【摘要】 新药研发失败率高，需要在快速推进、降低开发风险与成本和科学决策之间取得平衡，就要做到“两个尽早”:尽早廉价淘汰可能失败的药物，尽早获取关键证据，提升后续研发信心，即“Quick win,fast fail”理念。零期临床作为一种早期探索性研究工具，具有剂量低、周期短、受试者少、申报要求灵活的特点，主要用于快速评估候选药物是否具体进一步开发的潜力，更高效地筛选和开发有潜力的候选药物。零期临床策略需要多种特定的技术和人才，包括放射性药物标记技术、高灵敏度分析技术、成像技术、药代动力学和药效学建模、伦理审查和受试者招募，以及多学科团队协作和高度专业化的研究机构。这些技术和人才的有效整合和协作是零期临床研究成功的关键。本文主要介绍了纳入零期临床新药研发新范式的获益-风险评估策略、所需技术、申报要求、及应用案例分析。更多还原

【Abstract】 Novel drug research and development has high failure rate and“a narrow escape from death”,which needs to strike a balance between rapid progress, development risk and cost reduction and scientific decision-making,it is necessary to achieve " two early " : eliminate drugs that may fail as early as possible with little investment,obtain key evidence as early as possible to enhance confidence in subsequent research and development,which is also known as " Quick win,fast fail" concept. Phase 0 clinical trial,as an early exploratory research tool,has the characteristics of low dose,short cycle,small number of subjects,and flexible application requirements. It is mainly used to rapidly evaluate the potential of drug candidates for further development,and more efficiently screen and develop potential drug candidates. The Phase0 clinical trial strategy requires a variety of specific technologies and qualified personnel,including radiopharmaceutical labeling techniques,high-sensitivity analysis techniques, imaging techniques,pharmacokinetic and pharmacodynamic modeling, ethical review and subject recruitment,as well as multidisciplinary team collaboration and highly specialized research institutions. The effective integration and collaboration of these technologies and personnel is the key to the success of Phase 0 clinical trial. This paper mainly introduces the benefit-risk assessment strategy, required technologies, application requirements,and case analysis of the new paradigm for novel drug development incorporating phase 0 clinical trial.更多还原