Rare Disease Advisory Council presses legislators on waivers, emergency protocols and 504 protections

The Rare Disease Advisory Council reviewed legislative priorities during its meeting, focusing on patient-specific emergency protocols (HB6920), funding authority for the council (HB6978), changes to the state definition of intellectual disability (H7108) and other bills that members say affect access to waivers, newborn screening and 504 plan protections.

Council co-chair Leslie Bennett, the patient advocate for rare diseases and meeting chair, summarized the council’s agenda and emphasized outreach to legislators. "We addressed mostly our legislative priorities, which are funding, emergency protocols, waivers, sickle cell, ensuring access to orphan drugs and rare disease treatments," Bennett said. Bennett also noted that the council could not vote on minutes because it did not have a quorum.

The meeting devoted substantial time to H7108, the bill that would change the state’s definition of intellectual disability and remove reliance on an IQ cutoff. Mary Caruso, a caregiver and chair of the advocacy subcommittee, said families fear that a broader definition would overwhelm existing waiver programs. "The problem is it would just inundate us. There'd be too many people. We cannot afford it," Caruso said, noting long waiting lists for current waivers such as Katie Beckett. Participants discussed a conditional or compassionate waiver for children with rapidly progressive rare diseases (for example, juvenile Tay‑Sachs) so those children can receive services without opening eligibility to a much larger population.

On the Katie Beckett waiver (bill number 7103), council members described proposals to expand or retool the program to open slots by shifting some recipients into other Department of Social Services waivers. Caruso said the current wait for Katie Beckett is about five years and that modest expansion would be difficult in the current fiscal climate.

The council reviewed HB6920, patient-specific emergency protocols, which Caruso and others supported as a response to treatment errors in emergency departments where third‑party contractors or outside emergency providers lack access to hospital records. "What this bill is — we're hoping that this bill will force them to have discussions with us," Bennett said.

Members also discussed HB6978, which would authorize the council to apply for grants and accept donations to fund its projects rather than creating a permanent state appropriation. Caruso and others said that private donations and grants are already available and that statutory authority to apply for them would help the council support awareness and advocacy work.

Sickle cell and gene‑therapy coverage was raised as another priority. Bennett said the Department of Social Services submitted a federal application related to sickle cell coverage on March 6 and that participation in the Centers for Medicare & Medicaid Services program could ease coverage for future genetic therapies.

Council members watched federal and state prescription‑drug proposals closely. The group discussed SB11 and related proposals (H7192) that would create a prescription drug affordability board, and they endorsed efforts to protect students’ 504 plan accommodations after members said 17–18 states are pursuing litigation that could affect those protections nationally. Emily Germaine Lee, the council’s physician representative, noted that many families in her clinic rely on 504 plans while in school.

Near the end of the meeting, a Department of Public Health representative reported that a newborn screening bill to add Duchenne muscular dystrophy (6919) has reached the House fiscal committee and that the agency estimated a startup cost near $600,000 for testing kits. The DPH representative said funding and RUSP (Recommended Uniform Screening Panel) actions would drive timing. "If it's on the RUSP, then it's on the RUSP. We do as we're directed," the DPH representative said.

Because the council lacked a quorum, members did not vote on minutes or on formal positions; instead they agreed to continue advocacy by contacting legislators, seeking meetings with the Human Services Committee to discuss intellectual‑disability waivers, circulating bill numbers to members and preparing testimony and cosponsor requests.

The council said it will follow up with legislators and with state agencies as the legislative session continues and will revisit formal votes when a quorum is present.