2017 Rare Disease & Orphan Products Breakthrough Summit

A two-day conference in Washington, D.C., earlier this month offered the opportunity for organizations such as Sofia Sees Hope to discover the latest in the rare disease community, meet the nation’s top rare-disease professionals, and gather resources and ideas to help with the Leber congenital amaurosis community.

More than 670 people attended the first day of the 2017 Rare Disease & Orphan Products Breakthrough Summit on Monday, Oct. 16.

The summit, presented by the National Organization for Rare Diseases (NORD) , included a range of topics, from the challenges of healthcare costs, to the ethical guidelines for patient organizations and themes of collaboration, urgency and transparency.

The summit’s second day featured presentations by the Food & Drug Administration’s leading Division Directors and discussions of improving the lives of patients with rare diseases.

The conference kicked off with a welcome by Peter L. Saltonstall, President and CEO of NORD, and Marshall L. Summar, M.D., Director of Children’s National Rare Disease Institute, Chief of Genetics and Metabolism at Children’s National Medical Center , and Chairman of NORD’s Board of Directors.

Key presentations included ensuring patient access, sustaining orphan drug development and availability, and reviewing achievements in rare disease drug, biologic and device development.

Mike Porath, founder and CEO of The Mighty and a parent of a child with a rare disease, gave a motivating, multi-faceted presentation in his keynote community address. His advice to families living with a rare disease included asking for help, embracing the moments and dreaming big to open doors for people with rare diseases.

Scott Gottlieb, M.D., Commissioner of the FDA, delivered the administration’s keynote address, talking about the group’s rare disease priorities, the rapid changes in rare disease product development and the basis of the Orphan Drug Act.