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Patient-Focused Drug Development Meeting

Voice of the Patient Report

The Voice of the Patient report from the PFDD has just been released! We were proud to partner with the Ara Parseghian Medical Research Fund, Dana’s Angels Research Trust, Firefly Fund, Hope For Marian, Niemann-Pick Canada and Jonathan’s Dream on the Patient Focused Drug Development meeting that took place with the FDA this spring, an important forum for patients and their caregivers to voice their perspectives, experiences, needs, and priorities on NPC. This report has just been published as a summary of this meeting and has been shared with the FDA.


Source: Niemann-Pick Niemann-Pick Type C Patient and Caregiver Voices: Externally-led, Patient-Focused Drug Development Meeting

Niemann-Pick Type C Externally-led Patient-Focused Drug Development Meeting Recording


What Is PFDD?
Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients and their caregivers will be able to voice their perspectives, experiences, needs, and priorities on specific diseases and their treatments.
What is the externally led Patient-Focused Drug Development (PFDD) meeting?
The PFDD meeting is an initiative lead by persons with Niemann-Pick Type C disease, their caregivers, and advocates gathering in a structured meeting to provide the FDA, drug developers, and NPC stakeholders with perspectives from the NPC 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 experience the unique opportunity to share their story – 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.
How do I get involved?
A PFDD meeting will be held on March 18, 2019 in Hyattsville, Maryland. Online livestreaming will be available for those unable to attend. The NPC community is invited to attend either in person or via live stream.
Additional Information
To learn more about PFDD and the upcoming meeting, please click here.
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, Niemann-Pick Canada, and National Niemann-Pick Disease Foundation.

Additional Travel Funding For Those Participating in PFDD

CTD Holdings is providing funding through NNPDF to support families with travel assistance to the upcoming NPC Externally-Led Patient-Focused Drug Development Meeting (PFDD). Support is intended for additional family members who would like to attend the meeting but are not eligible for subsidy otherwise. As the national patient organization, NNPDF is glad to assist in providing this support to the Niemann-Pick community.

Externally-led PFDD events are driven by people with NPC and their advocates to provide the FDA with personal perspectives and experiences from the community, including challenges faced on a daily basis in living with NPC and hopes for future treatments. It 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.

For more information on how to apply for this financial assistance, please contact Laurie Turner, Family Services Manager at

For more information on the PFDD, or to register for the event, visit

Click here to read CTD Holdings press release.