Niemann-Pick Type C externally led Patient-Focused Drug Development meeting
The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to provide the Food and Drug Administration (FDA), drug developers, and NPC stakeholders with perspectives from our community on a number of important issues.
The meeting will follow the successful model developed by the FDA for similar Agency-convened meetings and provide persons with NPC the unique opportunity to share their story and experiences—including the challenges they face on a daily basis in living with NPC and their hopes for future treatments–with senior FDA officials and industry to better inform how therapies are developed and how the agency evaluates and considers potential therapies for the disease.
You can learn more about the NPC PFDD meeting by clicking on the Learn More button below:
We used a format that closely follows the model FDA developed for conducting the agency’s PFDD meetings. This included a presentation by an NPC clinical expert, three patient/caregiver panels to capture the array of perspectives across the NPC community, and real-time polling questions about experiences with the disease. Attendees also had the opportunity to participate in facilitated discussion sessions throughout the day. The event was live-streamed to enable remote participation by the largest possible number of people with NPC and their caregivers.
To help us make the PFDD meeting as high-impact as possible, the Ara Parseghian Medical Research Fund requested that the NPC community take part in a Pre-meeting Survey.
We thank all of those who particpiated in the survey.
The information collected via this survey will be used to help us plan the meeting and will also be an important piece of the Voice of the Patient report that will be written after the meeting and shared with the FDA.
We would like to thank our collaborating organizations for this important effort, which include the Dana’s Angels Research Trust, Firefly Fund, Hide & Seek Foundation, Hope for Marian, Johnathan’s Dream, National Niemann-Pick Disease Foundation, and Niemann-Pick Canada,
We would also like to thank the organizations that are assisting in underwriting the cost of the PFDD meeting, which include Mallinckrodt Pharmaceuticals , Orphazyme, StrideBio, Inc. , and the Together Strong NPC Foundation. These organizations will not receive any special access or benefits in regard to the meeting or related materials, and the PFDD meeting and access to the Voice of the Patient report are open to all other interested industry, foundation, and government stakeholders.