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 followed 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.
To help us make the PFDD meeting as high-impact as possible, the APMRF and their collaborators also requested the NPC community take part in a Pre-meeting Survey.
We thank all of those who participated in the survey.
You can learn more about the NPC PFDD meeting and the survey 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.
Voice of the Patient Report
The Voice of the Patient report summarizes the key points discussed at the meeting, as well as information collected from the NPC community via pre- and post-meeting surveys. Those key points include the challenges presented by the disease and shares patients and caregivers views on current and future treatment options, which they hope will slow or prevent the pervasive symptoms of the disease. Additionally, the report emphasizes their desire to collaborate with the FDA and drug companies to advance research and therapy development. If you are interested in a hard copy of the report please email Sean Kassen at firstname.lastname@example.org.
Survey on Preferences for Treatments for Niemann-Pick Type C
In order to better understand the experiences of people with Niemann-Pick Type C (NPC) and their preferences for future treatments, the NPC Therapy Accelerator (NTA) conducted an online patient and caregiver survey. This survey included questions about NPC symptoms, treatments, the impact of the disease on daily life, and respondents’ preferences for benefits and risks of future treatments. This report presents the initial findings from the survey with the aim of helping U.S. Food and Drug Administration staff to better understand the experiences and priorities of people living with NPC and their caregivers.
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.