ACR 132: Rare Disease Day.

WHEREAS, A rare disease or disorder is a disease or disorder that affects a small number of patients; and

WHEREAS, In the United States, a rare disease or disorder affects fewer than 200,000 individuals; and

WHEREAS, As of the date of the adoption of this resolution, more than 30,000,000 individuals in the United States are living with at least 1 of the more than 10,000 known rare diseases or disorders; and

WHEREAS, Children with rare diseases or disorders account for a significant portion of the population affected by rare diseases or disorders in the United States; and

WHEREAS, Many rare diseases and disorders are serious and life-threatening; and

WHEREAS, This year marks the 43rd anniversary of the enactment of the federal Orphan Drug Act, a landmark law enabling tremendous advances in the research and treatment of rare diseases and disorders; and

WHEREAS, In 2022, the Center for Drug Evaluation and Research, in the United States Food and Drug Administration (FDA), established the Accelerating Rare disease Cures Program with a vision of speeding and increasing the development of effective and safe treatment options to address the unmet needs of patients with rare diseases; and

WHEREAS, Congress passed into law, as part of the Consolidated Appropriations Act of 2023 (H.Res. No. 2617), provisions creating the rare disease endpoint advancement pilot program in the FDA to support the development of novel efficacy endpoints to help facilitate the development and timely approval of rare disease treatments; and

WHEREAS, In 2024, California established the Jacqueline Marie Zbur Rare Disease Advisory Council to raise awareness of rare diseases and to provide recommendations to the Legislature to improve access to care; and

WHEREAS, Although the FDA has approved more than 1,100 drugs and biological products for an orphan indication for the treatment of a rare disease or disorder, approximately 90 percent of rare diseases do not have a treatment approved by the FDA for their condition; and

WHEREAS, Supported by California’s world-renowned research and education institutions, the California life sciences sector has led the way by developing innovative technologies and treatments. Nearly 200 rare disease drugs and treatments originated in California through a patient-first approach; and

WHEREAS, Limited treatment options and financing life-altering and lifesaving treatments can be challenging for individuals with rare diseases or disorders and their families; and

WHEREAS, Rare diseases and disorders include sickle cell anemia, spinal muscular atrophy, amyotrophic lateral sclerosis (ALS), thyroid eye disease, myotonic dystrophy, t-cell prolymphocytic leukemia, mucopolysaccharidosis (MPS), microtia, cystinosis, meatal atresia, conductive deafness, Batten disease, and achondroplasia; and

WHEREAS, Individuals with rare diseases or disorders can experience difficulty in obtaining accurate diagnoses and finding physicians or treatment centers with expertise in their rare disease or disorder; and

WHEREAS, The FDA and the National Institutes of Health support innovative research on the treatment of rare diseases and disorders; and

WHEREAS, Rare Disease Day is observed each year on the last day of February; and

WHEREAS, Rare Disease Day is a global event that was first established by EURORDIS (Rare Diseases Europe) in 2008 and first observed in the United States on February 28, 2009. It has grown to be observed by over 100 countries in 2026; and

WHEREAS, Rare Disease Day is expected to be observed globally for years to come, providing hope and information for rare disease and disorder patients around the world; now, therefore, be it

Resolved by the Assembly of the State of California, the Senate thereof concurring, That the Legislature designates February 28, 2026, as Rare Disease Day and, with respect to rare diseases and disorders, recognizes the importance of improving awareness, encouraging accurate and early diagnosis, and supporting national and global efforts to develop effective treatments, diagnostics, and cures; and be it further

Resolved, That the Chief Clerk of the Assembly transmit copies of this resolution to the author for appropriate distribution.