Patient Voice in Access Studies

This US based qualitative PRO research study was completed in collaboration with the National Niemann-Pick Disease Foundation (NNPDF). The research was conducted to document the patient experience in Niemann-Pick as it relates to health insurance literacy, coverage issues, and the burden experienced by patients and their families pertaining to the access of medications, services, and devices. This publication was presented at Global Genes’ 2022 RARE Patient Advocacy Summit and stresses the importance of understanding these issues as a patient organization aiming to promote advocacy efforts and improve patient outcomes.