title: The European hospital exemption clause-new option for gene therapy?
abstract: 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.
authors.affiliation: Division of Medical Biotechnology, Paul-Ehrlich-Institut , 63225 Langen, Germany. christian.buchholz@pei.de
authors.forename: Christian J
authors.initials: CJ
authors.lastname: Buchholz
authors.forename: Ralf
authors.initials: R
authors.lastname: Sanzenbacher
authors.forename: Silke
authors.initials: S
authors.lastname: Schüle
mesh_terms: D017690:Cell Transplantation / Q000331:legislation & jurisprudence; D002986:Clinical Trials as Topic; D003257:Consumer Product Safety / Q000331:legislation & jurisprudence*; D004345:Drug Industry / Q000331:legislation & jurisprudence; D005060:Europe; D005062:European Union; D015316:Genetic Therapy / Q000331:legislation & jurisprudence*; D017408:Guidelines as Topic; D006801:Humans; D007882:Legislation, Hospital; D040541:Marketing / Q000331:legislation & jurisprudence; D023822:Tissue Engineering / Q000331:legislation & jurisprudence