Dear Dr. Woodcock and Dr. Donohue:
As leaders across the Neimann-Pick Type C (NPC) Community, we are writing to inform you of a significant collaboration among our organizations aligning efforts to help bring treatments to patients and families. The NPC Therapy Acceleration Initiative will ensure a strategic and coordinated approach to our shared objectives. The externally-led Patient-Focused Drug Development Meeting for NPC in March of 2019 demonstrated the power of acting together and, by this letter, we would like to invite continued partnership with the Food & Drug Administration in this critical work.
The NPC Therapy Acceleration Initiative will engage the entire NPC community and, building on our Voice of the Patient report, will be guided by the patient perspective. We are in the process of mapping the array of ongoing programs across our organizations including research investments, biomarker development with the Critical Path Institute, newborn screening, and access to quality clinical care, among others, to improve efficient use of resources and to identify any gaps in support for rapid translation of discovery into treatments. Another early stage element of the Initiative is to create a comprehensive and transparent catalogue of all ongoing NPC programs among preclinical research groups and industry for community involvement.
The Initiative’s launch phase goals include ensuring the NPC patient perspective is an integrated part of the FDA’s review of potential NPC treatments. With the FDA’s guidance, we created a robust data set around the experience of NPC across the life stages and with particular depth on the effects which matter to people with NPC. We believe this information provides agency officials, industry, and the community-at-large a keen understanding of how potential treatments can impact this experience. Our post-meeting survey, designed to address questions raised in the PFDD meeting around the willingness of NPC patients to accept certain risks for potential benefits, created another valuable resource for regulatory application.
In light of the recent reorganization of the Center for Drug Evaluation and Research, including the division leading NPC therapy reviews, we respectfully request the opportunity to convene a follow-on discussion with agency leaders to ensure the full value of our PFDD work is realized. Many NPC patients are involved with experimental therapies and, as you can appreciate, the patients and their families feel strongly their perspectives should be fully communicated in the review process. We share this view and are prepared to organize a thoughtful exchange with agency representatives.
We certainly recognize you have many demands on your time, particularly in terms of responding to the COVID-19 pandemic. Thank you for considering these proposals to continue our working relationship. We look forward to learning your availability to discuss both the Initiative and the ongoing agency engagement with the NPC community. We are also prepared to assist in arranging any discussions with patients and families about a specific therapy under review. Please direct any questions to Sean Kassen at skassen@nd.edu or (574) 302-7568 who will assist in coordinating discussions with the Initiative.
Kindest Regards,
Sean Kassen, PhD, Director, Ara Parseghian Medical Research Fund
Joslyn Crowe, Director, National Niemann-Pick Disease Foundation
Chris Andrews, NPC Parent, Firefly Fund
Phil Marella, NPC Parent, Dana’s Angels Research Trust
Cristin Davidson, PhD, NPC Scientist