Today, FDA issued a revised draft Guidance document entitled, Content of and Process for Submitting Initial Pediatric Study Plans and Amended Pediatric Study Plans.  Quite a mouthful, but exceedingly important if you plan on submitting an NDA that is subject to PREA (Pediatric Research Equity Act).  Firms (of course) want to gain an additional 6-month extension on any existing exclusivity and have another 6-month period of protection, if there is a valid in force patent at the time of award of any period of 6-month pediatric exclusivity.

The Initial Pediatric Study Plan (iPSP) Guidance document addresses:

  • Who must submit an iPSP
  • When an iPSP must be submitted
  • What should be included in an iPSP
  • What should be included in a requested amendment to an agreed iPSP
  • A template that should be used for an iPSP submission

The document runs down the history of the PREA and the legislative and regulatory initiatives (some failed) that led to today’s requirements under PREA.  The Agency outlines the changes and updates that have been made since the first draft Guidance issued in July 2013 by noting:

This revision includes additional clarifications regarding sections previously included in the 2013 guidance as well as new sections that have been added, including: section V.A., Materially Incomplete iPSPs, section VI., Relationship of Agreed iPSP to the Requirement to Submit a Pediatric Plan With an Application, section VII., Contents and Timing of Requested Amendment to an Initial PSP, section VIII., Non-Agreed Initial Pediatric Study Plans, and section IX., Reaching Agreement on the Non-Agreed Initial Pediatric Study Plan. Additionally, Appendix 1, Initial Pediatric Study Plan Template, has been updated.

The Guidance further outlines timing of submission of the iPSP and the importance of addressing the pediatric plan in a rational and sincere manner.  FDA also cautions that an applicant “should not submit a marketing application or supplement until agreement has been reached on the iPSP.”  This, of course, could include a deferral request or waiver of the requirements.  But, none the less. FDA maintains that the iPSP must be addressed ahead of any NDA (original or supplements) filing if the filing falls under PREA.  Issues associated with the content and format, as well as various timing for submission and FDA review with feedback to the sponsor are also addressed, but in more detail than we chose to cover in this post.  Please see and read the Guidance carefully if your product may fall under the provisions of PREA, so there are no surprises during the submission and review process.  The full content of the draft Guidance can be found here.