基因治療運用於人體試驗上之法規範與風險【醫事綜探】 試閱
Is it "gene therapy"?
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。
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 In July 2018 FDA issued six draft guidance documents on “gene therapy,” but none with a substantially new definition.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.
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