通过试验摘要域加快美国食品药品管理局(FDA)申报

试验摘要(TS)域是 电子申报所需标准化研究数据的重要组成部分。2016年7月,美国食品药品管理局(FDA)发布了3.1版《研究数据技术一致性指南》,其中建议加入一个TS域,以便在申报中确定研究开始日期。在临床研究中,研究开始日期以注册该项研究的任何受试者接收知情同意书的最早日期为准,然而,非临床研究的开始日期则以该项研究的起始(最终确定方案)日期为准。本文将回顾研究开始日期的重要性并提供建议,以帮助确保当前和遗留的研究得以成功申报。

Checking conformance to the standards

To determine if a complete Standard for the Exchange of Nonclinical Data (SEND) dataset is a requirement for inclusion in an eCTD (electronic Common Technical Document) submission, the study start date is the most crucial piece of information. As part of the CDER SBIA webinar series in October 2016, the FDA further clarified this stipulation. In short, this approach was introduced to "implement a process for rejection of submissions that do not conform to the required study data standards."1

More specifically referred to as "technical rejection criteria," the purpose of this initiative is to avoid Refuse to File (RTF)/ Refuse to Review (RTR) submissions, and instead, issue a rejection of a less severe nature. The technical rejection allows the sponsor to correct parts of the submission that failed a conformance validation rather than going back to square one and starting over.

Reviewing your legacy studies

For legacy studies started before December 17th, 2016 (NDA/BLA) or December 17, 2017 (IND), sponsors can avoid a technical conformance failure notice by doing one simple thing:

▶ Include a TS file containing one row of information - the study start date - in .xpt format. Then, with the concise nature of this file, there will be no question as to the status of the study and inclusion/exclusion of SEND formatted data.

Handling blended studies

It is reasonable to think that for some time, sponsor's submissions will include a blend of studies that are in scope and legacy studies that are out of scope from a regulatory requirement standpoint. When the agency receives the submission, the TS files will be used to sort the studies within their system. Legacy studies will go to a "queue" with no further checks required. In-scope studies will go to a different "queue." Here, further evaluation of the define.xml and DM.xpt files will commence.

Example of the TS.xpt for legacy studies

Reference:

  1. CDER Small Business and Industry Assistance (CDER SBIA) Webinar – Study Data Standards in eCTD: What You Need to Know about the New Technical Rejection Criteria. Presented October 12, 2016.