Schüssler-Lenz M, Schneider C K
Abteilung Medizinische Biotechnologie, Paul-Ehrlich-Institut, 63225, Langen.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2010 Jan;53(1):68-74. doi: 10.1007/s00103-009-0984-4.
For advanced therapies, the same basic principles for assessment apply as for any other biotechnological medicinal product. Nevertheless, the extent of data for quality, safety, and efficacy can be highly specific. Until recently, advanced therapies were not uniformly regulated across Europe, e.g., tissue engineered products were regulated either as medicinal products or medical devices. Thus, for some products no data from clinical studies are available, e.g., for autologous chondrocyte products. The draft guideline on Good Clinical Practice for clinical trials with advanced therapies describes specific additional requirements, e.g., ensuring traceability. Most clinical studies with advanced therapies in Germany are still in early phase I or II trials with highly divergent types of products and clinical indications. The Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMEA) has been established to meet the scientific and regulatory challenges with advanced therapies.
对于先进疗法，其评估的基本原理与任何其他生物技术药品相同。然而，质量、安全性和有效性的数据范围可能具有高度特异性。直到最近，先进疗法在欧洲并未得到统一监管，例如，组织工程产品要么作为药品要么作为医疗器械进行监管。因此，对于某些产品，没有临床研究数据，例如自体软骨细胞产品。先进疗法临床试验的《药物临床试验质量管理规范》草案描述了特定的额外要求，例如确保可追溯性。德国大多数先进疗法的临床研究仍处于I期或II期早期试验阶段，产品类型和临床适应症差异很大。欧洲药品管理局（EMEA）设立了先进疗法委员会（CAT），以应对先进疗法带来的科学和监管挑战。
