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.

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.

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.

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 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 90 percent of rare diseases do not have a treatment approved by the FDA for their condition; and

WHEREAS, Supported by Californias 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, 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 100 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.

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 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 90 percent of rare diseases do not have a treatment approved by the FDA for their condition; and

WHEREAS, Supported by Californias 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, 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 100 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.

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 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 90 percent of rare diseases do not have a treatment approved by the FDA for their condition; and

WHEREAS, Supported by Californias 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, 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 100 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.

 THIRD READING 

Bill No: ACR 33

Author: Zbur (D), et al.

Introduced: 2/14/25

Vote: 21

SUBJECT: Rare Disease Day

SOURCE: Author

DIGEST:

This resolution designates 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.

ANALYSIS: This resolution makes the following legislative findings:

1. In the United States, a rare disease or disorder affects fewer than 200,000 individuals. Children with rare diseases or disorders account for a significant portion of the population affected by rare diseases or disorders in the United States.
2. 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. Many rare diseases and disorders are serious and life-threatening.
3. 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.
4. 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.

This resolution designates 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.

Related/Prior Legislation

HR 70 (Maienschein, 2024) – Adopted in the Assembly.

HR 16 (Maienschein, 2023) – Adopted in the Assembly.

HR 91 (Maienschein, 2022) – Adopted in the Assembly.

HR 25 (Bonta, Maienschein, and Waldron, 2021) – Adopted in the Assembly.

FISCAL EFFECT: Appropriation: No Fiscal Com.: No Local: No

SUPPORT: (Verified 3/4/25)

None received

OPPOSITION: (Verified 3/4/25)

None received

Prepared by: Aizenia Randhawa / SFA / (916) 651-1520

3/5/25 15:32:46

**** END ****

ASSEMBLY THIRD READING

ATR Bill Id:ACR 33

Author:(Zbur)

As Introduced Ver:February 14, 2025

Majority vote

SUMMARY

Designates February 28, 2025, as Rare Disease Day, and recognizes the importance of improving awareness, encouraging accurate and early diagnosis, and supporting national and global efforts to develop effective treatments, diagnostics, and cures.

Major Provisions

1. A rare disease or disorder, many of which are serious and life-threatening, affect a small number of patients. In the United States, a rare disease or disorder affects fewer than 200,000 individuals.
2. 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.
3. 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.
4. 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 % of rare diseases do not have a treatment approved by the FDA for their condition.
5. 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.
6. 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.
7. Limited treatment options and financing life-altering and lifesaving treatments can be challenging for individuals with rare diseases or disorders and their families.
8. Rare Disease Day is observed each year on the last day of February. 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 100 countries in 2024. 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.

COMMENTS

According to the Author

Rare disease patients face unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists, and obtaining fair insurance coverage. My sister, Jacqueline Marie Zbur, endured a three-year battle with ALS, a rare and devastating neurological disease that results in a gradual shut down of one’s body. Recognizing February 28th as Rare Disease Day serves as a crucial step in addressing the unmet needs of the rare disease community by raising public awareness and highlighting the necessity for enhanced research and innovation to continue improving health outcomes for patients.

Arguments in Support

None on file

Arguments in Opposition

None on file

FISCAL COMMENTS

No fiscal impact. This resolution is keyed non-fiscal by Legislative Counsel.

VOTES

ASM RULES: 10-0-1

YES: Pacheco, Lackey, Ahrens, Mark González, Irwin, Krell, Patel, Michelle Rodriguez, Sanchez, Zbur

ABS, ABST OR NV: Flora

UPDATED

VERSION: February 14, 2025

CONSULTANT: Michael Erke / RLS. / (916) 319-2800 FN: 0000028

Date of Hearing: February 18, 2025

ASSEMBLY COMMITTEE ON RULES

Blanca Pacheco, Chair

ABPCA Bill Id:ACR 33 (

Author:Zbur) – As Introduced Ver:February 14, 2025

SUBJECT: Rare Disease Day.

SUMMARY: Designates February 28, 2025, as Rare Disease Day, and recognizes the importance of improving awareness, encouraging accurate and early diagnosis, and supporting national and global efforts to develop effective treatments, diagnostics, and cures. Specifically, this resolution makes the following legislative findings:

1. A rare disease or disorder, many of which are serious and life-threatening, affect a small number of patients. In the United States, a rare disease or disorder affects fewer than 200,000 individuals.
2. 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.
3. 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.
4. 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.
5. 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.
6. 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.
7. Limited treatment options and financing life-altering and lifesaving treatments can be challenging for individuals with rare diseases or disorders and their families.
8. Rare Disease Day is observed each year on the last day of February. 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 100 countries in 2024. 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.

FISCAL EFFECT: This resolution is keyed non-fiscal by Legislative Counsel.

REGISTERED SUPPORT / OPPOSITION:

Support

None on file

Opposition

None on file

Analysis Prepared by: Michael Erke / RLS. / (916) 319-2800