篇名

基因治療運用於人體試驗上之法規範與風險   免費試閱

並列篇名

Is it "gene therapy"?

作者
中文摘要

確切而言,何謂基因治療?市面上已有超過700種為基因治療而設的新藥物研發問世。距離美國食品藥物管理局在1993年第一次定義基因治療至今,已在此領域累積有一段歷史。由於科技發展的日新月異,使得法規範如何定義與規制「基因治療」的必要性,已大於僅只研究其字面上意義的需求。2018年7月,美國食品藥物管理局公布了關於基因治療的六項指導規範草案,這六項草案就基因治療的定義,與1993年相比,在本質上均是了無新意。法規範應對「基因治療」下明確定義,此事之所以重要,主要原因在於當某一藥物被歸類為基因治療藥物後,似乎就暗示著比其他生技治療還更進步、更高檔,其價格亦因此而飆升。但最終說回來,將某種治療模式稱之為「基因治療」而與其他種類的治療予以區分,最主要的還是在法規範分類劃分上,由立法動機明文後,往後在流水線的個案申請許可上、在特定藥商製造的指導方針上、在藥價的制定上、在公領域的囤藥考量上,都有其類型化的意義。

英文摘要

What, exactly, is “gene therapy”? As of this writing, there are more than 700 active investigational new drug applications for “gene therapies”. And yet, FDA”s definition of “gene therapy” dates back to 1993 — ancient history relative to recent advances in the field. Crystallizing a modern definition of “genetherapy”, concerning what constitutes “gene therapy” is important beyond mere semantics. In July 2018 FDA issued six draft guidance documents on “gene therapy,” but none with a substantially new definition. The term also carries significant baggage regarding drug pricing, with “gene therapies” seeming to command higher premiums relative to other advanced biotherapeutics. Ultimately, the difference between calling a therapy a “gene therapy” and something else is the difference between a slate of regulatory incentives, streamlined approval, specific manufacturing guidances, and, likely, premium prices and public cache.We review the contours of the term here, and propose that “gene therapy” — in the lay, scientific, and legal sense—should be defined as an intentional and expected permanent alteration of a specific DNA sequence of the cellular genome. Defining gene therapy in this fashion would provide clarity to scientists and regulators alike.

起訖頁

138-144

出版單位
DOI

10.3966/241553062020090047012  複製DOI  DOI查詢

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