PDF(Pubmed)
Abstract：
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.
摘要：
如果允许使用经过验证的“平台”技术而无需验证其用途，则可以显着降低药物开发成本。最新的美国食品和药物管理局（FDA）指南带来了更多的清晰度，以及更加关注现在可以用于更快的药物开发的最复杂的技术。FDA强调使用脂质纳米颗粒（LNPs）包装和提供mRNA疫苗，基因治疗,和短(2-20长度)合成核苷酸(siRNA)。此外,单克隆抗体是靶向细胞发展。FDA提供了请求平台状态以从其优势中受益的系统过程。它将先进的科学和合理性纳入FDA批准药物和生物制品的监管步骤。
