FDA Tells Industry: Use the Correct Study Data Formats or We Won't Approve Your Drug

Posted 06 February 2014 | By Alexander Gaffney, RAC 

New draft guidance published by the US Food and Drug Administration (FDA) is intended to outline the standardized study data format that will be required for all electronic drug application submissions once those standards go into effect in several years.


The Food and Drug Administration Safety and Innovation Act (FDASIA) requires nearly every new, generic, biologic and biosimilar drug submitted to FDA to eventually use an electronic format specified by FDA. Investigational New Drug (IND) applications, used to support clinical trials, will also be required to use the format once it goes into effect, likely in 2016.

The intent is to make it easier and cheaper for FDA (and industry) to review, analyze and archive submissions.

On 5 February 2014, FDA published a roadmap for the rollout of its electronic submission platform, but as Focus noted, it offered few details other than the fact that FDA would be providing guidance to industry through multiple, rather than one, guidance documents.

New Draft Guidance

The first of those specific guidance documents, Providing Regulatory Submissions in Electronic Format-Standardized Study Data, has now been published. The relatively brief 11-page document explains one of the most essential components of any drug submission: the data meant to form the major basis of the approval decision.

The so-called "eStudy Data guidance" goes on to note that the data submitted to FDA "must be in a format that the Agency can process, review and archive," such as those listed in its Data Standards Catalog. If a submitter fails to use an appropriate standard, FDA may refuse to file its electronic submission.

Accordingly, "When planning a study (including the design of case report forms, data management systems, and statistical analysis plans), the sponsor or applicant must determine which FDA-supported standards, formats and terminologies to use," the agency wrote. Alternatively, the company may request a waiver.

FDA makes specific note of four types of standards it says industry should pay especially close attention to:

  1. File Format Standards
  2. Study Data Exchange Standards
  3. Analysis Standards
  4. Terminology Standard

All are critically important components to a submission, and FDA lists several preferential standards for each, such as several supported by the Clinical Data Interchange Standards Consortium (CDISC).

Waivers and Meetings

"FDA will not provide waivers to submit data that do not conform to any FDA-supported study data standard," the guidance notes. However, the agency may grant waivers seeking to use alternate versions of the same standards, such as one previously supported by FDA.

The guidance also goes on to describe support sponsors can receive from FDA during the submission process with respect to study data standardization. Sponsors may, for example, use established FDA-sponsor meetings like pre-IND and end-of-phase 2 meetings to discuss data standardization.

In general, FDA said that "the premarketing application meeting is considered too late to initiate data standardization discussions."

Comments on the draft guidance are due by 7 April 2014.


Providing Regulatory Submissions in Electronic Format-Standardized Study Data (Draft)

Federal Register

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