International Society for Cell & Gene Therapy Process and Product Development Subcommittee, Vancouver, Canada; PerkinElmer, Hopkinton, Massachusetts, USA.
International Society for Cell & Gene Therapy Process and Product Development Subcommittee, Vancouver, Canada; Novo Nordisk, Fremont, California, USA.
Cytotherapy. 2022 Jun;24(6):583-589. doi: 10.1016/j.jcyt.2021.12.012.
Cell and gene therapies are demonstrating clinical efficacy, but prohibitive product costs and operational complexity bottlenecks may limit expanded patient access to these innovative and transformative products. An initial survey and subsequent article published through the International Society for Cell & Gene Therapy in 2017 presented a roadmap on how specific steps, from tissue procurement and material acquisition to facility operation and production, contribute to the high cost of cell and gene therapies. Herein the authors expanded the investigation to provide considerations to better understand how post-production procedures can impact a product's accessibility to patients. The administration of a drug product to and follow-up in a patient involve key decisions in several post-production process areas, such as product storage, distribution and handling logistics and compliance, across the value chain through integrated data management solutions. Understanding as well as carefully evaluating these specific components is not widely considered during early process development but is critical in developing a viable product life cycle.
细胞和基因疗法正在展示临床疗效，但产品成本过高和运营复杂性瓶颈可能限制这些创新和变革性产品扩大患者的可及性。国际细胞与基因治疗学会于 2017 年进行的一项初步调查和随后发表的一篇文章提出了一个路线图，说明了从组织采购和材料获取到设施运营和生产等具体步骤如何导致细胞和基因疗法的高成本。在此，作者扩展了调查范围，以提供更多的考虑因素，更好地了解生产后程序如何影响产品对患者的可及性。药物产品的管理和在患者中的后续治疗涉及到几个生产后流程领域的关键决策，例如在整个价值链中通过集成数据管理解决方案进行产品存储、分发和处理物流以及合规性。在早期工艺开发过程中，人们并没有广泛考虑到对这些特定组件的理解和仔细评估，但这对于开发可行的产品生命周期至关重要。
