The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the “Draft Revision of Guidance for Industry on Providing Regulatory Submissions in Electronic Format – Certain Human Pharmaceutical Product Applications and Related Submissions Using the Electronic Common Technical Document Specifications.” BIO supports the release of this draft Guidance as FDA and Industry work together to implement the Prescription Drug User Fee Agreement (PDUFA) V Agreement (the Agreement).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.
The Food and Drug Administration Safety and Innovation Act (FDASIA) requires that certain submissions, including NDAs and BLAs, be submitted in electronic format, beginning no earlier than two years after publication of the final version of a draft guidance. The final guidance is to be issued after public notice and opportunity to comment and is to specify the format of the electronic submissions.
The PDUFA V Agreement also references the statutory electronic submission trigger of final guidance that specifies format. And in recognition of the significant investments associated with the electronic submission mandate, the Agreement further provides that the Agency will “periodically publish final guidance specifying the completed data standards, formats, and terminologies that sponsors must use to submit data in applications.”
BIO applauds FDA on the timely release of this Guidance and recognizes FDA’s efforts to provide Industry with the necessary requirements for a valid electronic submission as per the intent of the PDUFA V Agreement. Specifically, we appreciate the clarity and certainty the Guidance provides by directly citing eCTD version 3.2.2 under the eCTD Specifications Requirements section (see Guidance lines 128-129). We ask that such clarity and certainty be extended throughout the Guidance by referencing specific software versions and formats for all data formats and specifications as opposed to referencing additional technical resources found on FDA’s eCTD website (see Guidance lines 136-137 and the attached chart).
While we understand and appreciate FDA’s intent to provide the additional technical specifications as separate, stand-alone documents to “reflect the evolving nature of the technology and the experience of those using the technology,” BIO believes that 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. BIO would welcome the opportunity to work with the Agency to find an alternative method of communicating the availability of technical specifications that allows for public comment and advance notice, but does not necessarily rise to the level of a formal guidance.
BIO appreciates this opportunity to comment on the “Draft Revision of Guidance for Industry on Providing Regulatory Submissions in Electronic Format – Certain Human Pharmaceutical Product Applications and Related Submissions Using the Electronic Common Technical Document Specifications.” Specific, detailed comments are included in the following chart. We would be pleased to provide further input or clarification of our comments, as needed.
See FDASIA Section 1136.