Department of Pharmaceutical Sciences, College of Health Sciences, University of Brasilia, Brasilia, Brazil.
National Health Surveillance Agency (Anvisa), Brasilia, Brazil.
Adv Exp Med Biol. 2023;1430:117-133. doi: 10.1007/978-3-031-34567-8_7.
Advanced therapy products, considered special medications, require Anvisa approval for use and commercialization in Brazil. They include advanced cellular therapy products, tissue engineering products, and gene therapy products, which due to their complexity involve innovation and risks, optimized regulatory channels for their development and life cycle monitoring. The scientific elements and the compliance with applicable regulatory aspects are fundamental pillars for the advancement of clinical trials, the positive evidence of the benefit-risk profile, and the definition of the critical quality attributes, from the perspective of making safe, efficacy, and high-quality products available to the population. The approval models of these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population, with accelerated regulatory analyses, use in emergency situations by risk controls and specific monitoring mechanisms, principally those related to rare diseases without other therapeutic alternatives. The opportune access to the advance therapy product with safety, efficacy, and quality involves innovative normative elements that include the long-term follow-up of the safety and efficacy and of the adaptive pharmacovigilance requisites, as well as the traceability mechanisms for starting materials, products, and patients.
治疗先进产品被视为特殊药物，需要获得 Anvisa 的批准才能在巴西使用和商业化。它们包括先进的细胞治疗产品、组织工程产品和基因治疗产品，由于其复杂性涉及创新和风险，因此需要优化其开发和生命周期监测的监管渠道。科学要素和符合适用监管方面是推进临床试验、证明受益风险概况积极证据以及从确保安全、疗效和高质量产品供应给民众的角度定义关键质量属性的基本支柱。巴西这些产品的批准模式适应技术和患者目标人群的特殊性和特点，采用加速监管分析，通过风险控制和特定监测机制在紧急情况下使用，主要是针对没有其他治疗选择的罕见病。安全、有效和高质量地获得治疗先进产品的机会涉及到创新性的规范要素，包括长期的安全性和疗效监测以及适应性药物警戒要求，以及起始材料、产品和患者的可追溯性机制。
