Niemann-Pick Type C Externally Led Patient-Focused Drug Development Meeting Announcement

Dear Friends in the NPC Community,

The Ara Parseghian Medical Research Fund is excited to announce that, in collaboration with the organizations listed below, we will host the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting. In late August, we submitted a Letter of Intent to the Food and Drug Administration (FDA) for approval to conduct this landmark initative and this past week we received that approval.

What is the externally led Patient-Focused Drug Development (PFDD) meeting? The PFDD meeting is a groundbreaking initiative culminating in a structured meeting driven by persons with NPC disease and their advocates to provide the 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 inform how therapies are developed and how the agency evaluates and considers potential therapies for the disease.

When is the meeting? We are working on finalizing the date with the FDA, but it will most likely be in early Spring in the Washington DC area. We feel strongly that due to the ongoing clinical trials of many potential life-saving treatments for NPC disease, it is critical that we move forward with this meeting in the near future. We will alert the community as soon as the date is agreed.

What will the meeting include? The meeting will include a clinical expert presentation, 2-3 patient/caregiver panels including a representative array of perspectives across the NPC community, and real-time polling capability. While we will encourage participation in person, the event also will be live-streamed to enable remote participation by the largest possible number of people with NPC and their caregivers.

Premeeting Survey: Prior to the meeting we will be conducting a survey to collect valuable information in regards to burden of the disease and experience with current therapies. We are hoping to have this available by early December and will encourage all families effected by NPC disease take the time to fill out the survey.

Key Outcomes: The NPC PFDD meeting and related survey will:
1) Build greater awareness for the disease, its symptoms and daily impact, and the enormous unmet medical need;
2) Provide insights on drug development and disease assessment tools, such as the NPC-CSS, in capturing what is important from the perspective of the patient;
3) Provide direction and focus for future research efforts through the lens of patient experience;
4) Formally capture patient input in a Voice of the Patient report for the FDA and the NPC community at large;
5) Inform the benefit-risk framework for NPC for FDA regulatory reviews.

Additional Details: We will be establishing a NPC PFDD meeting website that will include all of this information as we finalize a date and time and registration instructions. It also will become the landing spot for all of the information gathered and specifically the pre-meeting survey and Voice of the Patient report that will be made accessible to all companies, the FDA, clinicians, researchers, and families effected by NPC disease.

Collaborating organizations include: Ara Parseghian Medical Research Fund, Dana’s Angels Research Trust, Firefly Fund, Hide and Seek Foundation, Hope for Marian, Jonathon’s Dream, and Niemann Pick Canada.