「真實世界証據(RWE)」於新藥上市審批之法規探討：以台灣新藥查驗登記為例

Use「Real World Evidence」on Regulation of New Drug Application (NDA) in Taiwan

吳秉珊(Bing-Shan WU)

指導教授 ： 鄭慧文

臺北醫學大學/藥學院/藥學系(碩博士班)/碩士(2019年)

摘要

為減輕聯邦政府的癌症研究負擔、改善精神健康服務的提供、解決鴉片類藥物濫用之問題。美國參議院於2016年通過「21世紀治愈法案 ( 21st Century Cures Act )」，並於第3022條款中，新增一條修正條款：「利用真實世界證據(Utilizing Real World Evidence)」，期望有助於加速醫療產品之創新、減輕監管負擔、加強科學和方法研究的力度。法案之通過更是牽動全球生醫和保健領域的發展。FDA早先於2017年8月發布「利用真實世界證據支持醫療器械的監管決策指引 (Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices—Guidance for Industry and Food and Drug Administration Staff)」；對於RWE應用於藥品之法規制定，直至2018年12月才發布「FDA 真實世界證據計劃的框架」(Framework For FDA’s Real-World Evidence Program)。
長久以來，我國對於藥品研發制定之法規規範，多半參考美國（FDA）、歐盟（EMA）以及國際醫藥法規協合會（ICH）作為修訂標準。儘管我國已訂立並修正許多加速藥品上市之審查程序，但伴隨「利用真實世界證據(Utilizing Real World Evidence)」議題之浮現，筆者期望透過情境分析法之模式，對先前已核准上市的藥品，可遵循利用真實世界數據之精神，分別探討上市後藥品之新適應症、新複方以及轉類的，並針對現行法條可修正的部分進行討論，以支持先前已核准上市藥品之證據需求，加速藥品上市之審查時程。
伴隨著21世紀治愈法案的通過也逾2年時間，今年4月，針對使用真實世界證據用於上市後藥品新適應症之申請有一大突破。用於治療乳腺癌的藥物Ibrance根據詳細說明該藥實際使用情況的電子健康記錄 (electronic health records, EHRs) 和上市後報告中獲得的真實世界數據 (Real-World Data, RWD)，取得美國食品藥物管理署核准用於男性乳腺癌。這項突破勢必將牽動全球抑或是台灣醫藥產業界的發展與政策制定之衝擊。

關鍵字

真實世界証據 ； 真實世界數據 ； 新藥查驗登記

並列摘要

To alleviate the government's burden of cancer research, improve the provision of mental health services, and address the issue of opioid abuse, the US Senate passed the 21st Century Cures Act in 2016 and added a new amendment into the Section 3022: “Utilizing real-world evidence”. It is expected to accelerate Innovation in medical products, reduce regulatory burdens, and strengthen scientific and methodological research. The adoption of the Cures Act. is also affecting the development of the global biomedical and health care fields. The US FDA released the “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices—Guidance for Industry and Food and Drug Administration Staff” in August 2017; and the“Framework For FDA’s Real-World Evidence Program” is the guidance focus on pharmaceuticals, not released by the FDA until December 2018.
Regulations on drug researches and developments in Taiwan have mostly been following the same laws as those of the United States (FDA), the European Union (EMA), and other countries due to being acknowledged as the standards. With the emergence of the topic of “Using Real-World Evidence (RWE)”, we considered that will be a chance to improve the process of submitting the application for regulatory of drug registration. We used the pattern of Scenario analysis, meaning that drugs that have previously been approved for listing could follow the idea of using RWE in the section 3022 of the 21st Century Cures Act.
In this study, we will focus on evaluating the potential of using of RWE as to help support the approval of a new indication, combinations, and Rx-to-OTC switches for an already approved drug, or to help support or satisfy post-approval study requirements. At the end of this study, we will also discuss on how to revise the current 'Regulations for Registration of Medicinal Products' as to support the evidence’s needs and speed up the process for the drug listing process. There are many countries, including Taiwan FDA, will continue to develop the ways to evaluate and explore the benefits of optimizing RWE. In addition, regarding the reliability and relevance for the data collecting and analyzing, we also looking forward to the collaboration with the stakeholders and patients for understanding what are their perspectives in the process of developing a RWE program.

並列關鍵字

Real World Evidence (RWE) ； Real World Data (RWD) ； New Drug Application (NDA)

參考文獻

論文主體

[1] Proposed FDA Work Plan for 21st Century Cures Act Innovation Account Activities. (2017).

[2] Senate and House of Representatives of the United States of America in Congress, 21st century cure act. Description (2016).

[3] R. E. Sherman, et al. Accelerating development of scientific evidence for medical products within the existing US regulatory framework. Nat Rev Drug Discov 2017;16:297-298.

[4] U.S. Food and Drug Administration, 21st Century Cures Act. (2018), available at https://www.fda.gov/regulatoryinformation/lawsenforcedbyfda/significantamendmentstothefdcact/21stcenturycuresact/default.htm.