What is the Niemann-Pick Type C (NPC) externally-led Patient-Focused Drug Development (PFDD) Meeting?

The NPC PFDD meeting is a groundbreaking initiative featuring a meeting driven by persons with Niemann-Pick Type C disease and their advocates 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.

What is patient-focused drug development and what is a PFDD meeting?

Patient-focused drug development (PFDD) seeks to better emphasize the voice of the patient in the therapy development process so that these therapies are better tailored to the needs and preferences of patients.  Although the concept has been around for many years, the FDA ramped up its PFDD work in 2015 when it began convening PFDD meetings on specific diseases and conditions.  These meetings featured patients and caregivers speaking on panels and in facilitated discussions about the impact of the disease on their daily lives and their experiences with current treatments.  After each meeting, the FDA authors a report that summarizes the key points discussed in the meeting.  Since 2015, the FDA has held over 20 PFDD meetings.  These meetings proved so successful that the agency now invite patient organizations  to host their own PFDD meeting, as the NPC community is now doing. To learn more about these meetings and see examples of other organizations’ PFDD meeting reports, please visit this page.

What are the goals and objectives of the NPC PFDD meeting?

1) Build greater awareness for the disease, its symptoms and daily impact, and the enormous unmet medical need;

2) Provide insight 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; and

5) Inform the benefit-risk framework for NPC for FDA regulatory reviews.

What can I expect at the meeting?

The agenda of the meeting is currently being developed and will be posted on the website a few weeks before the meeting.  As with past PFDD meetings, the NPC PFDD meeting will focus on the symptoms of the disease, its impact on daily life, and experiences with current approaches to treating the disease.  This meeting is all about NPC patients and caregivers, who will have an opportunity to share their thoughts through a mix of panels, facilitated discussion, and polling questions.  There will also be presentations by an expert NPC clinician and representatives from the FDA.  Although other NPC stakeholders are invited to attend the meeting, they will attend primarily as listeners and will be given the opportunity to speak only during a brief public comment period.  The meeting sessions and discussions will be facilitated by the consultants listed below.

The meeting will take place on Monday, March 18 from roughly 9am-5pm in Hyattsville, Maryland. There is no cost to attend the meeting and lunch will be provided free of charge.  We hope that many NPC community members will be able to attend the meeting in person, but for those who are unable to attend in person, a video livestream will be available.  Those joining the live stream will be able to participate in the live polling questions along with those in the room. 

What happens after the meeting?

Following the externally-led PFDD meeting on NPC, we will use the findings from the meeting to produce a Voice of the Patient report, which will summarize the key points discussed during the meeting as well as data from the pre-meeting survey. The APMRF will share this report, along with the completed meeting webcast and transcript, with the NPC community and the FDA.  These materials will be made available on NPC PFDD website and through our distribution lists, including social media platforms.

The Collaboration:

The APMRF would like to thank our collaborating organizations for their contribution to this important effort, which include the Dana’s Angels Research TrustFirefly FundHide & Seek FoundationHope for MarianJohnathan’s DreamNational Niemann-Pick Disease Foundation, and Niemann-Pick Canada.

APMRF has been and will continue to collaborate with medical research centers, academics, and industry in the development and hosting of this meeting.   All decisions regarding survey content, meeting participants, meeting agenda, and the Voice of the Patient report will be made by APMRF in consultation with the other participating NPC organizations listed on the home page as well the hired consultants from Faegre Baker Daniels Consulting and YourEncore.

We thank these organizations that are assisting in underwriting the cost of the PFDD meeting: Mallinckrodt Pharmaceuticals , OrphazymeStrideBio, 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.