The EveryLife Foundation for Rare Diseases is convening its annual Community Congress meeting in Washington, D.C. on November 16th. The event will give participants an opportunity to learn about the pressing health policy issues facing the rare disease community. There is no fee to attend but advance registration is required.
Attendees will hear a keynote from Dr. Phil Reilly, Partner at Third Rock Ventures and author of the new book, “Orphan: The Quest to Save Children with Rare Genetic Disorders.” The conference will also feature a panel of Washington insiders who will discuss how the election could impact the policy landscape for rare diseases.
Key topics for the meeting will be policy proposals to enhance and expand newborn screening as well as advocacy strategies to advance the 21st Century Cures Act before the end of the year. Advocates will have a chance to ask questions and provide input on the Foundation’s goals for 2017 and beyond.
The EveryLife Foundation’s Community Congress is designed to bring patient organizations and industry together to take on policy issues for the rare disease community. The Congress has working groups focusing on Public Policy, Regulatory Science, and Newborn Screening. Each group is co-chaired by an individual from industry and a leader from a patient organization, and is supported by staff at the EveryLife Foundation and individual working group members.
To date, the Public Policy group played a key role in planning a recent workshop on issues relating to early access for experimental medicines. The Regulatory Science working group provided feedback on a Food and Drug Administration (FDA) guidance last year and is holding a webinar on October 25th about the Accelerated Approval pathway. The Newborn Screening working group played an important role in helping to advance state legislation in California, which was recently signed into law by Governor Brown. The law requires California to enhance and expand newborn screening for all babies born in the state.