Department of Global Regulatory Affairs, Vifor Pharma Ltd, Glattbrugg, Switzerland.
Nanomedicine (Lond). 2015 Mar;10(4):659-74. doi: 10.2217/nnm.14.189.
Nanomedicines are highly complex products and are the result of difficult to control manufacturing processes. Nonbiological complex drugs and their biological counterparts can comprise nanoparticles and therefore show nanomedicine characteristics. They consist of not fully known nonhomomolecular structures, and can therefore not be characterized by physicochemical means only. Also, intended copies of nanomedicines (follow-on similars) may have clinically meaningful differences, creating the regulatory challenge of how to grant a high degree of assurance for patients' benefit and safety. As an example, the current regulatory approach for marketing authorization of intended copies of nonbiological complex drugs appears inappropriate; also, a valid strategy incorporating the complexity of such systems is undefined. To demonstrate sufficient similarity and comparability, a stepwise quality, nonclinical and clinical approach is necessary to obtain market authorization for follow-on products as therapeutic alternatives, substitution and/or interchangeable products. To fill the regulatory gap, harmonized and science-based standards are needed.
纳米药物是高度复杂的产品，是难以控制的制造工艺的结果。非生物复杂药物及其生物对应物可以包含纳米颗粒，因此表现出纳米药物的特征。它们由不完全了解的非同分子结构组成，因此不能仅通过物理化学手段来表征。此外，纳米药物的预期复制品（后续类似物）可能具有临床意义上的差异，这给监管带来了挑战，即如何为患者的利益和安全提供高度保证。例如，目前非生物复杂药物预期复制品的上市许可监管方法似乎并不合适；此外，还没有定义一种有效的策略来整合此类系统的复杂性。为了证明足够的相似性和可比性，需要采用逐步的质量、非临床和临床方法，为后续产品获得市场授权，作为治疗替代品、替代产品和/或可互换产品。为了填补监管空白，需要制定协调一致的、基于科学的标准。
