Schüle S, Renner M, Longhurst S, Narayanan G
Division of Medical Biotechnology, Paul-Ehrlich-Institut, Langen.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2010 Jan;53(1):30-7. doi: 10.1007/s00103-009-0988-0.
Over the last two decades, clinical trials using gene therapy medicinal products (GTMPs) have been carried out for a large number of rare, inherited monogeneic disorders as well as common multigeneic diseases such as cancer, cardiovascular and infectious diseases including AIDS. Despite some early difficulties and setbacks, the gene therapy field has slowly progressed and, nowadays, offers the promise of novel treatments for a growing number of diseases. On the other hand, gene therapy approaches are often associated with additional risks due to limited clinical experience with a given gene transfer system, long-lasting effects of the therapeutic gene, and/or a complex mode of action. As a result, specific regulations and guidelines have been introduced within the EU to help address these uncertainties. This article summarises the legislative framework and will provide an overview on the regulatory requirements for clinical trials and marketing authorisation applications.
在过去二十年中，针对大量罕见的遗传性单基因疾病以及常见的多基因疾病（如癌症、心血管疾病和包括艾滋病在内的传染病）开展了使用基因治疗药物（GTMPs）的临床试验。尽管早期遇到了一些困难和挫折，但基因治疗领域已缓慢取得进展，如今，为越来越多的疾病提供了新的治疗希望。另一方面，由于特定基因转移系统的临床经验有限、治疗基因的长期影响和/或复杂的作用模式，基因治疗方法往往伴随着额外风险。因此，欧盟已出台具体法规和指南，以帮助应对这些不确定性。本文总结了立法框架，并将概述临床试验和上市许可申请的监管要求。
