“如果FDA根据相关科学确定,1项充分且良好对照的临床研究的数据和确证性证据足以确定有效性,FDA可认为这项数据和证据构成实质性证据。”尽管自1998年以来,FDA这一有效性证据标准没有改变,但药物开发和科学不断发展,使药物开发方案的性质发生变化,需要提供更多指导原则阐明该标准的灵活性。为满足这种需求,美国食品药品管理局(FDA)于2023年9月发布了“通过1项充分且良好对照的临床研究和确证性证据证明有效性的实质性证据的供企业用的指导原则草案”,对FDA的2019年有效性指导原则草案和1998年有效性指导原则做了补充。该指导原则描述了在评估1项充分且良好对照的临床研究和确证性证据是否足以证明有效性的实质性证据时,需要考虑的因素,还提供了确证性证据的数据类型的示例。我国目前还没有类似的指导原则,详细介绍该指导原则,期望对我国这方面的研究和监管有益。

“If [FDA] determines, based on relevant science, that data from one adequate and well-controlled clinical investigation and confirmatory evidence are sufficient to establish effectiveness, [FDA] may consider such data and evidence to constitute substantial evidence.” Although the Food and Drug Administration(FDA)'s evidentiary standard for effectiveness has not changed since 1998, drug development and science have continued to evolve, leading to changes in the nature of drug development programs,more guidances need to be provided to clarify the flexibility of this standard. To meet this demand, FDA released the "Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence Draft Guidance for Industry" in September 2023, supplementing the FDA's 2019 Effectiveness draft guidance and 1998 Effectiveness guidance. This guidance describes factors to consider when assessing whether a single adequate and well-controlled clinical investigation and confirmatory evidence are sufficient to demonstrate substantial evidence of effectiveness,and also provides examples of the data types of confirmatory evidence. There is currently no similar guidance in our country. This article provides a detailed introduction to the FDA's guidance, hoping to be beneficial for research and regulation in this area in China.

R951