Readying a Rare Disease Community for the Availability of Treatments

Niemann-Pick disease is a rare and often-fatal genetic, metabolic disorder with no approved treatments, but that could soon change as new therapies advance through the development and approval process. In anticipation of the first therapies for the disorder becoming available, the National Niemann-Pick Disease Foundation has been working to prepare patients and their families for new obstacles they may need to navigate to obtain access and reimbursement for these treatments. We spoke to Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation, about what her organization is doing to prepare the community for the availability of the first therapies for the conditions, the issues people will need to consider, and how families may need to think differently about their futures.