Last week we met with FDA to discuss status and potential release of guidance. As you know, we submitted draft guidance to FDA in June, 2014. FDA posted the guidance for public comment for a period of 30 days, from September 6 through October 6, 2014.
Our discussion last week focused on comparing the draft documents or what we might see in the FDA document. Dr. Dunn said FDA guidance was informed by both the policy forum, as well as, the guidance submitted in June. He said that the PPMD-led Draft Guidance stands — and still exists as a living document. FDA intends to refer to it and reference it in the future (particularly calling out our emphasis on Caregivers which may be less present in the FDA version). The focus of this document is about what to know in order to develop new therapies for children.
We are on the eve of a groundbreaking moment for our community, a moment that we made happen, together. And while we anticipate that a level of specificity may differ between the FDA’s version of the guidance and the guidance we submitted, we look forward to FDA releasing their guidance.
We have much more work to do together, as a community, but are so proud that this community effort continues to resonate with regulatory agencies. More to come…