Walter C, Rohde B, Wicke D C, Pohler C, Lührmann A, von der Leyen H
Hannover Clinical Trial Center GmbH, Campus Medizinische Hochschule Hannover, Carl-Neuberg-Str. 1, 30625, Hannover, Deutschland.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2011 Jul;54(7):803-10. doi: 10.1007/s00103-011-1308-z.
Novel therapies, e.g., cell and gene therapy or tissue engineering, are summarized in the European Union as advanced therapy medicinal products (ATMPs). In terms of composition and product properties, ATMPs are highly complex, and given their multiple potential actions they are subject to continuously developing regulatory requirements. Due to promising basic research findings, there are high expectations by the society toward the therapeutic potential of ATMPs. It is of utmost importance to develop a scientifically sound preclinical and clinical development plan before entering into the first clinical trial. Due to the complex features of ATMPs, this development plan should be discussed early with the regulatory authorities to define the specifics and challenges of each individual product. For planning as well as operational realization of the initial clinical trial involving ATMPs, specific requirements that need to be addressed are discussed in this paper.
新型疗法，如细胞和基因疗法或组织工程，在欧盟被归类为高级治疗用医药产品（ATMPs）。就成分和产品特性而言，ATMPs高度复杂，鉴于其多种潜在作用，它们需要遵循不断发展的监管要求。由于基础研究成果令人鼓舞，社会对ATMPs的治疗潜力寄予厚望。在进入首次临床试验之前，制定一个科学合理的临床前和临床开发计划至关重要。鉴于ATMPs的复杂特性，应尽早与监管机构讨论该开发计划，以明确每个产品的具体情况和挑战。本文讨论了在规划以及开展涉及ATMPs的首次临床试验时需要解决的具体要求。
