On December 5, 2023, the Rare Disease Legislative Advocates (RDLA) held an RDLA Caucus briefing. This briefing, held in collaboration with the Rare Disease Congressional Caucus, provided an update on the status of rare disease-related legislation currently under brewing in the US Congress.

A congressional caucus is a group of legislators that meet with the goal of working together to promote and pass shared legislative objectives, typically centered around a specific political perspective or a pressing issue. The Rare Disease Congressional Caucus is a caucus with bicameral membership and bipartisan membership. Co-chairs of the caucus include Rep. Doris Matsui (D-CA), Rep. Gus Bilirakis (R-FL), Senator Amy Klobuchar (D-MN), and Senator Roger Wicker (R-MS).

Nicholas Manetto of Faegre Drinker Consulting shared updates on the Accelerating Kids Access to Care Act, which aims to:

• Establish a streamlined federal screening pathway for enrollment on out-of-state Medicaid
• Improve access to out-of-state care for children diagnosed with rare disorders

Out-of-state care is a common requirement for rare disease patients, who may not have access to specialty care within their home state. It has support from 25 senators and 100 representatives, as well as endorsement from over 200 advocacy groups. It’s currently in senate committees and having potential costs evaluated through the Congressional Budget Office (CBO).

Kyle Underwood lives with Hunter syndrome and shared his personal story. While he leads a relatively normal life, he believes that the legislation would have enabled him to get higher quality, top-class treatment more quickly.

Cynthia Bens from the Personalized Medicine Coalition discussed the Precision Medicine Answers for Kids Today Act. It would institute diagnostic coverage guidance to states for Medicaid and fund a demonstration program lasting three years for 15 states that would cover genetic testing/genome sequencing for children living with undiagnosed chronic conditions. It would also fund research on genetic testing access.

Jackie Weinreich, a staffer for Rep. Matsui, introduced the PROTECT Rare Act. Drugs are often prescribed off-label for the treatment of rare disease, but insurance companies often push back and deny coverage for off-label use.

• The bill would allow Medicaid/Medicare to expand criteria for medically accepted indications, improving off-label coverage.
• Insurance companies would be forced to expedite the review of off-label prescriptions, particularly in cases involving rare disease.

To view the full briefing, click here.