Niemann-Pick Type C externally-led Patient-Focused Drug Development (PFDD) meeting and pre-meeting survey

Dear Friends in the NPC Community,

The Ara Parseghian Medical Research Fund is excited to announce that, in collaboration with the organizations listed below, on March 18th, 2019 we will host the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) meeting with the FDA.

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

You can learn more at the website:

How can I get involved? You can help make this initiative a success by filling out the pre-meeting survey and attending the meeting, either in person or via the live webcast.  Please find more details on these opportunities below and on our website.

When is the meeting? 

  • Date: March 18th, 2019
  • Location: College Park Marriot Hotel & Conference Center in Hyattsville, MD (Washington DC metro area).
  • Schedule: The schedule is yet to be set, but will tentatively be held from 9am-5pm


Meeting Format: 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.

Who can attend: Any person associated with NPC disease including patients, parents & relatives, caregivers, organizations and drug development companies.  Please note that because this meeting is intended to collect information for the FDA, the focus will be on families from the USA. However, anyone can attend and participate in the survey.

Live Stream:  For those who are unable to attend in person, the entire meeting will be streamed online and recorded.

Registration: Registration is not yet available, but we hope to have the site running in the next few weeks.  You will receive an email when registration is available.  More details to come.

Pre-meeting Survey

Prior to the meeting, we are conducting a survey to collect valuable information in regard to burden of the disease and experience with current therapies. We ask that each patient, parent, or caregiver take the time to fill out the pre-meeting survey.  The survey will be available until January 10, 2019.  If you have multiple children with NPC disease, please fill it out separately for each child.  You can find the survey at our website.

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.