Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill, upon appropriation by the Legislature, would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council appointed by the State Public Health Officer, as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its web page publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report on its web page on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill, upon appropriation by the Legislature, would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council appointed by the State Public Health Officer, as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its web page publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report on its web page on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill bill, upon appropriation by the Legislature, would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council appointed by the State Public Health Officer, as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its website a list of existing, web page publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report to the agency and the Legislature on its web page on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council, composed of a minimum of 17 members, appointed Council appointed by the State Public Health Officer, as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its website a list of existing, publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report to the agency and the Legislature on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council, composed of a minimum of 17 members, appointed as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its website a list of existing, publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report to the agency and the Legislature on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

Existing law establishes the California Health and Human Services Agency, which includes the State Department of Public Health, among other state departments charged with the administration of health, social, and other human services. Under existing law, the State Department of Public Health has authority over various programs promoting public health, including genetic disease testing.

This bill would establish within the agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council, composed of a minimum of 17 members, appointed as specified. The council would generally act as the advisory body on rare diseases to the Legislature and state and private entities that provide services to, or that are charged with the care of, persons with rare diseases. The council specifically would be required, among other duties, to publish on its website a list of existing, publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment. The bill would require the council to report to the agency and the Legislature on the activities of the advisory council and its findings and recommendations on issues, as specified. The bill would make legislative findings and declarations in support of its provisions.

CONCURRENCE IN SENATE AMENDMENTS

CSA1 Bill Id:AB 2613

Author:(Zbur)

As Amended Ver:August 23, 2024

Majority vote

SUMMARY

Establishes within the California Health and Human Services Agency (CalHHS), until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC). Requires the RDAC to act as the advisory body on rare diseases to the Legislature, consult with experts on rare diseases to develop recommendations to improve patient access to needed services, apply for and accept grants, and report its activities and findings on its webpage, among other duties.

Senate Amendments

1. Change the legislative findings regarding the number of known rare diseases from 7,000 to 10,000.
2. Make this bill contingent upon appropriation of the Legislature.
3. Require the Secretary of CalHHS, rather than the Governor, to appoint the chair of the RDAC.
4. Limit the membership of the RDAC as specified in provisions of this bill.
5. Require the State Public Health Officer, rather than the chair of the RDAC, to appoint the Members of the RDAC.
6. Require members of the RDAC to either reside in California or be employed by a California-based company or organization.
7. Change the requirement for the registered professional nurse member of the RDAC from requiring them to have expertise in providing care to patients with rare diseases to instead have experience in providing care to patients with rare diseases.
8. Change the requirement for the physician and surgeon members of the RDAC from requiring them to have expertise in treating patients with rare diseases, at least one of whom has also treated children with rare diseases to instead require these members to have experience treating patients with rare diseases, at least one of whom has also treated children.
9. Updates the reference from representative of the administration of a hospital located in this state that treats rare disease patients to a hospital administrator employed by a hospital that treats rare disease patient.
10. Delete the representative of the administration of an outpatient health care facility located in this state and treats rare disease patients.
11. Change the membership number of individuals who are either a rare disease patient or caregiver to a rare disease patient from five to up to five.
12. Change the membership number of representatives of patient advocacy organizations that operate within the state from two to up to two.
13. Delete the dates by which the chair of the RDAC and the members of the RDAC must be appointed.
14. Delete the prohibition for a single rare disease population to have more than two representation on the RDAC.
15. Delete the requirement for the RDAC to hold an initial organizational meeting within 60 days after the date of the last appointment made.
16. Delete the duties of the RDAC to evaluate and make recommendations to improve Medi-Cal and state-regulated private health insurance coverage of drugs and biological products for rare disease patients; engage with the Medi-Cal Drug Use Review Board, and prepare a first-year landscape survey of the needs of rare disease patients, caregivers, and providers in the state.
17. Delete the requirement for the RDAC to report to CalHHS and the Legislature by specified dates and instead require the council to report on their website.

COMMENTS

Background on Rare Diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the United States. There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans. Examples of rare diseases include life-threatening and physically or mentally disabling conditions such as ALS; Huntington disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy.

According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations and many rare diseases have a long list of characteristics that present serious challenges for public health practitioners. Among rare diseases it is common to find that: 1) diagnoses are difficult and delayed; 2) case definitions for surveillance are usually lacking; 3) International Classification of Diseases codes for record keeping are poorly defined or not assigned; 4) underlying molecular or physiologic mechanisms are unknown; 5) specialized and coordinated medical care is in short supply, and treatments can be complex; g standards of care for treatment and rehabilitation are not evidence-based because health research is necessarily done at small scale; 6) longitudinal data collections are scarce; 7) the development of new medications and treatments can be fragmented and slow; 8) screening strategies lack efficiency; and, 9) scope and capacity of most registries and databases are limited.

According to the Author

This bill will create a RDAC in California. The author's sister, Jacqueline Marie Zbur, endured a three-year battle with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. The author states that the RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and will guide the Legislature in supporting the rare disease community. The author notes that rare disease patients face unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access for quality care and therapies, and obtaining fair insurance coverage of their treatment and care. The author contends that this bill makes important progress towards addressing challenges for the rare disease community by fostering recommendations on – and understanding of – disease representation, specialist physician directories, financial impacts to patients and the State, and what private and public resources may be available to patients and their families. The author also notes that this bill gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare diseases. The author contends that it is time for California to join 27 other states in adopting a RDAC, through which a diverse group of experts will be able to develop recommendations for the State on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.

Arguments in Support

A coalition of organizations representing individuals with rare diseases including the National Organization for Rare Disorders, the California Center for Rare Diseases at the University of California, Las Angeles, Hemophilia Council of California, and the California Chronic Care Coalition state, for patients living with one of the over 10,000 known rare conditions, it can take several years to receive an accurate diagnosis and effective treatment. Further, only a handful of rare diseases are well understood, with most not receiving sufficient attention or funding for research. This lack of awareness often contributes to the obstacles to timely treatment and care faced by many rare disease patients. The coalition states that creating an RDAC in California will give rare disease patients a unified voice in state government to help address challenges that are faced by the rare disease community by serving as the advisory body on rare diseases to the Legislature and state departments, and relieve some of the burden on the state by expeditiously delivering direct feedback, solutions, and resources to California.

Arguments in Opposition

The Department of Finance is opposed to this bill because the Council's workload creates new General Fund costs not included in the 2024 Budget Act. Finance notes that the Council's recommendations may create unknown General Fund cost pressures to expand Medi-Cal pharmacy benefits to cover additional drugs and biological products to treat rare diseases. The author's intent is to address the significant challenges people with rare diseases face by creating an advisory council in memory of Jaqueline Marie Zbur, the author's sister, who endured a three-year battle with Amyotrophic Lateral Sclerosis.

FISCAL COMMENTS

According to the Senate Appropriations Committee, CalHHS anticipates delegating the responsibility to the California Department of Public Health (DPH). DPH estimates annual General Fund costs of approximately $371,000 from 2024-25 through 2028-29, and additional General Fund costs of $278,000 in 2025-26, 2026-27, and 2027-28 for state administration to support the council.

VOTES:

ASM HEALTH: 16-0-0

YES: Bonta, Waldron, Aguiar-Curry, Arambula, Wendy Carrillo, Flora, Haney, Jones-Sawyer, Maienschein, McCarty, Joe Patterson, Rodriguez, Sanchez, Santiago, Schiavo, Weber

ASM APPROPRIATIONS: 15-0-0

YES: Wicks, Sanchez, Arambula, Bryan, Calderon, Wendy Carrillo, Dixon, Mike Fong, Grayson, Haney, Hart, Jim Patterson, Pellerin, Ta, Villapudua

ASSEMBLY FLOOR: 72-0-8

YES: Addis, Aguiar-Curry, Alanis, Alvarez, Arambula, Bains, Bauer-Kahan, Bennett, Berman, Boerner, Bonta, Bryan, Juan Carrillo, Wendy Carrillo, Chen, Connolly, Davies, Dixon, Flora, Mike Fong, Vince Fong, Gabriel, Gallagher, Garcia, Gipson, Grayson, Haney, Hart, Hoover, Irwin, Jackson, Jones-Sawyer, Kalra, Lackey, Lee, Low, Lowenthal, Maienschein, McCarty, McKinnor, Muratsuchi, Stephanie Nguyen, Ortega, Pacheco, Papan, Jim Patterson, Joe Patterson, Pellerin, Petrie-Norris, Quirk-Silva, Ramos, Rendon, Reyes, Rodriguez, Blanca Rubio, Sanchez, Santiago, Schiavo, Soria, Ta, Ting, Valencia, Villapudua, Waldron, Wallis, Ward, Weber, Wicks, Wilson, Wood, Zbur, Robert Rivas

ABS, ABST OR NV: Calderon, Cervantes, Megan Dahle, Essayli, Friedman, Holden, Mathis, Luz Rivas

SENATE FLOOR: 40-0-0

YES: Allen, Alvarado-Gil, Archuleta, Ashby, Atkins, Becker, Blakespear, Bradford, Caballero, Cortese, Dahle, Dodd, Durazo, Eggman, Glazer, Gonzalez, Grove, Hurtado, Jones, Laird, Limón, McGuire, Menjivar, Min, Newman, Nguyen, Niello, Ochoa Bogh, Padilla, Portantino, Roth, Rubio, Seyarto, Skinner, Smallwood-Cuevas, Stern, Umberg, Wahab, Wiener, Wilk

UPDATED

VERSION: August 23, 2024

CONSULTANT: Eliza Brooks / HEALTH / (916) 319-2097 FN: 0004706

 THIRD READING  

Bill No: AB 2613

Author: Zbur (D), et al.

Amended: 8/23/24 in Senate

Vote: 21

SENATE HEALTH COMMITTEE: 11-0, 6/26/24

AYES: Roth, Nguyen, Glazer, Gonzalez, Grove, Hurtado, Limón, Menjivar, Rubio, Smallwood-Cuevas, Wiener

SENATE APPROPRIATIONS COMMITTEE: 6-0, 8/15/24

AYES: Caballero, Jones, Ashby, Becker, Seyarto, Wahab

NO VOTE RECORDED: Bradford

ASSEMBLY FLOOR: 72-0, 5/22/24 - See last page for vote

SUBJECT: Jacqueline Marie Zbur Rare Disease Advisory Council

SOURCE: National Organization for Rare Disorders

DIGEST:

Establishes, upon appropriation by the Legislature for this purpose, the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC) as an advisory body to advise and report to the California Health and Human Services Agency (CHHSA) and the Legislature on strategies to address barriers and improve treatment services for those with rare diseases in this state.

Senate Floor Amendments of 8/23/24 incorporate technical assistance from Governor Newsom’s Administration to narrow the scope of RDAC, which in turn may help mitigate its fiscal impact, as follows:

1. Require the Secretary of CHHSA, rather than the Governor, to appoint the RDAC chair;
2. Require RDAC appointed members to reside in the state or be employed by a California-based organization;
3. Delete the requirement that RDAC hold its initial meeting within 60 days after the date the last member is appointed;
4. Reduce the number of times per year RDAC is required to meet from at least six times to at least two;
5. Delete various RDAC duties, including evaluating and recommending improvements to public and private health insurance care coverage of drugs and biological products for rare disease patients; engaging with the Medi-Cal Drug Use Review Board; and, preparing a first year landscape survey of the needs of rare disease patients, caregivers, and providers in the state;
6. Delete required reports from RDAC to the Legislature; and,
7. Make RDAC contingent upon appropriation from the Legislature.

ANALYSIS:

Existing law:

1. Establishes in state government CHHSA, within which, among other departments, offices and councils, exists the Department of Health Care Services (DHCS) and the California Department of Public Health (CDPH). [GOV §12800, §12803]
2. Establishes in state government the State Public Health Officer, who serves as the director of CDPH. [HSC §131005]
3. Establishes the California Children’s Services Program (CCS), administered by DHCS, under which individuals under the age of 21 who have specified health conditions and meet financial requirements are eligible to receive medically necessary services and treatments. [HSC §123800, et seq.]
4. Establishes the Genetically Handicapped Persons Program (GHPP), administered by DHCS, under which individuals who have specified genetic conditions are eligible to receive medical care and social support services. [HSC §125125, et seq.]
5. Establishes the Hereditary Disorders Act, under which CDPH administers the Genetic Disease Screening Program (GDSP), which provides a statewide program for newborn and prenatal testing for genetic disorders and tracking of birth defects. Requires GDSP to provide genetic screenings and follow up services for persons who have screenings. [HSC §124975, et seq.]

This bill:

1. Establishes, within CHHSA and until January 1, 2029, and upon appropriation from the Legislature, the Jacqueline Marie Zbur RDAC as an advisory body.
2. Requires the Secretary of CHHSA to appoint the RDAC chair. Prohibits the chair from holding any other position, employment, or appointment within state government.
3. Requires the State Public Health Officer to appoint 17 members to serve without compensation (except for specified reimbursements, if funds are made available for this purpose), as follows:
   1. Two licensed physicians who have experience in treating patients with rare diseases, at least one of whom treats children with rare diseases;
   2. One registered licensed nurse who has expertise in providing care to patients with rare diseases;
   3. One representative of a hospital located in this state and treats rare disease patients;
   4. One representative of an outpatient health care facility located in this state that treats rare disease patients;
   5. One representative of the California health care coverage industry;
   6. One representative of the biopharmaceutical industry;
   7. One representative of the scientific community who is engaged in rare disease research;
   8. One rare disease geneticist or genetic counselor;
   9. Up to five individuals who are either a rare disease patient or a caregiver to a rare disease patient. Prohibits RDAC from including more than three individuals from each category;
   10. One medical social worker or mental health provider who works with rare disease patients; and,
   11. Up to two representatives of patient advocacy organizations that operate within this state.
4. Requires members appointed by the chair to serve on RDAC until January 1, 2029, upon which the provisions of this bill sunset. Requires any vacancy to be filled in the same manner as provided for in the original appointment.
5. Requires each RDAC member to sign a conflict of interest statement annually disclosing any economic or other relationship with an entity that could influence the member’s decisions. Require at least 20% of RDAC’s members to not have a conflict of interest with respect to an insurer, pharmaceutical benefits manager, or pharmaceutical manufacturer.
6. Requires RDAC to perform all of the following duties:
   1. Act as the advisory body on rare diseases to the Legislature and state departments, agencies, commissions, and authorities, as well as private agencies, that provide services to, or that are charged with the care of, persons with rare diseases;
   2. Consult with experts on rare diseases to develop recommendations to improve patient access to, and quality of, rare disease specialists, affordable and comprehensive health care coverage, relevant diagnostics, timely treatment, and other needed services;
   3. Create and maintain a web page and identify, consolidate, and publish a list of publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment; and,
   4. Identify areas of unmet need for research and opportunities for collaboration with stakeholders and RDACs in other states that can inform future studies and work done by RDAC in this state.
7. Requires RDAC to meet periodically, but at least two times annually. Permits RDAC to call to its assistance, and avail itself of the services of, the employees of any state, county, or municipal department, board, bureau, commission, or agency as it may require and as may be available, if those employees elect to participate.
8. Requires RDAC to apply for, and accept, any grant of funds from the federal government, private foundations, or other sources that may be available for programs related to rare diseases. Prohibits RDAC from accepting any funds from the employer of a currently seated council member.
9. Requires RDAC to report on activities, findings, and recommendations on issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases on its web page.

Comments

1. Author’s statement. According to the author, “this bill creates RDAC in the memory of his sister, Jacqueline Marie Zbur, who endured a three-year battle with amyotrophic lateral sclerosis, (ALS, also known as Lou Gehrig’s disease). RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and guide the Legislature in supporting the rare disease community, which faces unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access to quality care and therapies, and obtaining fair insurance coverage for their treatment and care. This bill makes important progress toward addressing challenges for the rare disease community by fostering recommendations on, and understanding of, disease representation, specialist physician directories, financial impacts to patients and the state, and what private and public resources may be available to patients and their families. RDAC gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare disease. It is time for California to join 28 other states in adopting an RDAC, through which a diverse group of experts will be able to develop recommendations for the state on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.”
2. Rare diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the U.S. Altogether, rare diseases affect an estimated 30 million Americans. Rare diseases include life-threatening and physically or mentally disabling conditions, such as Huntington’s disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy. According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations, and many rare diseases have a long list of characteristics that present serious challenges for practitioners. Among rare diseases it is common to find that:
   1. Diagnoses are difficult and delayed;
   2. Case definitions for surveillance are usually lacking;
   3. International Classification of Diseases (ICD) codes for record keeping are poorly defined or not assigned;
   4. Underlying molecular or physiologic mechanisms are unknown;
   5. Specialized and coordinated medical care is in short supply, and treatments can be complex;
   6. Standards of care for treatment and rehabilitation are not evidence-based because health research is done at small scale;
   7. Longitudinal data collections are scarce;
   8. The development of new medications and treatments can be fragmented and slow;
   9. Screening strategies lack efficiency; and,
   10. The scope and capacity of most registries and databases are limited.

The knowledge of most rare diseases is so insufficient that they are also known as orphan diseases (and their treatments known as orphan drugs) because of their failure to attract the interest of researchers, medical specialists, drug makers, and policy makers.

1. State programs related to rare diseases. DHCS and CDPH operate state programs that provide services and screenings for individuals with certain diseases and health problems, which include some rare diseases. These programs include the CCS program, GHPP, and GDSP:
   1. CCS. CCS is a state-only health program for children, up to the age 21, with certain diseases or health problems, including bleeding disorders, cystic fibrosis, Duchenne muscular dystrophy, and other conditions. CCS requires applicants to meet additional requirements, such as income limits. CCS provides coverage for a variety of medical visits, medical case management, and care at sickle cell disease centers;
   2. GHPP. GHPP is a state-only health program that provides care for adults, aged 21 and older, with certain genetic diseases, including rare blood diseases, cystic fibrosis, diseases of brain, nerves and metabolism, and other diseases. There is no income limit; however, applicants may be required to pay an annual enrollment fee, which is based on income and family size. GHPP benefits include coverage for hospital stays, outpatient medical care, pharmaceutical services, surgeries, nutritional products, durable medical equipment, care at a Special Care Center, and other services. Individuals with private health insurance or Medicare coverage may apply for GHPP, but the program will only cover limited services.
   3. GDSP. CDPH administers the GDSP, which screens newborns and pregnant women for genetic and congenital disorders. GDSP operates two primary screening programs: the Newborn Screening (NSB) program and the Prenatal Screening (PNS) program. Established in 1966, NSB provides screenings for every child born in California and includes screenings for more than 80 disorders. GDSP conducts screening, clinical, and surveillance activities statewide through public-private partnerships. The program contracts with five regional laboratories for initial screening activities and other private laboratories for confirmatory testing. GDSP also contracts with major medical centers, regional offices, and community organizations for diagnostic and follow-up services.
2. RDACs and the National Organization for Rare Disorders (NORD). According to NORD’s website, in 2015 the first RDAC was created in North Carolina by patients, caregivers, families, and providers. RDAC members typically include a variety of rare disease stakeholders, including patients, caregivers, health care providers, health insurers, biotech industry, researchers, patient advocacy organizations, and state government officials. NORD states that with over 10,000 known unique rare diseases, it is difficult for state government officials to have an in-depth understanding of the rare disease community’s needs, leading to a lack of awareness that contributes to the obstacles that rare disease patients and their loved ones face. RDACs can use their diverse membership and broad community support to identify barriers and propose solutions to help the rare disease community by giving stakeholders an opportunity to share experiences and steer discussions that develop solutions to the issues they face daily.

According to NORD, 28 states have a RDAC, and it is working to establish one in every state through Project RDAC. RDACs are organized and operated differently in each state. Some of the distinguishing features include the type of entity that houses the RDAC, the composition and size, and the duties and accountability requirements. Twenty-two states administer the RDAC through a government agency; four states through a university; one state through a nonprofit; and, one state through a grantee from its Department of Health.

1. Federal Orphan Drug Act (ODA). Under the ODA, the Federal Drug Administration (FDA) can grant special status to a drug or biological product to treat a rare disease or condition. This status, known as orphan designation, qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. The Office of Orphan Products Development (OOPD) administers the program that provides orphan drug status to drugs and biologics. According to a June 2023 white paper from the National Institute of Health’s National Library of Medicine, from 1983 to 2022, there were 1122 total approvals (including new molecule, indication, and formulation approvals) of orphan-designated products and 882 initial approvals (first approval falling under one orphan designation) representing 392 rare diseases.

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

According to the Senate Appropriations Committee, CHHSA anticipates delegating the responsibility to CDPH, which estimates annual General Fund costs of approximately $371,000 from 2024-25 through 2028-29, and additional General Fund costs of $278,000 in 2025-26, 2026-27, and 2027-28 for state administration to support the RDAC.

SUPPORT: (Verified 8/23/24)

National Organization for Rare Disorders (source)

Acadia Pharmaceuticals
Alliance for Patient Access
ALS Association

Biotechnology Innovation Organization
California Chronic Care Coalition
California Life Sciences
Cystic Fibrosis Research Institute
Hemophilia Council of California
International Foundation for Autoimmune & Autoinflammatory Arthritis

Liver Coalition of San Diego
Mellie J Foundation Corporation
National Marrow Donor Program

National PKU Alliance
Santa Monica Democratic Club
Travere Therapeutics

Three Individuals

OPPOSITION: (Verified 8/23/24)

None received

ARGUMENTS IN SUPPORT: Supporters of this bill state that rare disease patients in California face unique challenges every day, from obtaining an accurate diagnosis and accessing medical specialists with knowledge of their condition, to battling for fair insurance coverage of their treatment and care. Though the word “rare” implies that these diseases impact a small number of people, an estimated 7,000 rare diseases exist. While over 600 drugs and biologics have been approved for the treatment of rare diseases, according to the U.S. Food and Drug Administration, millions of Americans still have rare diseases for which there are no approved treatments. While the public is familiar with ALS and are sympathetic to those affected, many patients and families affected by less widely known rare diseases, bear a large share of the burden of funding research and raising public awareness to support the search for treatments. Supporters argue that the wide reach of rare diseases suggests that a greater voice in the policymaking process is necessary, as the unique needs and challenges of this community should be considered. In creating RDAC, California would join 28 other states (Alabama, Colorado, Connecticut, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Louisiana, Maine, Maryland, Massachusetts, Minnesota, Mississippi, Missouri, Nevada, New Hampshire, New Jersey, New York, North Carolina, Ohio, Pennsylvania, South Carolina, Tennessee, Utah, Virginia, and West Virginia) that have already enacted similar legislation in support of their rare disease communities.

ASSEMBLY FLOOR: 72-0, 5/22/24

AYES: Addis, Aguiar-Curry, Alanis, Alvarez, Arambula, Bains, Bauer-Kahan, Bennett, Berman, Boerner, Bonta, Bryan, Juan Carrillo, Wendy Carrillo, Chen, Connolly, Davies, Dixon, Flora, Mike Fong, Vince Fong, Gabriel, Gallagher, Garcia, Gipson, Grayson, Haney, Hart, Hoover, Irwin, Jackson, Jones-Sawyer, Kalra, Lackey, Lee, Low, Lowenthal, Maienschein, McCarty, McKinnor, Muratsuchi, Stephanie Nguyen, Ortega, Pacheco, Papan, Jim Patterson, Joe Patterson, Pellerin, Petrie-Norris, Quirk-Silva, Ramos, Rendon, Reyes, Rodriguez, Blanca Rubio, Sanchez, Santiago, Schiavo, Soria, Ta, Ting, Valencia, Villapudua, Waldron, Wallis, Ward, Weber, Wicks, Wilson, Wood, Zbur, Robert Rivas

NO VOTE RECORDED: Calderon, Cervantes, Megan Dahle, Essayli, Friedman, Holden, Mathis, Luz Rivas

Prepared by: Reyes Diaz / HEALTH / (916) 651-4111

8/26/24 11:53:17

**** END ****

 THIRD READING  

Bill No: AB 2613

Author: Zbur (D), et al.

Amended: 6/27/24 in Senate

Vote: 21

SENATE HEALTH COMMITTEE: 11-0, 6/26/24

AYES: Roth, Nguyen, Glazer, Gonzalez, Grove, Hurtado, Limón, Menjivar, Rubio, Smallwood-Cuevas, Wiener

SENATE APPROPRIATIONS COMMITTEE: 6-0, 8/15/24

AYES: Caballero, Jones, Ashby, Becker, Seyarto, Wahab

NO VOTE RECORDED: Bradford

ASSEMBLY FLOOR: 72-0, 5/22/24 - See last page for vote

SUBJECT: Jacqueline Marie Zbur Rare Disease Advisory Council

SOURCE: National Organization for Rare Disorders

DIGEST:

This bill establishes the Jacqueline Marie Zbur Rare Disease Advisory Council as an advisory body to advise and report to the California Health and Human Services Agency and the Legislature on strategies to address barriers and improve treatment services for those with rare diseases in this state.

ANALYSIS:

Existing law:

1. Establishes in state government the California Health and Human Services Agency (CHHSA) and within CHHSA, among other departments, offices and councils, the Department of Health Care Services (DHCS) and the California Department of Public Health (CDPH). [GOV §12800, 12803]
2. Establishes in state government the State Public Health Officer, who serves as the director of CDPH. [HSC §131005]
3. Establishes the California Children’s Services Program (CCS), administered by DHCS, under which individuals under the age of 21 who have specified health conditions and meet financial requirements are eligible to receive medically necessary services and treatments. [HSC §123800, et seq.]
4. Establishes the Genetically Handicapped Persons Program (GHPP), administered by DHCS, under which individuals who have specified genetic conditions are eligible to receive medical care and social support services. [HSC §125125, et seq.]
5. Establishes the Hereditary Disorders Act, under which CDPH administers the Genetic Disease Screening Program (GDSP), which provides a statewide program for newborn and prenatal testing for genetic disorders and tracking of birth defects. Requires GDSP to provide genetic screenings and follow up services for persons who have screenings. [HSC §124975, et seq.]

This bill:

1. Establishes, within CHHSA and until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC) as an advisory body.
2. Requires the Governor to appoint the RDAC chair by February 1, 2025.
3. Requires the State Public Health Officer to appoint 17 members by May 1, 2025, to serve without compensation (except for specified reimbursements), as follows:
   1. Two licensed physicians who have expertise in treating patients with rare diseases, at least one of whom treats children with rare diseases;
   2. One registered licensed nurse who has expertise in providing care to patients with rare diseases;
   3. One representative of a hospital located in this state and treats rare disease patients;
   4. One representative of an outpatient health care facility located in this state that treats rare disease patients;

One representative of the health care coverage industry;

• 1. One representative of the biopharmaceutical industry;
  2. One representative of the scientific community who is engaged in rare disease research;
  3. One rare disease geneticist or genetic counselor;
  4. Five individuals who are either a rare disease patient or a caregiver to a rare disease patient. Prohibits RDAC from including more than three individuals from each category;
  5. One medical social worker or mental health provider who works with rare disease patients; and,
  6. Two representatives of patient advocacy organizations that operate in this state.

1. Prohibits a single rare disease population to have more than two representatives on RDAC. Requires members appointed by the chair to serve on RDAC until January 1, 2029. Requires any vacancy to be filled in the same manner as provided for in the original appointment.
2. Requires each RDAC member to sign a conflict of interest statement annually disclosing any economic or other relationship with an entity that could influence the member’s decisions. Require at least 20% of RDAC’s members to not have a conflict of interest with respect to an insurer, pharmaceutical benefits manager, or pharmaceutical manufacturer.
3. Requires RDAC to perform all of the following duties:
   1. Act as the advisory body on rare diseases to the Legislature and state departments, agencies, commissions, and authorities, as well as private agencies, that provide services to, or that are charged with the care of, persons with rare diseases;
   2. Evaluate and make recommendations to improve Medi-Cal and state-regulated private health insurance coverage of drugs and biological products for rare disease patients;
   3. Engage with the Medi-Cal Drug Use Review Board (Medi-Cal DUR), and serve as an expert advisory committee, when Medi-Cal DUR makes recommendations or determinations regarding beneficiary access to drugs or biological products for rare diseases. Defines “beneficiary access” as developing prior authorization and reauthorization criteria for a rare disease drug, including placement on a preferred drug list or a formulary, as well as payment, cost sharing, drug utilization review, or medication therapy management;
   4. Prepare a first-year landscape survey of the needs of rare disease patients, caregivers, and providers in the state. Requires RDAC to convene public hearings, make inquiries, and solicit comments from the general public in the state to assist RDAC in fulfilling this duty;
   5. Consult with experts on rare diseases to develop policy recommendations to improve patient access to, and quality of, rare disease specialists, affordable and comprehensive health care coverage, relevant diagnostics, timely treatment, and other needed services;
   6. Create and maintain a website and identify, consolidate, and publish a list of publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment; and,
   7. Identify areas of unmet need for research and opportunities for collaboration with stakeholders and RDACs in other states that can inform future studies and work done by RDAC in this state.
4. Requires RDAC to hold an initial organizational meeting within 60 days after the date of the last appointment made by the chair and select a vice chair from among its members. Requires the chair to appoint a secretary who is an employee of CDPH or a member of RDAC.
5. Requires RDAC to meet periodically, but at least six times annually. Permits RDAC to call to its assistance, and avail itself of the services of, the employees of any state, county, or municipal department, board, bureau, commission, or agency as it may require and as may be available, if those employees elect to participate.
6. Requires RDAC to apply for, and accept, any grant of funds from the federal government, private foundations, or other sources that may be available for programs related to rare diseases. Prohibits RDAC from accepting any funds from the employer of a currently seated council member.
7. Requires RDAC to report to CHHSA and the Legislature its activities, findings, and recommendations on issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases through both an interim report submitted by July 1, 2026, and a final report submitted by December 31, 2028. Requires RDAC also to post the reports on its website.

Comments

1. Author’s statement. According to the author, “this bill creates RDAC in the memory of his sister, Jacqueline Marie Zbur, who endured a three-year battle with amyotrophic lateral sclerosis, (ALS, also known as Lou Gehrig’s disease). RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and guide the Legislature in supporting the rare disease community, which faces unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access to quality care and therapies, and obtaining fair insurance coverage for their treatment and care. This bill makes important progress toward addressing challenges for the rare disease community by fostering recommendations on, and understanding of, disease representation, specialist physician directories, financial impacts to patients and the state, and what private and public resources may be available to patients and their families. RDAC gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare disease. It is time for California to join 28 other states in adopting an RDAC, through which a diverse group of experts will be able to develop recommendations for the state on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.”
2. Rare diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the U.S. Altogether, rare diseases affect an estimated 30 million Americans. Rare diseases include life-threatening and physically or mentally disabling conditions, such as Huntington’s disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy. According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations, and many rare diseases have a long list of characteristics that present serious challenges for practitioners. Among rare diseases it is common to find that:
   1. Diagnoses are difficult and delayed;
   2. Case definitions for surveillance are usually lacking;
   3. International Classification of Diseases (ICD) codes for record keeping are poorly defined or not assigned;
   4. Underlying molecular or physiologic mechanisms are unknown;
   5. Specialized and coordinated medical care is in short supply, and treatments can be complex;
   6. Standards of care for treatment and rehabilitation are not evidence-based because health research is done at small scale;
   7. Longitudinal data collections are scarce;
   8. The development of new medications and treatments can be fragmented and slow;
   9. Screening strategies lack efficiency; and,
   10. The scope and capacity of most registries and databases are limited.

The knowledge of most rare diseases is so insufficient that they are also known as orphan diseases (and their treatments known as orphan drugs) because of their failure to attract the interest of researchers, medical specialists, drug makers, and policy makers.

1. State programs related to rare diseases. DHCS and CDPH operate state programs that provide services and screenings for individuals with certain diseases and health problems, which include some rare diseases. These programs include the CCS program, GHPP, and GDSP:
   1. CCS. CCS is a state-only health program for children, up to the age 21, with certain diseases or health problems, including bleeding disorders, cystic fibrosis, Duchenne muscular dystrophy, and other conditions. CCS requires applicants to meet additional requirements, such as income limits. CCS provides coverage for a variety of medical visits, medical case management, and care at sickle cell disease centers;
   2. GHPP. GHPP is a state-only health program that provides care for adults, aged 21 and older, with certain genetic diseases, including rare blood diseases, cystic fibrosis, diseases of brain, nerves and metabolism, and other diseases. There is no income limit; however, applicants may be required to pay an annual enrollment fee, which is based on income and family size. GHPP benefits include coverage for hospital stays, outpatient medical care, pharmaceutical services, surgeries, nutritional products, durable medical equipment, care at a Special Care Center, and other services. Individuals with private health insurance or Medicare coverage may apply for GHPP, but the program will only cover limited services.
   3. GDSP. CDPH administers the GDSP, which screens newborns and pregnant women for genetic and congenital disorders. GDSP operates two primary screening programs: the Newborn Screening (NSB) program and the Prenatal Screening (PNS) program. Established in 1966, NSB provides screenings for every child born in California and includes screenings for more than 80 disorders. GDSP conducts screening, clinical, and surveillance activities statewide through public-private partnerships. The program contracts with five regional laboratories for initial screening activities and other private laboratories for confirmatory testing. GDSP also contracts with major medical centers, regional offices, and community organizations for diagnostic and follow-up services.
2. RDACs and the National Organization for Rare Disorders (NORD). According to NORD’s website, in 2015 the first RDAC was created in North Carolina by patients, caregivers, families, and providers. RDAC members typically include a variety of rare disease stakeholders, including patients, caregivers, health care providers, health insurers, biotech industry, researchers, patient advocacy organizations, and state government officials. NORD states that with over 10,000 known unique rare diseases, it is difficult for state government officials to have an in-depth understanding of the rare disease community’s needs, leading to a lack of awareness that contributes to the obstacles that rare disease patients and their loved ones face. RDACs can use their diverse membership and broad community support to identify barriers and propose solutions to help the rare disease community by giving stakeholders an opportunity to share experiences and steer discussions that develop solutions to the issues they face daily.

According to NORD, 28 states have a RDAC, and it is working to establish one in every state through Project RDAC. RDACs are organized and operated differently in each state. Some of the distinguishing features include the type of entity that houses the RDAC, the composition and size, and the duties and accountability requirements. Twenty-two states administer the RDAC through a government agency; four states through a university; one state through a nonprofit; and, one state through a grantee from its Department of Health.

1. Federal Orphan Drug Act (ODA). Under the ODA, the Federal Drug Administration (FDA) can grant special status to a drug or biological product to treat a rare disease or condition. This status, known as orphan designation, qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. The Office of Orphan Products Development (OOPD) administers the program that provides orphan drug status to drugs and biologics. According to a June 2023 white paper from the National Institute of Health’s National Library of Medicine, from 1983 to 2022, there were 1122 total approvals (including new molecule, indication, and formulation approvals) of orphan-designated products and 882 initial approvals (first approval falling under one orphan designation) representing 392 rare diseases.

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

According to the Senate Appropriations Committee, CHHSA anticipates delegating the responsibility to CDPH, which estimates annual General Fund costs of approximately $371,000 from 2024-25 through 2028-29, and additional General Fund costs of $278,000 in 2025-26, 2026-27, and 2027-28 for state administration to support the RDAC.

SUPPORT: (Verified 8/14/24)

National Organization for Rare Disorders (source)

Acadia Pharmaceuticals
Alliance for Patient Access
ALS Association

Biotechnology Innovation Organization
California Chronic Care Coalition
California Life Sciences
Cystic Fibrosis Research Institute
Hemophilia Council of California
International Foundation for Autoimmune & Autoinflammatory Arthritis

Liver Coalition of San Diego
Mellie J Foundation Corporation
National Marrow Donor Program

National PKU Alliance
Santa Monica Democratic Club
Travere Therapeutics

Three Individuals

OPPOSITION: (Verified 8/14/24)

None received

ARGUMENTS IN SUPPORT: Supporters of this bill state that rare disease patients in California face unique challenges every day, from obtaining an accurate diagnosis and accessing medical specialists with knowledge of their condition, to battling for fair insurance coverage of their treatment and care. Though the word “rare” implies that these diseases impact a small number of people, an estimated 7,000 rare diseases exist. While over 600 drugs and biologics have been approved for the treatment of rare diseases, according to the U.S. Food and Drug Administration, millions of Americans still have rare diseases for which there are no approved treatments. While the public is familiar with ALS and are sympathetic to those affected, many patients and families affected by less widely known rare diseases, bear a large share of the burden of funding research and raising public awareness to support the search for treatments. Supporters argue that the wide reach of rare diseases suggests that a greater voice in the policymaking process is necessary, as the unique needs and challenges of this community should be considered. In creating RDAC, California would join 28 other states (Alabama, Colorado, Connecticut, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Louisiana, Maine, Maryland, Massachusetts, Minnesota, Mississippi, Missouri, Nevada, New Hampshire, New Jersey, New York, North Carolina, Ohio, Pennsylvania, South Carolina, Tennessee, Utah, Virginia, and West Virginia) that have already enacted similar legislation in support of their rare disease communities.

ASSEMBLY FLOOR: 72-0, 5/22/24

AYES: Addis, Aguiar-Curry, Alanis, Alvarez, Arambula, Bains, Bauer-Kahan, Bennett, Berman, Boerner, Bonta, Bryan, Juan Carrillo, Wendy Carrillo, Chen, Connolly, Davies, Dixon, Flora, Mike Fong, Vince Fong, Gabriel, Gallagher, Garcia, Gipson, Grayson, Haney, Hart, Hoover, Irwin, Jackson, Jones-Sawyer, Kalra, Lackey, Lee, Low, Lowenthal, Maienschein, McCarty, McKinnor, Muratsuchi, Stephanie Nguyen, Ortega, Pacheco, Papan, Jim Patterson, Joe Patterson, Pellerin, Petrie-Norris, Quirk-Silva, Ramos, Rendon, Reyes, Rodriguez, Blanca Rubio, Sanchez, Santiago, Schiavo, Soria, Ta, Ting, Valencia, Villapudua, Waldron, Wallis, Ward, Weber, Wicks, Wilson, Wood, Zbur, Robert Rivas

NO VOTE RECORDED: Calderon, Cervantes, Megan Dahle, Essayli, Friedman, Holden, Mathis, Luz Rivas

Prepared by: Reyes Diaz / HEALTH / (916) 651-4111

8/18/24 9:19:20

**** END ****

SENATE COMMITTEE ON APPROPRIATIONS

Senator Anna Caballero, Chair

2023 - 2024 Regular Session

AB 2613 (Zbur) - Jacqueline Marie Zbur Rare Disease Advisory Council

Bill Summary:

AB 2613 would establish the Jacqueline Marie Zbur Rare Disease Advisory Council within the California Health and Human Services Agency (CHHS).

Fiscal Impact:

The CHHS anticipates delegating the responsibility to the California Department of Public Health (CDPH). The CDPH estimates annual General Fund costs of approximately $371,000 from 2024-25 through 2028-29, and additional General Fund costs of $278,000 in 2025-26, 2026-27, and 2027-28 for state administration to support the council.

Background:

According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the U.S., and there are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans. Rare diseases include life-threatening and physically or mentally disabling conditions, such as Huntington’s disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy. According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations, and many rare diseases have a long list of characteristics that present serious challenges for practitioners. Among rare diseases it is common to find that:

• Diagnoses are difficult and delayed;
• Case definitions for surveillance are usually lacking;
• International Classification of Diseases (ICD) codes for record keeping are poorly defined or not assigned;
• Underlying molecular or physiologic mechanisms are unknown;
• Specialized and coordinated medical care is in short supply, and treatments can be complex;
• Standards of care for treatment and rehabilitation are not evidence-based because health research is done at small scale;
• Longitudinal data collections are scarce;
• The development of new medications and treatments can be fragmented and slow;
• Screening strategies lack efficiency; and,
• The scope and capacity of most registries and databases are limited.

The knowledge of most rare diseases is so insufficient that they are also known as orphan diseases (and their treatments known as orphan drugs) because of their failure to attract the interest of researchers, medical specialists, drug makers, and policy makers.

Proposed Law:

Specific provisions of the bill would:

• Establish, within the CHHS, the Jacqueline Marie Zbur Rare Disease Advisory Council.
• Require the Governor to appoint the chair of the advisory council, and require the membership of the advisory council to be appointed by the State Public Health Officer, as specified.
• Specify the duties of the advisory council and require the advisory council to meet periodically, but at least six times annually.
• Require the advisory council to apply for, and accept, any grant of funds from the federal government, private foundations, or other sources that may be available for programs related to rare diseases.
• Require the advisory council to report to the CHHS and the Legislature on the activities of the advisory council and its findings and recommendations on issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases in this state, as specified.
• Sunset the bill’s provisions on January 1, 2029.

Related Legislation:

SB 247 (Eggman, 2021) was similar to this bill. The bill was vetoed by the Governor.

-- END --

SENATE COMMITTEE ON HEALTH

Senator Richard Roth, Chair

BILL NO: AB 2613

SUBJECT: Jacqueline Marie Zbur Rare Disease Advisory Council

SUMMARY:

Establishes the Jacqueline Marie Zbur Rare Disease Advisory Council as an advisory body to advise and report to the California Health and Human Services Agency and the Legislature on strategies to address barriers and improve treatment services for those with rare diseases in this state.

Existing law:

1. Establishes in state government the California Health and Human Services Agency (CHHSA) and within CHHSA, among other departments, offices and councils, the Department of Health Care Services (DHCS) and the California Department of Public Health (CDPH). [GOV §12800, 12803]
2. Establishes in state government the State Public Health Officer, who serves as the director of CDPH. [HSC §131005]
3. Establishes the California Children’s Services Program (CCS), administered by DHCS, under which individuals under the age of 21 who have specified health conditions and meet financial requirements are eligible to receive medically necessary services and treatments. [HSC §123800, et seq.]
4. Establishes the Genetically Handicapped Persons Program (GHPP), administered by DHCS, under which individuals who have specified genetic conditions are eligible to receive medical care and social support services. [HSC §125125, et seq.]
5. Establishes the Hereditary Disorders Act, under which CDPH administers the Genetic Disease Screening Program (GDSP), which provides a statewide program for newborn and prenatal testing for genetic disorders and tracking of birth defects. Requires GDSP to provide genetic screenings and follow up services for persons who have screenings. [HSC §124975, et seq.]

This bill:

1. Establishes, within CHHSA and until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC) as an advisory body.
2. Requires the Governor to appoint the RDAC chair by February 1, 2025. Requires the chair to appoint 17 members by May 1, 2025, to serve without compensation (except for specified reimbursements), as follows:
   1. Two licensed physicians who have expertise in treating patients with rare diseases, at least one of whom treats children with rare diseases;
   2. One registered licensed nurse who has expertise in providing care to patients with rare diseases;
   3. One representative of a hospital located in this state and treats rare disease patients;
   4. One representative of an outpatient health care facility located in this state that treats rare disease patients;
   5. One representative of the health care coverage industry;
   6. One representative of the biopharmaceutical industry;
   7. One representative of the scientific community who is engaged in rare disease research;
   8. One rare disease geneticist or genetic counselor;
   9. Five individuals who are either a rare disease patient or a caregiver to a rare disease patient. Prohibits RDAC from including more than three individuals from each category;
   10. One medical social worker or mental health provider who works with rare disease patients; and,
   11. Two representatives of patient advocacy organizations that operate in this state.
3. Prohibits a single rare disease population to have more than two representatives on RDAC. Requires members appointed by the chair to serve on RDAC until January 1, 2029. Requires any vacancy to be filled in the same manner as provided for in the original appointment.
4. Requires each RDAC member to sign a conflict of interest statement annually disclosing any economic or other relationship with an entity that could influence the member’s decisions. Require at least 20% of RDAC’s members to not have a conflict of interest with respect to an insurer, pharmaceutical benefits manager, or pharmaceutical manufacturer.
5. Requires RDAC to perform all of the following duties:
   1. Act as the advisory body on rare diseases to the Legislature and state departments, agencies, commissions, and authorities, as well as private agencies, that provide services to, or that are charged with the care of, persons with rare diseases;
   2. Evaluate and make recommendations to improve Medi-Cal and state-regulated private health insurance coverage of drugs and biological products for rare disease patients;
   3. Engage with the Medi-Cal Drug Use Review Board (Medi-Cal DUR), and serve as an expert advisory committee, when Medi-Cal DUR makes recommendations or determinations regarding beneficiary access to drugs or biological products for rare diseases. Defines “beneficiary access” as developing prior authorization and reauthorization criteria for a rare disease drug, including placement on a preferred drug list or a formulary, as well as payment, cost sharing, drug utilization review, or medication therapy management;
   4. Prepare a first-year landscape survey of the needs of rare disease patients, caregivers, and providers in the state. Requires RDAC to convene public hearings, make inquiries, and solicit comments from the general public in the state to assist RDAC in fulfilling this duty;
   5. Consult with experts on rare diseases to develop policy recommendations to improve patient access to, and quality of, rare disease specialists, affordable and comprehensive health care coverage, relevant diagnostics, timely treatment, and other needed services;
   6. Create and maintain a website and identify, consolidate, and publish a list of publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment; and,
   7. Identify areas of unmet need for research and opportunities for collaboration with stakeholders and RDACs in other states that can inform future studies and work done by RDAC in this state.
6. Requires RDAC to hold an initial organizational meeting within 60 days after the date of the last appointment made by the chair and select a vice chair from among its members. Requires the chair to appoint a secretary who is an employee of CDPH or a member of RDAC.
7. Requires RDAC to meet periodically, but at least six times annually. Permits RDAC to call to its assistance, and avail itself of the services of, the employees of any state, county, or municipal department, board, bureau, commission, or agency as it may require and as may be available, if those employees elect to participate.
8. Requires RDAC to apply for, and accept, any grant of funds from the federal government, private foundations, or other sources that may be available for programs related to rare diseases. Prohibits RDAC from accepting any funds from the employer of a currently seated council member.
9. Requires RDAC to report to CHHSA and the Legislature its activities, findings, and recommendations on issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases through both an interim report submitted by July 1, 2026, and a final report submitted by December 31, 2028. Requires RDAC also to post the reports on its website.

FISCAL EFFECT:

According to the Assembly Appropriations Committee, CDPH estimates annual costs of approximately $650,000 from January 1, 2025, through January 1, 2029, for a total of approximately $2.6 million (General Fund). CDPH anticipates the need for three full-time, limited-term staff positions to coordinate and oversee the administrative processes and activities of RDAC, and funds needed for members' travel and per diem. According to the Legislative Analyst's Office, the General Fund faces a structural deficit in the tens of billions of dollars over the next several fiscal years.

PRIOR VOTES:

COMMENTS:

1. Author’s statement. According to the author, this bill creates RDAC in the memory of his sister, Jacqueline Marie Zbur, who endured a three-year battle with amyotrophic lateral sclerosis, (ALS, also known as Lou Gehrig’s disease). RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and guide the Legislature in supporting the rare disease community, which faces unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access to quality care and therapies, and obtaining fair insurance coverage for their treatment and care. This bill makes important progress toward addressing challenges for the rare disease community by fostering recommendations on, and understanding of, disease representation, specialist physician directories, financial impacts to patients and the state, and what private and public resources may be available to patients and their families. RDAC gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare disease. It is time for California to join 28 other states in adopting an RDAC, through which a diverse group of experts will be able to develop recommendations for the state on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.
2. Rare diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the U.S. Altogether, rare diseases affect an estimated 30 million Americans. Rare diseases include life-threatening and physically or mentally disabling conditions, such as Huntington’s disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy. According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations, and many rare diseases have a long list of characteristics that present serious challenges for practitioners. Among rare diseases it is common to find that:
   1. Diagnoses are difficult and delayed;
   2. Case definitions for surveillance are usually lacking;
   3. International Classification of Diseases (ICD) codes for record keeping are poorly defined or not assigned;
   4. Underlying molecular or physiologic mechanisms are unknown;
   5. Specialized and coordinated medical care is in short supply, and treatments can be complex;
   6. Standards of care for treatment and rehabilitation are not evidence-based because health research is done at small scale;
   7. Longitudinal data collections are scarce;
   8. The development of new medications and treatments can be fragmented and slow;
   9. Screening strategies lack efficiency; and,
   10. The scope and capacity of most registries and databases are limited.

The knowledge of most rare diseases is so insufficient that they are also known as orphan diseases (and their treatments known as orphan drugs) because of their failure to attract the interest of researchers, medical specialists, drug makers, and policy makers.

1. State programs related to rare diseases. DHCS and CDPH operate state programs that provide services and screenings for individuals with certain diseases and health problems, which include some rare diseases. These programs include the CCS program, GHPP, and GDSP:
   1. CCS. CCS is a state-only health program for children, up to the age 21, with certain diseases or health problems, including bleeding disorders, cystic fibrosis, Duchenne muscular dystrophy, and other conditions. CCS requires applicants to meet additional requirements, such as income limits. CCS provides coverage for a variety of medical visits, medical case management, and care at sickle cell disease centers;
   2. GHPP. GHPP is a state-only health program that provides care for adults, aged 21 and older, with certain genetic diseases, including rare blood diseases, cystic fibrosis, diseases of brain, nerves and metabolism, and other diseases. There is no income limit; however, applicants may be required to pay an annual enrollment fee, which is based on income and family size. GHPP benefits include coverage for hospital stays, outpatient medical care, pharmaceutical services, surgeries, nutritional products, durable medical equipment, care at a Special Care Center, and other services. Individuals with private health insurance or Medicare coverage may apply for GHPP, but the program will only cover limited services.
   3. GDSP. CDPH administers the GDSP, which screens newborns and pregnant women for genetic and congenital disorders. GDSP operates two primary screening programs: the Newborn Screening (NSB) program and the Prenatal Screening (PNS) program. Established in 1966, NSB provides screenings for every child born in California and includes screenings for more than 80 disorders. GDSP conducts screening, clinical, and surveillance activities statewide through public-private partnerships. The program contracts with five regional laboratories for initial screening activities and other private laboratories for confirmatory testing. GDSP also contracts with major medical centers, regional offices, and community organizations for diagnostic and follow-up services.
2. RDACs and the National Organization for Rare Disorders (NORD). According to NORD’s website, in 2015 the first RDAC was created in North Carolina by patients, caregivers, families, and providers. RDAC members typically include a variety of rare disease stakeholders, including patients, caregivers, health care providers, health insurers, biotech industry, researchers, patient advocacy organizations, and state government officials. NORD states that with over 10,000 known unique rare diseases, it is difficult for state government officials to have an in-depth understanding of the rare disease community’s needs, leading to a lack of awareness that contributes to the obstacles that rare disease patients and their loved ones face. RDACs can use their diverse membership and broad community support to identify barriers and propose solutions to help the rare disease community by giving stakeholders an opportunity to share experiences and steer discussions that develop solutions to the issues they face daily.

According to NORD, 28 states have a RDAC, and it is working to establish one in every state through Project RDAC. RDACs are organized and operated differently in each state. Some of the distinguishing features include the type of entity that houses the RDAC, the composition and size, and the duties and accountability requirements. Twenty-two states administer the RDAC through a government agency; four states through a university; one state through a nonprofit; and, one state through a grantee from its Department of Health.

1. Federal Orphan Drug Act (ODA). Under the ODA, the Federal Drug Administration (FDA) can grant special status to a drug or biological product to treat a rare disease or condition. This status, known as orphan designation, qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. The Office of Orphan Products Development (OOPD) administers the program that provides orphan drug status to drugs and biologics. According to a June 2023 white paper from the National Institute of Health’s National Library of Medicine, from 1983 to 2022, there were 1122 total approvals (including new molecule, indication, and formulation approvals) of orphan-designated products and 882 initial approvals (first approval falling under one orphan designation) representing 392 rare diseases.
2. Prior legislation. SB 247 (Eggman of 2021), AB 2283 (Eggman of 2020), and AB 1016 (Maienschein of 2019) would have established the RDAC within CHHSA. SB 247 was vetoed by Governor Newsom who stated: while it is important for the public, providers, state agencies, and private partners to have access to information on rare diseases, the purpose and duties of the RDAC in this bill are extensive and costly. Bills with a significant fiscal impact, such as this measure, should be considered in the annual budget process. For this reason, I cannot sign this bill. AB 2283 was not heard by the Assembly Health Committee. AB 1016 was held on the Assembly Appropriations Committee suspense file.
3. Support. Supporters of this bill state that rare disease patients in California face unique challenges every day, from obtaining an accurate diagnosis and accessing medical specialists with knowledge of their condition, to battling for fair insurance coverage of their treatment and care. Though the word “rare” implies that these diseases impact a small number of people, an estimated 7,000 rare diseases exist. While over 600 drugs and biologics have been approved for the treatment of rare diseases, according to the U.S. Food and Drug Administration, millions of Americans still have rare diseases for which there are no approved treatments. While the public is familiar with ALS and are sympathetic to those affected, many patients and families affected by less widely known rare diseases, bear a large share of the burden of funding research and raising public awareness to support the search for treatments. Supporters argue that the wide reach of rare diseases suggests that a greater voice in the policymaking process is necessary, as the unique needs and challenges of this community should be considered. In creating RDAC, California would join 28 other states (Alabama, Colorado, Connecticut, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Louisiana, Maine, Maryland, Massachusetts, Minnesota, Mississippi, Missouri, Nevada, New Hampshire, New Jersey, New York, North Carolina, Ohio, Pennsylvania, South Carolina, Tennessee, Utah, Virginia, and West Virginia) that have already enacted similar legislation in support of their rare disease communities.
4. Amendments. SB 247 required the State Public Health Officer to appoint the public members of the RDAC, not an unspecified Governor’s appointee. The author may wish to amend this bill to align with that requirement.

SUPPORT AND OPPOSITION:

Support: National Organization for Rare Disorders (sponsor)

Acadia Pharmaceuticals
Alliance for Patient Access
ALS Association
California Chronic Care Coalition
California Life Sciences
Cystic Fibrosis Research Institute
Hemophilia Council of California
International Foundation for Autoimmune & Autoinflammatory Arthritis
Mellie J Foundation Corporation
National Marrow Donor Program

National PKU Alliance
Santa Monica Democratic Club
Travere Therapeutics

Three Individuals

Oppose: None received

-- END --

ASSEMBLY THIRD READING

ATR Bill Id:AB 2613

Author:(Zbur)

As Amended Ver:April 25, 2024

Majority vote

SUMMARY

Establishes within the California Health and Human Services Agency, until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC), composed of a minimum of 17 members. Requires the advisory council to prepare a landscape survey of the needs of rare disease patients, caregivers, and providers; make recommendations to improve access to treatments in Medi-Cal and private health coverage; apply for and accept grants; and report its activities and findings to the Legislature, among other duties.

Major Provisions

COMMENTS

Background on Rare Diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the United States. There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans. Examples of rare diseases include life-threatening and physically or mentally disabling conditions such as ALS; Huntington disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy.

According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations and many rare diseases have a long list of characteristics that present serious challenges for public health practitioners. Among rare diseases it is common to find that: 1) diagnoses are difficult and delayed; 2) case definitions for surveillance are usually lacking; 3) International Classification of Diseases codes for record keeping are poorly defined or not assigned; 4) underlying molecular or physiologic mechanisms are unknown; 5) specialized and coordinated medical care is in short supply, and treatments can be complex; g standards of care for treatment and rehabilitation are not evidence-based because health research is necessarily done at small scale; 6) longitudinal data collections are scarce; 7) the development of new medications and treatments can be fragmented and slow; 8) screening strategies lack efficiency; and, 9) scope and capacity of most registries and databases are limited.

According to the Author

This bill will create a RDAC in California. The author's sister, Jacqueline Marie Zbur, endured a three-year battle with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. The author states that the RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and will guide the Legislature in supporting the rare disease community. The author notes that rare disease patients face unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access for quality care and therapies, and obtaining fair insurance coverage of their treatment and care. The author contends that this bill makes important progress towards addressing challenges for the rare disease community by fostering recommendations on – and understanding of – disease representation, specialist physician directories, financial impacts to patients and the State, and what private and public resources may be available to patients and their families. The author also notes that this bill gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare diseases. The author contends that it is time for California to join 27 other states in adopting a RDAC, through which a diverse group of experts will be able to develop recommendations for the State on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.

Arguments in Support

A coalition of organizations representing individuals with rare diseases including NARD, the California Center for Rare Diseases at the University of California, Las Angeles, Hemophilia Council of California, and the California Chronic Care Coalition state, for patients living with one of the over 10,000 known rare conditions, it can take several years to receive an accurate diagnosis and effective treatment. Further, only a handful of rare diseases are well understood, with most not receiving sufficient attention or funding for research. This lack of awareness often contributes to the obstacles to timely treatment and care faced by many rare disease patients. The coalition states that creating an RDAC in California will give rare disease patients a unified voice in state government to help address challenges that are faced by the rare disease community by serving as the advisory body on rare diseases to the Legislature and state departments, and relieve some of the burden on the state by expeditiously delivering direct feedback, solutions, and resources to California.

Arguments in Opposition

None on file.

FISCAL COMMENTS

According to the Assembly Appropriations Committee, the Department of Public Health (DPH) estimates annual costs of approximately $650,000 from January 1, 2025, through January 1, 2029, for a total of approximately $2.6 million (General Fund). DPH anticipates the need for three full-time, limited-term staff positions to coordinate and oversee the administrative processes and activities of the council, and funds needed for council members' travel and per diem. According to the Legislative Analyst's Office, the General Fund faces a structural deficit in the tens of billions of dollars over the next several fiscal years. 

VOTES

ASM HEALTH: 16-0-0

YES: Bonta, Waldron, Aguiar-Curry, Arambula, Wendy Carrillo, Flora, Haney, Jones-Sawyer, Maienschein, McCarty, Joe Patterson, Rodriguez, Sanchez, Santiago, Schiavo, Weber

ASM APPROPRIATIONS: 15-0-0

YES: Wicks, Sanchez, Arambula, Bryan, Calderon, Wendy Carrillo, Dixon, Mike Fong, Grayson, Haney, Hart, Jim Patterson, Pellerin, Ta, Villapudua

UPDATED

VERSION: April 25, 2024

CONSULTANT: Eliza Brooks / HEALTH / (916) 319-2097 FN: 0003253

Date of Hearing: May 8, 2024

ASSEMBLY COMMITTEE ON APPROPRIATIONS

Buffy Wicks, Chair

ABPCA Bill Id:AB 2613

Author:(Zbur) – As Amended Ver:April 25, 2024

Urgency: No State Mandated Local Program: No Reimbursable: No

SUMMARY:

This bill establishes within the California Health and Human Services Agency (CalHHS), until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (advisory council), composed of a minimum of 17 members representing various specified health professions, diseases, patients, caregivers, and industries.

The bill requires the advisory council to prepare a landscape survey of the needs of rare disease patients, caregivers, and providers; make recommendations to improve access to treatments in Medi-Cal and private health coverage; apply for and accept grants; and report its activities and findings to the Legislature, among other duties.

FISCAL EFFECT:

The California Department of Public Health (CDPH) estimates annual costs of approximately $650,000 from January 1, 2025, through January 1, 2029, for a total of approximately $2.6 million (General Fund). CDPH anticipates the need for three full-time, limited-term staff positions to coordinate and oversee the administrative processes and activities of the council, and funds needed for council members’ travel and per diem.

According to the Legislative Analyst’s Office, the General Fund faces a structural deficit in the tens of billions of dollars over the next several fiscal years. 

COMMENTS:

1. Purpose. This bill is sponsored by the National Organization for Rare Disorders. According to the author:

Created in the memory of my sister, Jacqueline Marie Zbur, who endured a three-year battle with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease, the RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and will guide the Legislature in supporting the rare disease community. Rare disease patients face unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access for quality care and therapies, and obtaining fair insurance coverage of their treatment and care. AB 2613 makes important progress towards addressing challenges for the rare disease community by fostering recommendations on – and understanding of – disease representation, specialist physician directories, financial impacts to patients and the State, and what private and public resources may be available to patients and their families across the State. The RDAC gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare disease. It is time for California to join 27 other states in adopting an RDAC, through which a diverse group of experts will be able to develop recommendations for the State on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.

1. Background. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the United States. There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans.

According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, a rare disease is likely to present serious challenges for public health practitioners for many reasons. Among these challenges are: (a) diagnoses are difficult and delayed; (b) case definitions for surveillance are usually lacking; (c) International Classification of Diseases codes for record keeping are poorly defined or not assigned; (d) underlying molecular or physiologic mechanisms are unknown; (e) specialized and coordinated medical care is in short supply, and treatments can be complex; (f) standards of care for treatment and rehabilitation are not evidence-based because health research is necessarily done at small scale; (g) longitudinal data collections are scarce; (h) the development of new medications and treatments can be fragmented and slow; (i) screening strategies lack efficiency; and, (j) scope and capacity of most registries and databases are limited.

1. Budget Request. The author has submitted to the Assembly Committee on Budget a request for $275,000 per year for three years, for a total of $825,000.

4) Prior Legislation.

• 1. SB 247 (Eggman), of the 2021-22 Legislative Session, would have established the RDAC within the California Health and Human Services Agency. SB 247 was vetoed by the Governor, who cited costs and stated such bills should be considered in the annual budget process.
     
  2. AB 2283 (Eggman), of the 2019-20 Legislative Session, was identical to SB 247 and was referred to, but not heard by, the Assembly Health Committee.
     
  3. AB 1016 (Maienschein), of the 2019-20 Legislative Session, would have established an RDAC. AB 1016 was held in this committee.
     

Analysis Prepared by: Allegra Kim / APPR. / (916) 319-2081

Date of Hearing: April 23, 2024

ASSEMBLY COMMITTEE ON HEALTH

Mia Bonta, Chair

ABPCA Bill Id:AB 2613

Author:(Zbur) – As Introduced Ver:February 14, 2024

SUBJECT: Jaqueline Marie Zbur Rare Disease Advisory Council.

SUMMARY: Establishes within the California Health and Human Services Agency (CHHSA), until January 1, 2029, the Jacqueline Marie Zbur Rare Disease Advisory Council (advisory council), composed of a minimum of 17 members. Specifically, this bill:

1. Establishes the advisory council, a Rare Disease Advisory Council (RDAC), within CHHSA.
   
2. Requires the Governor, on or before February 1, 2025, to appoint the first chair of the advisory council.
   
3. Requires the chair of the advisory council, on or before January 1, 2025, to appoint the minimum membership of the advisory council composed of the following 17 members:
   1. Two physicians and surgeons licensed to practice in this state who have expertise in treating patients with rare diseases, at least one of whom shall also treat children with rare diseases;
   2. One registered professional nurse licensed to practice in this state who has expertise in providing care to patients with rare diseases;
   3. One representative of the administration of a hospital located in this state and treats rare disease patients;
   4. One representative of the administration of an outpatient health care facility located in this state and treats rare disease patients;
   5. One representative of the health care coverage industry;
   6. One representative of the biopharmaceutical industry;
   7. One representative of the scientific community who is engaged in rare disease research;
   8. One rare disease geneticist or genetic counselor;
   9. Five individuals who are either a rare disease patient or a caregiver to a rare disease
      patient. Prohibits more than three individuals from each category;
   10. One medical social worker or mental health provider who works with rare disease patients; and,
   11. Two representatives of patient advocacy organizations that operate within this state.
       
4. Permits the advisory council to advise the CHHSA on additional at-large appointments to the advisory council that may be necessary to carry out its duties. Authorizes at-large appointments to the advisory council to serve on an ad hoc basis.
   
5. Requires all members of the advisory council to serve without compensation, but allows the members appointed pursuant to 2) above to be reimbursed for travel and other miscellaneous expenses necessary to perform their duties within the limits of funds made available to the advisory council for its purposes.
   
6. Prohibits a single rare disease population from having more than two representatives on the advisory council.
   
7. Requires members of the advisory council appointed pursuant to 2) above to serve January 1, 2029. Requires any vacancy in the membership of the advisory council to be filled in the same manner as provided for in the original appointment.
   
8. Requires each member of the advisory council to annually sign a conflict of interest statement disclosing any economic or other relationship with an entity that could influence the member’s decisions. Prohibits at least 20% of the advisory council’s members from having a conflict of interest with respect to an insurer, pharmaceutical benefits manager, or pharmaceutical manufacturer above.
   
9. Requires the advisory council to hold an initial organizational meeting within 60 days after the date of the last appointment made pursuant to 2).
   
10. Requires, at the initial organizational meeting, the advisory council to select its vice chair from among its members.
    
11. Requires, at the initial organizational meeting, the chair to appoint a secretary at their discretion.
    
12. Authorizes the chair to appoint a secretary who is also a member of the advisory council or an employee of the DPH.
    
13. Requires the advisory council to meet periodically, but at least six times annually.
    
14. Authorizes the advisory council to call to its assistance, and avail itself of the services of, the employees of any state, county, or municipal department, board, bureau, commission, or agency as it requires and as are available to it for its purposes, if those employees elect to participate.
    
15. Requires the advisory council to perform all of the following duties:
    1. Act as the advisory body on rare diseases to the Legislature, and state departments, agencies, commissions, and authorities, and private agencies, that provide services to, or that are charged with the care of, persons with rare diseases;
    2. Evaluate and make recommendations to improve Medi-Cal and state-regulated private health insurance coverage of drugs and biological products for rare disease patients;
    3. Engage with the Medi-Cal Drug Use Review Board, also known as Medi-Cal DUR, and serve as an expert advisory committee to the board, when the board makes recommendations or determinations regarding beneficiary access to drugs or biological products for rare diseases;
    4. Defines for purposes of this bill, “beneficiary access” means developing prior authorization and reauthorization criteria for a rare disease drug, including placement on a preferred drug list or a formulary, as well as payment, cost sharing, drug utilization review, or medication therapy management;
    5. Prepare a first-year landscape survey of the needs of rare disease patients, caregivers, and providers in the state. Requires the advisory council to convene public hearings, make inquiries, and solicit comments from the general public in the state to assist the advisory council in fulfilling this duty;
    6. Consult with experts on rare diseases to develop policy recommendations to improve patient access to, and quality of, rare disease specialists, affordable and comprehensive health care coverage, relevant diagnostics, timely treatment, and other needed services;
    7. Create and maintain an internet website;
    8. Identify, consolidate, and publish on the advisory council’s internet website a list of existing, publicly accessible resources on research, diagnosis, treatment, and education relating to rare diseases in California to foster recognition and access to treatment; and,
    9. Identify areas of unmet need for research and opportunities for collaboration with stakeholders and rare disease advisory councils in other states that can inform future studies and work done by the advisory council.
       
16. Requires the advisory council to apply for, and accept, any grant of funds from the federal government, private foundations, or other sources that are available for programs related to rare diseases.
    
17. Prohibits the advisory council from accepting any funds from the employer of a currently seated council member.
    
18. Requires the advisory council to report to the CHHSA and the Legislature on the activities of the advisory council and its findings and recommendations on issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases in this state, as follows:
    1. An interim report, submitted on or before July 1, 2026; and,
    2. A final report, submitted on or before December 31, 2028.
       
19. Requires the advisory council to submit reports to the Legislature in compliance with existing law.
    
20. Requires the advisory council to post the reports required by this bill on its internet website.
    
21. Repeals the provisions of this bill on January 1, 2029.

EXISTING LAW:

1. Establishes in state government the CHHSA and within CHHSA, among other departments, offices and councils, the Department of Health Care Services (DHCS) and the California Department of Public Health (DPH). [Government Code §12800, 12803]
2. Establishes in state government the State Public Health Officer, who serves as the director of DPH. [Health & Safety Code (HSC) §131005]

FISCAL EFFECT: Unknown. This bill has not yet been analyzed by a fiscal committee.

COMMENTS:

1. PURPOSE OF THIS BILL. According to the author, this bill will create a RDAC in California. The author’s sister, Jacqueline Marie Zbur, endured a three-year battle with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease. The author states that the RDAC will serve as a vital platform to amplify the voices of millions of Californians grappling with rare diseases and will guide the Legislature in supporting the rare disease community. The author notes that rare disease patients face unique challenges every day, including obtaining an accurate diagnosis, accessing medical specialists with knowledge of their condition, battling for access for quality care and therapies, and obtaining fair insurance coverage of their treatment and care. The author contends that this bill makes important progress towards addressing challenges for the rare disease community by fostering recommendations on – and understanding of – disease representation, specialist physician directories, financial impacts to patients and the State, and what private and public resources may be available to patients and their families. The author also notes that this bill gives rare disease patients a much needed voice to advocate and strategically plan for the future of rare diseases. The author contends that it is time for California to join 27 other states in adopting a RDAC, through which a diverse group of experts will be able to develop recommendations for the State on how best to address the challenges faced by the nearly three million Californians with rare diseases, their families, and their caregivers.
2. BACKGROUND.
   1. Background on Rare Diseases. According to the National Human Genome Institute, a rare disease is a condition that affects fewer than 200,000 people in the United States. There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans. Examples of rare diseases include life-threatening and physically or mentally disabling conditions such as ALS; Huntington disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn disease, cystic fibrosis, and Duchenne muscular dystrophy.

According to a 2016 essay published in Preventing Chronic Disease: Public Health Research, Practice, and Policy, rare disease patients are few and scattered across populations and many rare diseases have a long list of characteristics that present serious challenges for public health practitioners. Among rare diseases it is common to find that: i) diagnoses are difficult and delayed; ii) case definitions for surveillance are usually lacking; iii) International Classification of Diseases codes for record keeping are poorly defined or not assigned; iv) underlying molecular or physiologic mechanisms are unknown; v) specialized and coordinated medical care is in short supply, and treatments can be complex; g standards of care for treatment and rehabilitation are not evidence-based because health research is necessarily done at small scale; vi) longitudinal data collections are scarce; vii) the development of new medications and treatments can be fragmented and slow; viii) screening strategies lack efficiency; and, ix) scope and capacity of most registries and databases are limited.

The knowledge of most rare diseases is so insufficient that they are also known as orphan diseases (and their treatments known as orphan drugs) because of their failure to attract the interest of researchers, medical specialists, drug makers, and policy makers. The essay recommends five goals for a comprehensive public health approach to rare diseases and its potential impact on affected populations. The goals include: i) defining and examining the impact of the disease; ii) developing common surveillance practices; iii) improving knowledge about the disease; iv) support health care systems in treating the diseases; and, v) reducing the impact of the diseases on patients, their relatives and caregivers, and society in general.

• 1. Office of Rare Diseases. In 2002, Congress enacted the Rare Diseases Act (RDA) which established within the Office of Rare Diseases within National Institutes of Health (NIH) to recommend an agenda for conducting and supporting research on rare diseases through the national research institutes and centers. Under the RDA, the NIH can enter into cooperative agreements with and make grants for regional centers of excellence on rare diseases.
  2. State Programs Addressing Rare Diseases. DHCS and DPH operate state programs that provide services and screenings for individuals with certain diseases and health problems, which include some rare diseases. These programs include the California Children’s Services Program (CCS), Genetically Handicapped Persons Program (GHPP), and Genetic Disease Screening Program (GDSP).
  3. RDAC. This bill creates the advisory council which will be tasked with: i) evaluating and making recommendations to improve Medi-Cal and state-regulated private insurance coverage of medications that treat rare disease, as well as engaging the Medi-Cal DUR Board; ii) preparing a survey of the rare disease landscape in California, and convening public hearings to assist the advisory council in fulfilling its duties; iii) consulting with experts in rare diseases to develop policy recommendations; iv) creating and maintaining a website for the advisory council; v) identifying and publishing a list of resources on said website; and, vi) identifying areas of unmet need for research and opportunities for collaboration with stakeholders and RDACs in other states that can inform future studies and work done by the advisory council to increase awareness of rare diseases and provide a forum for stakeholders knowledgeable about the needs of rare disease patients to help shape policies to improve health outcomes for rare disease patients. There have been several legislative attempts in State of California to create a RDAC.

In 2015, the first RDAC was created in North Carolina by patients, caregivers, families and providers. Since then, an additional 26 states have established RDACs. With the support of the National Association for Rare Disorders (NARD), the sponsor of this bill, and other patient organizations and stakeholders in the rare disease community, RDACs work to strategically identify and address barriers that prevent individuals living with rare diseases from obtaining proper treatment and care for their condition. In 2023, three states signed RDAC legislation into law.

1. SUPPORT. A coalition of organizations representing individuals with rare diseases including NARD, the California Center for Rare Diseases at the University of California, Las Angeles, Hemophilia Council of California, and the California Chronic Care Coalition state, for patients living with one of the over 10,000 known rare conditions, it can take several years to receive an accurate diagnosis and effective treatment. Further, only a handful of rare diseases are well understood, with most not receiving sufficient attention or funding for research. This lack of awareness often contributes to the obstacles to timely treatment and care faced by many rare disease patients. The coalition states that creating an RDAC in California will give rare disease patients a unified voice in state government to help address challenges that are faced by the rare disease community by serving as the advisory body on rare diseases to the Legislature and state departments, and relieve some of the burden on the state by expeditiously delivering direct feedback, solutions, and resources to California.
   
   
2. RELATED LEGISLATION. AB 2680 (Aguiar-Curry) of 2024 renames the Alzheimer’s Disease and Related Disorders Committee to the Alzheimer’s Disease and Related Conditions Advisory Committee, expands the number of members serving on the committee from 14 to at least 21, but not more than 25 and prescribes the qualifications of certain members on the committee. AB 2680 passed the Assembly Health Committee 16-0.
3. PREVIOUS LEGISLATION.
   1. SB 247 (Eggman) of 2021 would have established the RDAC within the California Health and Human Services Agency and would have specified the purposes of the RDAC, including coordinating statewide efforts for the study of the incidence of rare diseases within the state, and acting as the advisory body on rare diseases to the Legislature and state and private agencies that provide services to persons with rare diseases, adopting implementing regulations, and researching and determining the most appropriate method to collect data on rare diseases, and identifying best practices for rare disease care. SB 247 was vetoed by the Governor, who stated, in part: “While it is important for the public, providers, state agencies, and private partners to have access to information on rare diseases, the purpose and duties of the Advisory Council as would be required by SB 247 are extensive and costly. Bills with a significant fiscal impact, such as this measure, should be considered in the annual budget process. For this reason, I cannot sign this bill”.
      
   2. AB 2283 (Eggman) of 2020 was identical to SB 247. AB 2283 was not heard by the Assembly Health Committee.
      
   3. AB 1016 (Maienschein) of 2019 would have established a RDAC. AB 1016 was held on the Assembly Appropriations suspense file.
      
   4. ACR 28 (Gipson) of 2019 would have recognized September 2020 as Sickle Cell Disease Awareness Month and encouraged the Legislature to appropriate funds for research, treatment, monitoring, education, and outreach related to the disease. ACR 28 was not heard by the Senate Health Committee.
      
4. SUGGESTED AMENDMENT. To clarify language related to the appointment of the secretary of the RDAC, the author may wish to amend this bill as follows:

124965.4. The advisory council shall hold an initial organizational meeting within 60 days after the date of the last appointment made pursuant to subdivision (c) of Section 124965.2. At that initial organizational meeting, the advisory council shall select its vice chair from among its members, and the chair shall appoint, at their discretion, a secretary. The chair may appoint, but is not required to appoint, a secretary who is also a member of the advisory council or an employee of the State Department of Public Health. The secretary shall be an employee of the Department or a member of the advisory council.

REGISTERED SUPPORT / OPPOSITION:

Support

Acadia Pharmaceuticals

ALS Association
Axis Advocacy
CA Action Link for Rare Diseases
California Chronic Care Coalition

California Coalition for PKU and Allied Disorders
California Hepatitis C Task Force
California Life Sciences
Chronic Care Policy Alliance
Cystic Fibrosis Research Institute
Dravet Syndrome Foundation
Epilepsy Foundation of San Diego County
Fair Time for Women
Family Voices of Ca
Hemophilia Council of California
Liver Coalition of San Diego
Lupus Foundation of Southern California
National Organization for Rare Disorders
National Organization for Rare Disorders (NORD)
National Pku Alliance
Neuropathy Action Foundation
NMDP
Parkinson's Association of San Diego
Rasopathies Network
Santa Monica Democratic Club
TSC Alliance

Opposition

None on file.

Analysis Prepared by: Eliza Brooks / HEALTH / (916) 319-2097