Niazi Sarfaraz K
College of Pharmacy, University of Illinois, Chicago, IL 60612, USA.
Pharmaceutics. 2024 Jul 10;16(7):918. doi: 10.3390/pharmaceutics16070918.
Drug development costs can be significantly reduced if proven "platform" technologies are allowed to be used without having to validate their use. The most recent US Food and Drug Administration (FDA) guideline brings more clarity, as well as a greater focus on the most complex technologies that can now be used for faster drug development. The FDA has highlights the use of lipid nanoparticles (LNPs) to package and deliver mRNA vaccines, gene therapy, and short (2-20 length) synthetic nucleotides (siRNA). Additionally, monoclonal antibody cell development is targeted. The FDA provides a systematic process of requesting platform status to benefit from its advantages. It brings advanced science and rationality into regulatory steps for the FDA's approval of drugs and biologicals.
如果允许使用经过验证的“平台”技术而无需对其使用进行验证，药物开发成本可以显著降低。美国食品药品监督管理局（FDA）的最新指南带来了更多的清晰度，同时也更加关注那些现在可用于加快药物开发的最复杂技术。FDA强调了使用脂质纳米颗粒（LNP）来包装和递送mRNA疫苗、基因疗法以及短（2至20个长度）的合成核苷酸（siRNA）。此外，单克隆抗体细胞开发也在目标范围内。FDA提供了一个申请平台地位以受益于其优势的系统流程。它为FDA批准药物和生物制品的监管步骤带来了先进的科学性和合理性。
