On January 26, 2023, the Rare Disease Legislative Advocates (RDLA) hosted their 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. In this program, representatives from a number of organizations focused on rare disease advocacy discussed their policy goals for the upcoming year.

Speakers for this program included:

• Jamie Sullivan, EveryLife Foundation for Rare Diseases
• Corinne Alberts, National Organization for Rare Disorders
• Jenifer Waldrop, Rare Disease Diversity Coalition
• Saira Sultan, Haystack Project
• Eric Gascho, National Health Council
• David Davenport, Personalized Medicine Coalition
• Ellie Dehoney, Research!America

All of these speakers discussed the policy goals that their respective organizations were aiming to pursue in 2023. There was a lot of overlap in the goals that each organization shared, indicating that there was a broad sense of unity in what the rare disease and chronic illness community is hoping to achieve in terms of political goals.

A major goal shared by many of the speakers was improving the diagnosis process for rare disease patients, a process that is often so long and arduous that it has frequently been dubbed the ‘diagnostic odyssey.’

Improving access to care is the solution to this issue. This can mean pursuing legislation that changes the regulations surrounding genetic testing, which is often essential for diagnosis, particularly for heritable disorders. Another focus that will contribute to solving this problem is the modernization of newborn screening resources.

With the new Congress, some of the legislators that were working hard to push the priorities of the rare disease community, such as G.K. Butterfield, have retired, meaning that new allies will have to be found. Thankfully, some progress has already been made on this front.

Some specific bills that are goals of the rare community on the federal level include:

• Cures 2.0
• VALID Act (oversight of lab-developed tests)
• Precision Medicine Answers for Kids Today Act
• 2024 NIH and FDA budget appropriations

A full recording of the program can be found here.