The US Food and Drug Administration (FDA) has released a revised draft guidance document which instructs sponsors of pharmaceutical products on how to submit new product and trial applications using the electronic common technical document (eCTD) format.

Background

The document, first released in draft form in January 2013, describes new requirements passed under the Food and Drug Administration Safety and innovation Act (FDASIA) of 2012.

The law, which contains a wide range of regulatory reforms and enhancement programs, requires all pharmaceutical companies to submit their 505(b) (new drug), 505(i) (clinical trial), 505(j) (generic drug), 351(a) (biologic) and 351(k)(biosimilar) submissions to FDA using the eCTD format.

That format has been in use by FDA since 2003, and has been recommended by the agency since 2008. The format eliminates the need for lengthy paper submissions, allowing companies to make more rapid digital submissions to the agency using a format which is common among the US, EU, Japan and other nations.

The original draft guidance document called for a phased-in approach meant to give sponsors time to acclimate to the new eCTD requirements. Under the plan, all new drug applications (NDAs), abbreviated new drug applications (ANDAs), biologic license applications (BLAs), and biosimilar applications (351(k)s) would need to be submitted starting 24 months after the issuance of the final guidance. All investigational new drug applications (INDs), used to obtain approval for clinical trials, would need to use the eCTD format starting 36 months after the issuance of the final guidance document.

The document is also exceptionally unusual in that Congress, when it passed FDASIA, ordered FDA to establish standards for the submission of the eCTD using a guidance document, and not a regulation. Ordinarily, guidance documents are strictly non-binding interpretations of FDA regulations. In other words, FDA’s guidance document establishes not just that companies should submit applications using the eCTD, but that companies must submit those applications, and using a specific standard.

Document Changes

FDA’s revised guidance document is largely the same as its previous guidance, with a few notable changes.

First, FDA now explains that certain regulatory documents now fall under the guidance, including advertising materials, most drug master files and promotional labeling.

In addition, FDA also now explains that the use of its Electronic Submissions Gateway (ESG) will be required, that specific file formats will be required to be submitted, and that the use of FDA software will be necessary to file submissions.

Notably, because FDA has issued a revised draft guidance instead of a final guidance, the eCTD submission requirements will now not go into effect for at least another two years, and likely closer to three.

Comments on the guidance are due to FDA by 23 September 2014.

Guidance for Industry: Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications (FR)