The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the “Draft Guidance for Industry on Providing Submissions in Electronic Format – Summary Level Clinical Site Data for Center for Drug Evaluation and Research’s Inspection Planning.” BIO supports the release of this draft Guidance as FDA and Industry work together to implement the Prescription Drug User Fee Agreement (PDUFA) V Agreement (the Agreement).
BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products, thereby expanding the boundaries of science to benefit humanity by providing better healthcare, enhanced agriculture, and a cleaner and safer environment.
BIO supports the Agency’s efforts to implement a risk-based approach to inspections, to plan inspections efficiently and effectively, and to meet PDUFA goal dates, which will be facilitated by the early submission of electronic summary level clinical site data to the Office of Scientific Investigations (OSI). The consistency, predictability, and clarity of data requests from OSI is essential as Sponsors prepare to provide complete clinical site information predicated under the PDUFA V New Molecular Entity (NME) Review Program.
In order to improve the efficiency of the drug review process, the PDUFA V Agreement sets a timeline for mandatory electronic submission and lays out a process for the development and adoption of the standards and format of the electronic submissions. BIO believes that PDUFA V governs OSI electronic data requests for selection of clinical sites for pre-approval inspections. Accordingly, the Notice and Guidance should reference the PDUFA V implementation timeframe and specify the required standards, formats, and specifications. Alternatively, this Guidance should be issued as an addendum to the recently released Draft Guidance for Industry Providing Regulatory Submissions in Electronic Format – Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications, issued in accordance with the Food and Drug Administration Safety and Innovation Act (FDASIA) (Pub. L. 112-144), which describes how FDA plans to implement the requirements for the electronic submission of applications for certain human pharmaceutical products.
BIO also believes that the requirement for specificity necessitates more than reference to the publication of a data standard on the FDA Data Standards Resources webpage. Reference to the FDA website for technical specifications documents and other resources that are subject to change without notice, not only undermines the intent and spirit of the PDUFA V Agreement, but also hinders the successful implementation of data standardization efforts by removing the security that comes with clear and transparent requirements and requests. Without such security, both industry and FDA are inhibited from undertaking long-term planning decisions and making the necessary technology investments that truly improve the efficiency of the review process.
It is BIO’s understanding that OSI has developed a three part standard information request, which is currently distributed to Sponsors prior to, or during Pre-NDA/Pre-BLA meetings, and that this Guidance only addresses the third part of such request. BIO requests OSI make the standard information request publicly available, and address the voluntary nature of the request and its relationship to the PDUFA V Agreement. We also request FDA issue guidance on Parts I and II of the standard information request under the PDUFA V framework. Without the public release of the standard information request and associated formal guidance it is difficult for Sponsors to satisfy the requirements for submission of complete and timely clinical site information, electronically or otherwise, without incurring significant resource burdens.
BIO appreciates this opportunity to comment on the “Draft Guidance for Industry on Providing Submissions in Electronic Format – Summary Level Clinical Site Data for Center for Drug Evaluation and Research’s Inspection Planning.” Specific, detailed comments to the Guidance and the technical specifications, which are incorporated into the Guidance by reference, but not otherwise subject to a public review process, are included in the following chart. We would be pleased to provide further input or clarification of our comments, as needed.