On behalf of the Niemann-Pick Type C Therapy Accelerator (NTA) team, we would like to share with you a recent patient and caregiver survey in order to better understand the experiences of people with Niemann-Pick Type C (NPC) and their preferences for future treatments. This survey included questions about NPC symptoms, treatments, the impact of the disease on daily life, and respondents’ preferences for benefits and risks of future treatments. This report presents the initial findings from the survey with the aim of helping the U.S. Food and Drug Administration staff to better understand the experiences and priorities of people living with NPC and their caregivers. We are also sharing the report with pharmaceutical companies, organizations and NPC families as we work together to find treatments and ultimately a cure for NPC disease.
This survey builds on two previous surveys conducted in connection with the 2019 NPC patient-focused drug development (PFDD) meeting. The NTA members, who organized the PFDD meeting along with other advocacy groups and community members, have since created this new initiative to promote collaboration and data sharing among members of the NPC research community including pharmaceutical companies, clinicians, and academic researchers. Through this survey and other research efforts, the NTA aims to spur innovative research on treatments and, eventually, cures for this devastating disease.
*Please note we are creating an additional report that includes data from international NPC patients to be used for an international audience. We hope to have this complete in the next 4-6 weeks.
Below is a link to the published survey:
Thank you
Sean Kassen
Joslyn Crowe
Phil Marella
Cristin Davidson
Chris Andrews