SACRAMENTO, CA, April 24, 2024—Today, California is one step closer to establishing a Rare Disease Advisory Council (RDAC) to help the 1-in-10 Californians living with rare disease access critical care and services. Legislation to establish an RDAC was introduced by Assemblymember Rick Zbur in February and the bill reached a milestone in passing out of the California Assembly Committee on Health yesterday.
The California RDAC will provide opportunities for patients and advocates throughout the state to educate lawmakers about the needs of those with rare diseases and make formal policy recommendations to state leaders about pressing issues. Patients with rare disorders face unique challenges every day, and often lawmakers struggle to understand the complex needs rare disease patients and their families.
“As of today, 28 states have established a Rare Disease Advisory Council. If California wishes to remain a leader in implementing policies that support patients living with rare diseases, then we cannot miss the opportunity to establish a Rare Disease Advisory Council this session. AB2613 offers the chance to do so, which could help improve health outcomes and patient quality of life in our state,” said Lindsey Viscarra, State Policy Manager, National Organization for Rare Disorders, who provided testimony during yesterday’s hearing. “NORD thanks Assemblymember Zbur for introducing this legislation to support Californians who are living with rare disease.”
AB2613 is a priority bill for the National Organization for Rare Disorders (NORD) and its Rare Action Network, and also for the more than two dozen patient organizations who are advocating for the bill’s passage. NORD, our coalition partners, and grassroots advocates are urging California state leaders to support this effort and ensure rare disease patients have a say in important healthcare and other policy decisions.
Mike Hu, a NORD Rare Action Network advocate and member of the California RDAC Coalition, sees a tremendous need for establishing the Rare Disease Advisory Council:
“I am a father of two boys, who are 16 and 13 now. They are both affected by an ultra-rare genetic disease called Mucopolysaccharidosis II, or MPS II. It’s a systemic disease that affects every tissue and organ, which causes physical disabilities, developmental delays, and eventually leads to organ failures and death in early adulthood. With only about 400 diagnosed patients in the entire country, virtually no one knows about the disease, including many of their doctors. During the past 13 years battling with this rare disease, my family have experienced the many challenges that the rare disease community face every single day, such as the diagnostic odyssey, accessing proper medical care and therapies, battling insurance coverage, public school placement, obtaining regional center support, just to name a few. As a rare disease family, we must educate everyone who interacts with my sons, and advocate for every bit of resource and support they need. Our goal is for the RDAC to raise awareness about rare disorders, provide valuable knowledge and information to policymakers, and to be a resource to individuals and families like ours as well as to medical providers who support these patients. “
With over 10,000 rare diseases, it is difficult for state policymakers and government officials to have an in-depth understanding of the complex needs of their state’s rare disease community. This lack of understanding contributes to obstacles that many rare disease patients face, such as delays in obtaining an accurate diagnosis, lack of treatment options, high out-of-pocket costs, and limited access to medical specialists. Rare Disease Advisory Councils are designed to help identify and address the unique challenges faced by individuals living with a rare disease. Through Project RDAC, NORD is building coalitions of advocates to support creation of an RDAC and developing the tools and resources necessary to ensure RDACs benefit the rare disease community in their state.
The push to establish a California Rare Disease Advisory Council has drawn the support of nonprofit patient advocacy groups, medical professionals, and rare disease patients and caregivers. Next, the bill must be heard in the Fiscal Committee before a vote on the Assembly floor, which must take place by May 24 for the bill to continue to advance in the 2024 Legislative session. NORD encourages any individuals looking to get involved in rare disease advocacy to join the NORD Rare Action Network today and for California residents to contact their elected officials today urging support for the California Rare Disease Advisory Council.
