Abstract：
Despite long-term research achievements, the development of cell therapy (CT) products remains challenging. This is because the risks experienced by the subject and therapeutic effects in the clinical trial stage are unclear due to the various uncertainties of CT when administered to humans. Nevertheless, as autologous cell products for systemic administration have recently been approved for marketing, CT product development is accelerating, particularly in the field of unmet medical needs. The human experience of CT remains insufficient compared with other classes of pharmaceuticals, while there are countless products for clinical development. Therefore, for many sponsors, understanding the rationale of human application of an investigational product based on the consensus and improving the ability to apply it appropriately for CT are necessary. Thus, defining the level of evidence for safety and efficacy fundamentally required for initiating the clinical development and preparing it using a reliable method for CT. Furthermore, the expertise should be strengthened in the design of the first-in-human trial, such as the starting dose and dose-escalation plan, based on a sufficiently acceptable rationale. Cultivating development professionals with these skills will increase the opportunity for more candidates to enter the clinical development phase.
摘要：
尽管取得了长期的研究成果，细胞治疗（CT）产品的开发仍然具有挑战性。这是因为受试者经历的风险和临床试验阶段的治疗效果是不清楚的,这是由于对人类施用时CT的各种不确定性。然而，作为全身给药的自体细胞产品最近已被批准上市，CT产品开发正在加速，特别是在未满足的医疗需求领域。与其他类别的药物相比，CT的人类经验仍然不足，而临床开发的产品则不计其数。因此,对于许多赞助商来说，有必要根据共识了解人类应用研究产品的基本原理，并提高将其适当应用于CT的能力。因此，定义启动临床开发并使用可靠的CT方法进行准备所需的安全性和有效性的证据水平。此外，在首次人体试验的设计中应加强专业知识，如起始剂量和剂量递增计划，基于足够可接受的理由。培养具有这些技能的开发专业人员将增加更多候选人进入临床开发阶段的机会。
