Rare Disease advisory council mobilizes for waiver expansions, orphan‑drug protections and emergency protocols

The Rare Disease Advisory Council (RDAC) on Jan. 15 agreed to make waiver expansions, protections for orphan drugs and emergency‑protocol guidance its central goals for the 2025 legislative session and formed advocacy and awareness subcommittees to support that work.

The council’s advocacy discussion centered on expanding the Katie Beckett waiver, pursuing a small compassionate waiver for children with serious rare conditions, and pressing for safeguards when the state reviews drug affordability. Leslie Bennett, the RDAC patient advocate, said the group has asked state legislators to introduce bills: Senator Cohen sponsored a Katie Beckett expansion and a compassionate‑waiver concept; Senator Anwar filed placeholder bills to fund the RDAC and to seek procedural protections for rare‑disease drugs during pricing reviews.

Why it matters: council…