Rare Disease Council pushes state bills, prioritizes emergency-protocol legislation amid ER outsourcing concerns

The Rare Disease Advisory Council (RDAC) on April 22 reviewed pending state legislation the council is following and urged members to contact legislators to press for support on several priority bills, including one that would require medical personnel to follow patient-specific emergency protocols for people with rare diseases.

The council cochair, Leslie Bennett, said the legislation would ask “physicians, nurses, physician assistants, and everybody else to follow patient specific emergency protocols for those with rare diseases and special health care needs.” Bennett described the measure as a way to prevent incorrect, protocol-driven treatments when a patient’s condition requires an expert-approved, individualized plan.

Why it matters: Council members said enforcement of patient-specific emergency protocols could reduce preventable hospital stays and medical errors for people with rare conditions. At the same time, several bills the council supports carry large fiscal notes that make passage uncertain this year.

Details and council priorities - HB 6919: would add Duchenne muscular dystrophy to the newborn screening panel. Bennett said federal funding for the test is unlikely and that “the fiscal note for it is in excess of $300,000 per year,” making its prospects uncertain in appropriations. - HB 6920: emergency-protocols bill. The council flagged this as a high priority and encouraged outreach to members of the Connecticut General Assembly (CGA) to request floor action and letters of support. - HB 6978: ARDEC funding; described by the council as a higher-priority item with minimal fiscal impact. - HB 7103: expansion of the Katie Beckett waiver; Bennett said the fiscal note is over $300,000 so it is likely to be reviewed closely in appropriations. - HB 7108: intellectual disabilities and autism provisions; the full bill carries a fiscal note the council estimated “in excess of $500,000.” The council plans outreach to the Human Services Committee to explain rare-disease overlap with intellectual disability and autism. - HB 1473: sickle cell bill; the council described this as likely to pass and as opening pathways for future cell and gene therapies. - S.B. 11: prescription drug bill under debate; the measure would create a prescription drug advisory council with a seat for a physician experienced in rare disease. - HB 7219: would protect Section 504 plans (classroom accommodations); the council urged members to contact legislators because the bill carries a fiscal note under $200,000 and aims to preserve access to school accommodations. - HB 6771 and HB 6895: biomarker-testing bills. The American Cancer Society asked the council to support HB 6771 pending an amendment; the council said it opposes HB 6895 because it would impose a time limit (reported as several years) on insurer coverage for biomarker testing.

Concerns about emergency protocols and ER outsourcing Council members described real-world cases in which standard emergency-room protocols caused harm for patients whose rare conditions required different treatment. Bennett recounted a case in which her daughter was treated for presumed status epilepticus rather than a metabolic crisis and “ended up in the hospital for an extra week” because the initial treatment was inappropriate.

Members raised two recurring objections to the emergency-protocols bill: (1) liability and the risk that nonexperts could author patient-specific letters that others would feel compelled to follow, and (2) uncertainty about who qualifies as an “expert” authorized to write such a protocol. Emily (professor at Yale School of Medicine and pediatric endocrinologist at Connecticut Children’s) and others suggested defining expert credentials or relying on specialty clinicians to reduce risk.

Members also linked the emergency-protocols work to reports that some hospitals are outsourcing emergency departments to third-party operators. The council expressed concern that outsourced ER management could impair access to patient medical records and increase the chance that staff will follow corporate protocols rather than an individual patient’s plan. Council members said the effect is likely to be greatest at smaller critical-access hospitals serving rural patients.

Council next steps Council leaders urged members to contact legislators—with special focus on the Appropriations Committee—about bills that carry high fiscal notes. Bennett recommended arranging meetings with the Human Services Committee on the intellectual-disability proposals and with emergency medical services to discuss how individualized protocols could be handled in practice. The council also asked members to prioritize two or three bills to actively support and to send letters to CGA members.

Ending RDAC members stressed that funding constraints at the federal level are shifting costs to the state this session, and that bills with fiscal notes above roughly $200,000 are likely to receive close scrutiny in appropriations. The council will continue active outreach and follow-up with legislative committees and with emergency-services stakeholders.