Committee advances bill to bar prior authorization for FDA-approved treatments used for rare diseases

The committee voted to advance Senate Bill 39, which would prohibit prior authorization and step-therapy requirements for FDA-approved drugs when used to treat conditions categorized as rare diseases.

Sponsor and supporters argued the change addresses delays and denials unique to rare-disease care, where few or no disease-specific clinical trials exist and off-label use of FDA-approved medicines is common. Former committee chair Debbie Armstrong described her own experience with a rare autoimmune muscle disease to illustrate the stakes: she said repeated denials delayed needed treatment until appeals succeeded, during a period when she lost functional ability; treatment restored much of her independence once approved.

The Office of the Superintendent of Insurance (OSI) life-and-health division director told the committee removing prior authorization in these narrow, defined cases would reduce barriers and speed access. Disability advocates and patient representatives voiced support, saying denials often fell hardest on people without experience navigating insurance appeals.

Committee members moved and seconded a due-pass recommendation for the bill as amended; the roll call recorded a unanimous 6–0 do-pass. Sponsors and OSI said technical details in the amended language clarify the bill applies to FDA-approved drugs (including off-label uses) for diseases that meet the statutory ‘‘rare disease’’ definition (fewer than 200,000 people in the U.S.). The measure will go forward for a full committee vote when the panel regains quorum and then proceed through the legislative process.