California lawmakers should be commended. They have taken a crucial step toward combatting rare diseases by establishing the state’s first Rare Disease Advisory Council.
A New, Collective Voice for Rare Diseases
Rare diseases impact one in 10 Californians, a community that will now be served better because of a dedicated platform focused on addressing specific needs of rare disease patients across the Golden State.
The Council will advise California’s state agencies and its legislature on policy issues relevant to rare diseases, bringing the expertise of health care providers, caregivers, researchers, industry stakeholders, and patients directly to the lawmaking process.
California joins 28 other states with similar councils, a growing majority where the rare disease community now has a voice in policies that shape patient care.
Building Awareness and Support in California
Rare diseases are so named because each impacts fewer than 200,000 Americans. But rare diseases are, collectively, not rare at all. In fact, the National Institutes of Health estimates there may be over 7,000 rare diseases, collectively impacting about 30 million people. That’s 10% of the country’s population, or about the same number living with diabetes.
California’s new council, like its counterparts in other states, is charged with educating policymakers, government agencies and the public about rare diseases. Such information may include research and funding opportunities, as well as diagnosis and treatment options, ensuring that patients, their families and health care providers have a central repository for current information. The council also offers broader rare disease stakeholders including health insurers, biotech innovators, disease researchers and patient advocacy organizations an avenue to unite with these core groups under a visible, dedicated umbrella.
Using Real-World Experiences to Shape Policy
Because rare diseases are so diverse, policymakers may be overwhelmed by the idea of serving this unique constituency.
But by sharing lived experiences and publishing resources, the council can help educate legislators and state government officials about the collective priorities of Californians with rare diseases. These policymakers, in turn, can implement meaningful change on behalf of the community. In this way, the council’s advocacy can help patients and providers get the support and protections they need under the law and through health care agencies.
This type of activity, undertaken by rare disease councils across the country, is a crucial part of a long-term effort to raise awareness of rare diseases and increase support for their research – the type that will one day make them history.
