The Food and Drug Administration Safety and Innovation Act (FDASIA) amended the Federal Food, Drug, and Cosmetic Act (FD&C Act) to require that certain submissions under the FD&C Act and the Public Health Service Act be submitted in electronic format specified by FDA.  On May 15, 2015, FDA published the final “Guidance on Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications.”  Unlike other guidances which are not legally binding, Congress granted explicit authorization to FDA to implement the statutory electronic submission requirements in guidance.  FDASIA allows 24 months after the final guidance is issued for sponsors to conform.

NDA, ANDA, BLA and master files must be submitted in eCTD format beginning May 15, 2017.  IND submissions must be submitted in eCTD format beginning May 15, 2018.  Additionally, electronic submissions must use the version of eCTD currently supported by FDA and specified in the Data Standards Catalog.

This requirement also includes all subsequent submissions, including amendments, supplements, and reports.  Study data must also be submitted electronically using the format described in FDA’s guidance for both clinical and nonclinical studies.  FDA uses the standards specified in the Data Standards Catalog (Catalog).  The Catalog provides: Currently supported and/or required standards and their uses, the start date of a particular standard, the end date of a particular standard, the date the requirement to use a particular standard will begin, the date such requirement ends (or will end) and other pertinent information.

Study data standards: standards way of exchanging study data between computer systems

Examples:

The Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model

(SDTM) and Standard Exchange for Nonclinical Data (SEND) standards for tabulations data.

Analysis standards: standard data structure intended to support analysis

Analysis standards include extraction, transformation, and derivations of the original data. The CDISC Analysis Data Model (ADaM) is an example of a study data standard for analysis data.

ARE THERE ANY WAIVERS FROM THE REQUIREMENT?

monopoly

ARE THERE ANY EXEMPTIONS FROM THE REQUIREMENT?

The only exemptions to the guidance are noncommercial INDs (products that are not intended to be distributed commercially and includes investigator-sponsored INDs and expanded access INDs (e.g., emergency use INDs and treatment INDs).

exempt

AND WHAT IF A SPONSOR DOES NOT COMPLY?

“Submissions that are not submitted electronically and electronic submissions that are not in a format that FDA can process, review, and archive will not be filed or received.”

rejected

Our Recommendations

  • Discussions about nonclinical study data standardization plans may be initiated at the pre-IND stage and should continue throughout development.
  • Initial discussions about which data standards to use for study data should take place as early as possible during drug development, especially for safety data, but should in any event occur no later than the end of Phase 2.

QUESTIONS?

Have questions about how this new requirement will affect your application or planned submissions?  Click here to ask the regulatory experts at Facet Life Sciences.