Sipos Bence, Katona Gábor, Csóka Ildikó
Faculty of Pharmacy, Institute of Pharmaceutical Technology and Regulatory Affairs, University of Szeged, Eötvös Str. 6, H-6720 Szeged, Hungary.
Pharmaceutics. 2021 May 12;13(5):702. doi: 10.3390/pharmaceutics13050702.
Nanoparticle research and development for pharmaceuticals is a challenging task in the era of personalized medicine. Specialized and increased patient expectations and requirements for proper therapy adherence, as well as sustainable environment safety and toxicology topics raise the necessity of well designed, advanced and smart drug delivery systems on the market. These stakeholder expectations and social responsibility of pharma sector open the space and call new methods on the floor for new strategic development tools, like Quality by Design (QbD) thinking. The extended model, namely the R&D QbD proved to be useful in case of complex and/or high risk/expectations containing or aiming developments. This is the case when we formulate polymeric micelles as promising nanotherapeutics; the risk assessment and knowledge-based quality targeted QbD approach provides a promising tool to support the development process. Based on risk assessment, many factors pose great risk in the manufacturing process and affect the quality, efficacy and safety profile. The quality-driven strategic development pathway, based on deep prior knowledge and an involving iterative risk estimation and management phases has proven to be an adequate tool, being able to handle their sensitive stability issues and make them efficient therapeutic aids in case of several diseases.
在个性化医疗时代，用于制药的纳米颗粒研发是一项具有挑战性的任务。患者对适当治疗依从性的特殊且不断提高的期望和要求，以及可持续环境安全和毒理学问题，凸显了市场上设计精良、先进且智能的药物递送系统的必要性。制药行业的这些利益相关者期望和社会责任为新的战略发展工具（如质量源于设计（QbD）理念）开辟了空间并催生了新方法。扩展模型，即研发QbD，在涉及复杂和/或高风险/高期望的开发项目中已证明是有用的。当我们将聚合物胶束制备成有前景的纳米治疗药物时就是这种情况；基于风险评估和基于知识的质量靶向QbD方法为支持开发过程提供了一个有前景的工具。基于风险评估，许多因素在制造过程中构成重大风险并影响质量、疗效和安全性。基于深入的先验知识以及包含迭代风险估计和管理阶段的质量驱动战略发展途径已被证明是一种适当的工具，能够处理它们敏感的稳定性问题，并使其在多种疾病中成为有效的治疗辅助手段。
