effectiveness of IND and to ensure the safety and health of the public. However, it takes 10 to 15 years for a new drug from development to being approved to market, to make hundreds of thousands or even millions of patient suffering from serious, life-threatening and terminal patient without any medicine to save their plight, and died in despair. In response to the special needs of said patients, the Congress and FDA had to create exceptionally the legislation of expanded access to use the IND to mitigate the strict relugatory system of drugs. Even the Patient Alliance was still further litigation to the federal court, and the states then engaged in the Right to Try legislation to protect the patients to obtain a right of access to experimental drugs that have passed the first phase of clinical trials. The legislation of expanded access to the unapproved IND explored by this article is to discuss the legal system of United States as a core, including the history of legislation on drug regulation in the United States, the overview of the process of application for the IND, to elaborate the evolution, standard, category of the legislation of expanded access to the IND, and to analyze the interests of the involved parties and the lacks of operation in the practical aspects, and the patients and the states try to challenge the legislation of expanded access to the IND. This article is limited by space, only “drugs” for the scope of discussion, medical devices and biological products would be omitted temporarily.