PPMD has been honored to lead the Duchenne community in developing patient-focused tools like our draft guidance and our benefit/risk surveys to help accelerate the FDA review of Duchenne treatments. Just last week, FDA issued its landmark draft guidance on Duchene therapy development, a document heavily informed by the project submitted nearly one year ago and led by PPMD.
PPMD has been encouraging Congress and the FDA to take additional actions to encourage more patient groups to develop such tools to strengthen the voice of the patient in the review process. But we need to know how FDA reviewers are – or are not – using such instruments during the critical product review stage.
To address this issue, Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) have introduced S. 1597, the Patient-Focused Impact Assessment Act. This bipartisan bill calls for FDA to develop a simple and publicly accessible checklist to show how reviewers are – or are not – using such PFDD tools like benefit/risk surveys, guidance documents and patient-reported outcomes when reviewing applications. The bill also calls for additional FDA clarity on actions patients and industry can take to develop PFDD products to drive further innovation in the field.
S. 1597 is cosponsored by Senators Susan Collins (R-ME), Michael Bennet (D-CO), Johnny Isakson (R-GA) and Al Franken (D-MN). We need you to reach out to your Senators today and urge that they sign on as a cosponsor of S. 1597.