Accélérer les demandes d'autorisation à la FDA grâce au domaine de résumé des essais

Un domaine de résumé d'essai représente une partie essentielle de la standardisation des données d'étude pour les demandes par voie électronique. En juillet 2016, la FDA américaine a publié la version 3,1 du Study Data Technical Conformance Guide, qui conseille d'inclure un domaine de résumé de l'essai pour identifier la date de début de l'étude dans la demande d'autorisation. Dans les études cliniques, la date de début de l'étude est la première date de consentement éclairé de tout sujet participant à l'étude, tandis que pour les études précliniques, c'est la date de lancement de l'étude (finalisation du protocole). Cet article revient sur l'importance de la date de début de l'étude et émet des recommandations pour favoriser la réussite de la demande d'autorisation pour les études actuelles et futures.

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


  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.