While there is growing ability to pursue the development of therapies for ultra-rare diseases, they remain challenging because of economics. One major barrier is the difficulty in getting reimbursement for therapies in the absence of well-powered clinical trials that recruit enough participants to satisfy payors demands for adequate proof of the value of a therapy. The Muscular Dystrophy Association, earlier this year, awarded the nonprofit biotechnology Cure Rare Disease a grant to research novel reimbursement strategies for ultra-rare disease therapies. We spoke to Rich Horgan, founder and president of Cure Rare Disease, about the evolution of his organization, its growing pipeline of therapies, and why developing a viable reimbursement model is essential to creating sustainable development of ultra-rare disease therapies.