The European Hospital Exemption Clause-New Option for Gene Therapy?

被引：9

作者：

Buchholz, Christian J. ^{[1
]}

Sanzenbacher, Ralf ^{[1
]}

Schuele, Silke ^{[1
]}

机构：

[1] Paul Ehrlich Inst, Div Med Biotechnol, D-63225 Langen, Germany

来源：

HUMAN GENE THERAPY | 2012年 / 23卷 / 01期

关键词：

X-LINKED ADRENOLEUKODYSTROPHY; LEBERS CONGENITAL AMAUROSIS; MEDICINAL PRODUCTS; LENTIVIRAL VECTOR;

D O I：

10.1089/hum.2011.2529

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Gene-therapy medicinal products are currently applied to patients enrolled in authorized clinical trials to demonstrate safety and efficacy. Given a positive outcome, marketing authorization can subsequently be achieved via the centralized procedure coordinated by the European Medicines Agency. With Regulation (EC) No. 1394/2007 in force, advanced therapy medicinal products, including gene- and cell-therapy products, can be excepted from the obligation of obtaining a marketing authorization via the centralized procedure under specific conditions (so-called "hospital exemption"). This hospital exemption allows the application of gene-therapy medicinal products prepared on a non-routine basis for an individual patient and used under the exclusive professional responsibility of a medical practitioner. Here, we explain the requirements to be fulfilled in order to fall under this exemption, the implementation of this regulation into the German national legislation, and its impact on gene-therapy product development in the future.

引用

页码：7 / 12

页数：6

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