On January 18, 2024, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives that are relevant to the treatment and management of rare diseases under the law. This month’s program focused on the upcoming year and the policy priorities that rare disease organizations are working to see progress on, both at the federal and state level.

Speakers for this month’s program included:

• Cynthia Bens, Personalized Medicine Coalition
• Shelia Murphy, Research!America
• Karin Hoelzer, National Organization for Rare Disorders
• Jamie Sullivan, EveryLife Foundation for Rare Diseases
• Jennifer Dexter, National Health Council

Many of the speakers and organizations represented during the program shared similar objectives. Cynthia highlighted several of them, such as:

• Improving regulations on subjects such as payment policies and coverage
• The Precision Medicine Answers for Kids Today Act
• Building support for personalized medicine initiatives
• Building out the Congressional Personalized Medicine Caucus

Shelia touched on other areas of focus such as aspiring to increase funding for various federal research agencies and scientific institutions, such as the National Institutes of Health (NIH) and Centers for Disease Control and Prevention (CDC). Another area that was mentioned was aiming to counteract provisions of the Inflation Reduction Act (IRA) that could have an unintended detrimental effect on rare disease drug development.

Karin noted that NORD will continue to push its Rare Disease Advisory Council initiative at the state level, with a steadily increasing number of states adopting the model in the last few years. The organization also wants to improve regulations surrounding telehealth options, which have become more valuable since the pandemic. Specific legislation that the group is supporting includes the Safe Step Act, which aims to regulate the exploitative ‘step therapy’ tactic often used by insurance companies, and the Accelerating Kids Access to Care Act.

Jamie touched on the need to improve consistency and approval and other regulatory decisions made by the US Food and Drug Administration (FDA). EveryLife also seeks to update and improve the Accelerated Approval regulatory pathway.

To see the full program, click here.