Abstract：
Advanced therapy medicinal products (ATMPs) are among the most complex pharmaceuticals with high human specificity. Species differences severely limit the clinical relevance of in vivo data. We conducted interviews with stakeholders involved in ATMP development about their perspective on the use of in vivo studies, the perceived hurdles and associated potential solutions regarding non-clinical development of ATMPs. In total, 17 stakeholders from 9 different countries were interviewed. A workshop was held with key stakeholders to further discuss major topics identified from the interviews. Conducting in vivo studies remains the status quo for ATMPs development. The hurdles identified included determining the amount of information required before clinical entry and effective use of limited human samples to understand a treatment or for clinical monitoring. A number of key points defined the need for future in vivo studies as well as improved application and implementation of New Approach Methodology (NAM)-based approach for products within a well-known modality or technology platform. These included data transparency, understanding of the added value of in vivo studies, and continuous advancement, evaluation, and qualification of NAMs. Based on the outcome of the discussions, a roadmap with practical steps towards a human-centric safety assessment of ATMPs was established.
摘要：
高级治疗药物（ATMP）是具有高度人类特异性的最复杂的药物之一。物种差异严重限制了体内数据的临床相关性。我们采访了参与ATMP开发的利益相关者，了解他们对体内研究使用的看法，关于ATMP非临床开发的感知障碍和相关潜在解决方案。总的来说,采访了来自9个不同国家的17名利益相关者。与主要利益攸关方举行了一次研讨会，进一步讨论从访谈中确定的主要议题。进行体内研究仍然是ATMP开发的现状。确定的障碍包括确定在临床进入之前所需的信息量和有效使用有限的人类样品以了解治疗或临床监测。许多关键点定义了对未来体内研究的需求以及基于新方法方法学(NAM)的方法在众所周知的模式或技术平台内的产品的改进应用和实施。这些包括数据透明度，了解体内研究的附加值，不断进步，评估，和NAM的资格。根据讨论的结果，制定了以人为中心的ATMP安全评估的实际步骤的路线图。
