A Study of the Legal Issues on Off-Label Use

Abstract

[[abstract]]藥物的開發與上市需經繁複的程序與鉅額的金錢花費，歷經實驗室研究、前臨床試驗階段、人體臨床試驗階段等多年的過程，才能取得上市許可。藥物經過衛生主管機關核可上市，須將使用適應症、劑量、使用路徑及適用年紀、族群，刊載於仿單及藥物包裝上。然藥物可能具有多種療效，雖實際上未取得衛生主管機關的核可，但卻普遍應用於臨床治療上，此即藥物標示外之使用（off-label use）。根據研究調查，藥物標示外之使用為一普遍現象，其使用率約佔所有處方之百分之二十。法令上對於藥物標示外之使用，並未有明確之規範，因此產生許多醫學倫理與法律責任之爭議。 藥物標示外使用既為醫界普遍存在之現象，醫師在診治病人時，無法避免藥物標示外使用，但是否須在使用前，告知病患處方為藥物標示外使用或該使用屬於衛生主管機關所核准之適應症，美國與我國之見解，素有不同。美國法院認為，藥物標示外使用不屬於醫療固有實質之風險，醫師不具告知同意之義務；然我國衛生主管機關及法院認為，醫師在處方藥物標示外使用前，應先取得病人之知情後同意，因而對於違反該義務之醫師，賦予相關之民、刑事及行政責任。 本文認為醫療行為之目的，乃在救治病人生命及恢復病人之健康，其過程本就充滿不確定性與高度危險性，醫療行為之歸責，應以可合理期待之安全性為考量。藥物標示外使用自是醫師臨床之自由裁量，不宜以法律全面限縮，然為避免醫療之濫用，亦不宜自由放任醫師恣意而為之。因此，本文建議，藥物標示外使用應以實證醫學為基礎，以風險為考量，以病人為中心，規範藥物標示外使用之原則及使用流程；並適當地修訂法律，明確定義藥物標示外使用及其合法性，訂定告知義務之範圍，且修訂藥害救濟法，對於具有高強度實證支持之藥物標示外使用，予以救濟。 醫學乃是基於科學之藝術，過度限縮或自由開放藥物標示外使用，皆不適宜。唯有適當法律管制，並兼顧醫師用藥自由裁量，才能確保用藥安全，增進全民之健康福祉。[[abstract]]All new drugs and medical device products must undergo a rigorous process of pre-clinical and clinical evaluation which is known to be complex costly and time-consuming After marketing approval the labels on drug packages should outline the indications for use dosage directions and safety information The use of drug and medical device products could expand over time Off-label use which means prescribing an approved drug for a use that is not claimed in the product information is common in medical practice especially for some serious or rare diseases As found in several reports the rates of off-label use were found to account for 20% of all prescriptions and most of the off-label drug uses were shown to have little or no scientific support Since there is no regulation for off-label use a number of clinical legal and ethical issues arise Off-label use of products is usually unavoidable but the legal liability is different between the U S and Taiwan In the U S physicians are required to explain medical risks medical benefits medical treatment and medical conditions The informed consent responsibility is limited to nondisclosure of relevant medical information Off-label use is not a material risk and physicians need not disclose the FDA status of drugs or medical device products In Taiwan however the court and the Department of Heath argue that informed consent is necessary before prescribing off-label products Physicians may incur civil criminal and administrative liability if they breach the duty of informed consent of off-label prescriptions Medicine is intended to save patients’ lives and to help patients reach renewed health During the process of treatment the risk versus benefit profile beyond the original product approval should first be taken into consideration Medicine is art based on science The decision whether or not to use a drug for an off-label purpose is a matter of medical judgment Clinical freedom should be applied only in the best interest of patients Too much legal interference will limit physicians’ access to potentially valuable medications However if there is no high-quality evidence of support off-label use may be only bad medicine which flies the flag of clinical freedom Finally this study has established the principles and flow chart of off-label use We suggest that some amendments to the laws are necessary including clear definition of off-label use setting up the scope of the duty of disclosure as well as revision of Drug Hazard Relief Act for relief of people injured by off-label use products with support of high-quality evidenc