20250ACR_003397CHP INTRODUCED 2025-02-14 PASSED_ASSEMBLY 2025-02-24 PASSED_SENATE 2025-03-13 ENROLLED 2025-03-14 CHAPTERED 2025-03-18 FILED 2025-03-18 2025 ACR CHP CHR 0 Introduced by Assembly Member Zbur (Principal coauthor: Assembly Member Dixon) (Principal coauthor: Senator Blakespear) (Coauthors: Assembly Members Addis, Aguiar-Curry, Ahrens, Alanis, Arambula, Bauer-Kahan, Bennett, Berman, Boerner, Bonta, Calderon, Caloza, Castillo, Chen, Connolly, Davies, DeMaio, Elhawary, Flora, Fong, Gabriel, Gallagher, Garcia, Gipson, Jeff Gonzalez, Mark González, Hadwick, Haney, Harabedian, Hart, Hoover, Irwin, Jackson, Kalra, Lackey, Lee, Lowenthal, Macedo, McKinnor, Nguyen, Ortega, Pacheco, Papan, Patel, Patterson, Pellerin, Petrie-Norris, Quirk-Silva, Ramos, Ransom, Rivas, Celeste Rodriguez, Michelle Rodriguez, Rogers, Blanca Rubio, Sanchez, Schiavo, Schultz, Sharp-Collins, Soria, Stefani, Ta, Tangipa, Valencia, Wallis, Ward, Wicks, and Wilson) LEAD_AUTHOR ASSEMBLY Zbur PRINCIPAL_COAUTHOR ASSEMBLY Dixon PRINCIPAL_COAUTHOR SENATE Blakespear COAUTHOR ASSEMBLY Addis COAUTHOR ASSEMBLY Aguiar-Curry COAUTHOR ASSEMBLY Ahrens COAUTHOR ASSEMBLY Alanis COAUTHOR ASSEMBLY Arambula COAUTHOR ASSEMBLY Bauer-Kahan COAUTHOR ASSEMBLY Bennett COAUTHOR ASSEMBLY Berman COAUTHOR ASSEMBLY Boerner COAUTHOR ASSEMBLY Bonta COAUTHOR ASSEMBLY Calderon COAUTHOR ASSEMBLY Caloza COAUTHOR ASSEMBLY Castillo COAUTHOR ASSEMBLY Chen COAUTHOR ASSEMBLY Connolly COAUTHOR ASSEMBLY Davies COAUTHOR ASSEMBLY DeMaio COAUTHOR ASSEMBLY Elhawary COAUTHOR ASSEMBLY Flora COAUTHOR ASSEMBLY Fong COAUTHOR ASSEMBLY Gabriel COAUTHOR ASSEMBLY Gallagher COAUTHOR ASSEMBLY Garcia COAUTHOR ASSEMBLY Gipson COAUTHOR ASSEMBLY Jeff Gonzalez COAUTHOR ASSEMBLY Mark González COAUTHOR ASSEMBLY Hadwick COAUTHOR ASSEMBLY Haney COAUTHOR ASSEMBLY Harabedian COAUTHOR ASSEMBLY Hart COAUTHOR ASSEMBLY Hoover COAUTHOR ASSEMBLY Irwin COAUTHOR ASSEMBLY Jackson COAUTHOR ASSEMBLY Kalra COAUTHOR ASSEMBLY Lackey COAUTHOR ASSEMBLY Lee COAUTHOR ASSEMBLY Lowenthal COAUTHOR ASSEMBLY Macedo COAUTHOR ASSEMBLY McKinnor COAUTHOR ASSEMBLY Nguyen COAUTHOR ASSEMBLY Ortega COAUTHOR ASSEMBLY Pacheco COAUTHOR ASSEMBLY Papan COAUTHOR ASSEMBLY Patel COAUTHOR ASSEMBLY Patterson COAUTHOR ASSEMBLY Pellerin COAUTHOR ASSEMBLY Petrie-Norris COAUTHOR ASSEMBLY Quirk-Silva COAUTHOR ASSEMBLY Ramos COAUTHOR ASSEMBLY Ransom COAUTHOR ASSEMBLY Rivas COAUTHOR ASSEMBLY Celeste Rodriguez COAUTHOR ASSEMBLY Michelle Rodriguez COAUTHOR ASSEMBLY Rogers COAUTHOR ASSEMBLY Blanca Rubio COAUTHOR ASSEMBLY Sanchez COAUTHOR ASSEMBLY Schiavo COAUTHOR ASSEMBLY Schultz COAUTHOR ASSEMBLY Sharp-Collins COAUTHOR ASSEMBLY Soria COAUTHOR ASSEMBLY Stefani COAUTHOR ASSEMBLY Ta COAUTHOR ASSEMBLY Tangipa COAUTHOR ASSEMBLY Valencia COAUTHOR ASSEMBLY Wallis COAUTHOR ASSEMBLY Ward COAUTHOR ASSEMBLY Wicks COAUTHOR ASSEMBLY Wilson Relative to Rare Disease Day.

Rare Disease Day Rare Disease Day. This measure would designate February 28, 2025, as Rare Disease Day and, with respect to rare diseases and disorders, would recognize the importance of improving awareness, encouraging accurate and early diagnosis, and supporting national and global efforts to develop effective treatments, diagnostics, and cures. NO

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 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 42nd 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 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 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, Sanfilippo syndrome, microtia, cystinosis, meatal atresia, and conductive deafness; 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 observed in the United States on February 28, 2009, and has grown to be observed by over countries in 2024; 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, 2025, 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.