Electronic Submission Capability to FDA for Academic Investigators—The Process, Challenges, and Opportunities Affecting the Translational Research Enterprise

The Prescription Drug User Fee Act (PDUFA) has undergone authorization five times, most recently as part of the FDA Safety and Innovation Act (FDASIA) signed into law on July 9, 2012. PDUFA V covers fiscal years 2013–2017 beginning on October 1, 2012. Section 1136 of FDASIA includes a requirement that submissions to FDA be in electronic format. To implement this requirement, FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologic Evaluation and Research (CBER) issued a Draft Guidance (January 2013) on the standards and format of electronic submissions including electronic Common Technical Document (eCTD) format.1 FDA will issue the Final Guidance no later than 12 months from the close of the 60-day public comment period upon the Draft Guidance. The Final Guidance and subsequent revisions will be binding on sponsors, applicants, and manufacturers no earlier than 24 months (36 months for Investigational New Drug submissions) after issuance of the Final Guidance. In short, the transition to mandatory eCTD compliance for drugs and biologic submissions is swiftly approaching. The Center for Devices and Radiologic Health (CDRH) has already issued its Final Guidance for “eCopy” standards in December 2012.2 For drugs and biologics, PDUFA V electronic submission mandates for eCTD do not apply to non-commercial research conducted by academic sponsor-investigators. However, academics are required to comply with the eCopy guidelines of CDRH. One could then question if eCTD capability for academia is even desirable given that transition from paper can be a resource-intensive process. The fact is that with PDUFAV mandates, the FDA submission practices of eCTD-compliant industry and academia will continue to diverge. On a number of levels, this divergence can complicate the translation of academic discoveries into private sector opportunities and biomedical advances. Clinical research at academic medical centers significantly and increasingly contributes to discovery, development, and repurposing of FDA-approved products. In addition to Institutional Review Board approval, such studies commonly require FDA oversight through Investigational New Drug (IND) or Investigational Device Exemption (IDE) submissions to FDA. An increasing number of academic medical centers offer regulatory support for their academic sponsor-investigators who hold these INDs and IDEs. First described by Dr. Harvey Arbit, these support units offer significant value to investigators and the university; they can reduce regulatory compliance risk while accelerating biomedical advances.3 We propose that like industry, academic regulatory affairs units, technology transfer offices, and the university research enterprise could benefit from the efficiencies and benefits of “eSub” capability. Presently, we describe one high-volume regulatory support program at a large academic health center and its acquisition of eSub capability for drugs (paper IND to eCTD) and devices (paper IDE to eCopy). We describe the steps involved and their challenges, some of which are likely unique to a non-industry setting. We also discuss our use of expert technical consulting and software solutions. Finally, we postulate how eSubmissions will impact our regulatory operations metrics, our Agency interaction, and our industry interaction. It is our hope that sharing this information will assist other academic health centers that are thoughtfully considering an investment in this technology.