“如果FDA根據(jù)相關(guān)科學(xué)確定,1項充分且良好對照的臨床研究的數(shù)據(jù)和確證性證據(jù)足以確定有效性,FDA可認為這項數(shù)據(jù)和證據(jù)構(gòu)成實質(zhì)性證據(jù)。”盡管自1998年以來,FDA這一有效性證據(jù)標(biāo)準(zhǔn)沒有改變,但藥物開發(fā)和科學(xué)不斷發(fā)展,使藥物開發(fā)方案的性質(zhì)發(fā)生變化,需要提供更多指導(dǎo)原則闡明該標(biāo)準(zhǔn)的靈活性。為滿足這種需求,美國食品藥品管理局(FDA)于2023年9月發(fā)布了“通過1項充分且良好對照的臨床研究和確證性證據(jù)證明有效性的實質(zhì)性證據(jù)的供企業(yè)用的指導(dǎo)原則草案”,對FDA的2019年有效性指導(dǎo)原則草案和1998年有效性指導(dǎo)原則做了補充。該指導(dǎo)原則描述了在評估1項充分且良好對照的臨床研究和確證性證據(jù)是否足以證明有效性的實質(zhì)性證據(jù)時,需要考慮的因素,還提供了確證性證據(jù)的數(shù)據(jù)類型的示例。我國目前還沒有類似的指導(dǎo)原則,詳細介紹該指導(dǎo)原則,期望對我國這方面的研究和監(jiān)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Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence Draft Guidance for Industry" in September 2023, supplementing the FDA's 2019 Effectiveness draft guidance and 1998 Effectiveness guidance.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.

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