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 members said waiver access and orphan‑drug availability directly affect medically fragile patients’ ability to get home‑based services and lifesaving treatments. The RDAC plans targeted outreach to the Connecticut General Assembly (CGA) public‑health and human‑services committees and is preparing materials for legislative staff.

Discussion and next steps: council members described long, opaque waiver wait lists — parents described waits “up to … like, four years” — and endorsed a package of actions: write to legislators, testify before committees (by Zoom or in person), and recruit public and professional allies for direct outreach. The advocacy subcommittee will produce templates and talking points; Mary Caruso and Colleen Brunetti will co‑lead portions of the outreach. Members were advised to identify their personal legislators and to contact members of the public‑health and human‑services committees when bills are filed.

On orphan‑drug reviews, members urged a statutory or procedural safeguard so state affordability reviews identify FDA orphan‑designated therapies before cost‑containment decisions. Members said a prior state review had treated Epidiolex and Trikafta as typical price hikes without recognizing those drugs’ orphan‑drug status and pediatric impact; they cited Colorado’s RDAC process and a CMS exclusion policy as possible models.

The council also discussed a proposed compassionate waiver for 25–50 slots for critically ill children; members said federal coordination (CMS) would influence feasibility. RDAC members stressed the need for clear emergency‑protocol language so standard emergency‑department pathways do not unintentionally harm patients with rare conditions.

Votes and organizational business: members approved the minutes from the Dec. 10 meeting on a voice vote after Colleen Brunetti moved to approve and Leslie Bennett seconded; no objections were raised and the chair declared the minutes approved. The RDAC created two standing subcommittees — advocacy and awareness — and began recruiting public members and organizational partners for both.

Members also flagged other bills to follow, including proposals to add Duchenne muscular dystrophy to newborn screening panels and measures on utilization review and adverse outcomes that may intersect with rare‑disease access.

What’s next: the RDAC said it will circulate bill numbers and committee assignments as bills are filed, distribute outreach templates and talking points, and staff the advocacy subcommittee to coordinate testimony and legislator meetings.