Lawmakers hear push for Vermont rare-disease advisory council as health department flags funding limits

The House Committee on Human Services on Monday heard several hours of testimony on H46, a bill to establish a Vermont Rare Disease Advisory Council, with patients and advocates urging lawmakers to act while state health officials warned the department lacks resources to host the council amid uncertain federal funding.

Why it matters: Proponents said a state-level advisory council would give patients and families a formal voice in policymaking, help coordinate newborn screening and registries, and offset recent federal changes that have reduced national advisory capacity. Health Department witnesses described existing screening and registry work in Vermont but said running a council would require new staffing and reimbursements that are not funded in the bill.

Kelly Dougherty, a Health Department presenter, told the committee that the department already runs multiple programs relevant to rare diseases, including the newborn screening program, the Vermont Birth Information Network (BIN), a cancer registry and an amyotrophic lateral sclerosis (ALS) registry. “We are sort of on the edge of our seats, wondering what’s gonna come next,” Dougherty said of recent federal program changes and grant terminations. She said newborns in Vermont are tested through a blood-spot panel of about 33 conditions, the BIN collects roughly 115 conditions and that statutes require cancer diagnoses to be reported to the state registry. Dougherty said the ALS registry (established by Act 149 of 2022) recorded 27 new Vermont ALS diagnoses in 2023.

Dougherty described a sudden federal reduction in several CDC and COVID-era supplements and named specific grants that were terminated March 24; she said the Health Department has absorbed some staff loss by attrition and is assessing which functions are statutorily required, which could be shifted to other funds, and which might need to stop if deeper cuts arrive. “We would not be able to keep doing everything that the health department does right now if we saw massive cuts,” she said.

Committee members asked whether the federal Advisory Committee on Heritable Disorders in Newborns and Children (the national panel that helped set the uniform screening panel) and other national programs being curtailed would affect Vermont screening and surveillance. Dougherty said the immediate practices in Vermont have not changed and the state continues current newborn screening, but added the department does not yet know the full effect of federal disbanding of advisory offices and loss of staff at the Centers for Disease Control and Prevention. “We don’t know yet,” she said, adding the state will follow developments and report back.

Advocates and patients urged lawmakers to create the council now. Leslie Canet, dean for the School of Science, Technology, Engineering and Mathematics at Vermont State University and a parent of a person with Fragile X, said the council would be “a platform for those who have rare diseases to have a stronger voice in state government” and asked the committee to advance H46. Carolyn Sheridan of the National Organization for Rare Disorders (NORD) told the committee that about 22 existing state councils are housed in health departments and several are hosted at universities, but that the health-department model became more common after the COVID-era influx of funds.

Patients and family members described personal impacts and the need for coordinated state supports. Katie Fockett, a Vermont patient with Ehlers-Danlos syndrome and Sjögren’s syndrome, said the bill’s timing is critical because the federal advisory committee’s termination “has created a serious gap in oversight for early detection programs” and that H46 explicitly asks the council to develop recommendations to improve newborn screening. Sarah Elliott, who has a form of dwarfism, said a state-level body could also inform nonmedical issues — housing, transportation and stigma — that affect quality of life. A parent, Suzette (last name not provided), described raising two children with CLN2 Batten disease and urged legislative action to speed treatment development and regulatory pathways.

On costs and placement, Dougherty said the Health Department would welcome participating in a council but lacks the resources to host and staff it, given federal funding uncertainty; she pointed to the bill’s absence of designated funding for council staffing, member reimbursements and travel. Committee members discussed alternatives, including university housing for the council; several witnesses noted some states host councils at universities while most currently reside within public health departments. One committee member said they would request a Joint Fiscal Office cost estimate for a council based on comparable state advisory bodies.

No formal action or vote on H46 occurred during the session. Committee members closed the hearing by asking staff to follow up with more information about national programs and cost estimates, and to circulate the health department’s presentation links and data.

The record: Health Department testimony cited Act 164 of 2024 (which requires a description of the public-health impact of rare diseases in Vermont), the state ALS law (Act 149 of 2022, chapter 4A, title 18) creating the ALS registry and statutory reporting requirements for the cancer registry. Advocates referenced national resources such as the National Organization for Rare Disorders and national newborn-screening panels. The Health Department requested clarification on the Birth Information Network’s data sources (Dougherty said BIN includes medical claims data and vital records and that she believed claims are from all insurers, but she offered to confirm).