Salazar-Fontana Laura I
LAIZ Regulatory Science Consulting, Lausanne, Switzerland.
Immunogenicity Integrated, Nay, France.
Front Med (Lausanne). 2022 May 13;9:855100. doi: 10.3389/fmed.2022.855100. eCollection 2022.
Cell and Gene Therapy Products (CGT), regulated as Advanced Therapy Medicinal Products (ATMP) in the European Union (EU), represent a novel and varied group of biotherapeutics developed to treat specific conditions for which there are limited or no effective treatments. The novelty and complexity of this product modality demands a regulatory risk-based approach to define a sound development plan, particularly, as most developers aim to target more than one market area simultaneously for clinical development and registration. This regulatory strategy should be built on solid scientific data that also addresses general regulatory recommendations to enable a benefit:risk analysis that is aligned with the particularities of each CGT product. This risk-based approach is especially helpful when no detailed product-specific regulatory guidelines are available. The goal of this article is to orient developers on how to build a combined EU/US regulatory strategy through the assessment of commonly understood quality (CMC), non-clinical, and clinical regulatory risks faced by ATMP/CGT.
细胞和基因疗法产品（CGT）在欧盟（EU）被监管为高级治疗用医药产品（ATMP），是一类新颖多样的生物疗法，旨在治疗目前有效治疗方法有限或没有有效治疗方法的特定病症。这种产品模式的新颖性和复杂性要求采用基于风险的监管方法来制定合理的开发计划，特别是因为大多数开发者旨在同时针对多个市场领域进行临床开发和注册。这种监管策略应建立在坚实的科学数据基础上，同时也应考虑一般监管建议，以便能够进行与每种CGT产品特殊性相一致的获益-风险分析。当没有详细的针对特定产品的监管指南时，这种基于风险的方法特别有用。本文的目的是通过评估ATMP/CGT常见的质量（CMC）、非临床和临床监管风险，指导开发者如何制定欧盟/美国联合监管策略。
