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ARIZONA STATE SENATE
Fifty-Seventh Legislature, First Regular Session
rare disease advisory council
Purpose
Establishes the Rare Disease Advisory Council (RDAC) within the Department of Health Services (DHS) to provide guidance and recommendations on the needs of individuals living with a rare disease. Outlines RDAC membership, powers and responsibilities.
Background
DHS is established to promote and protect the health of residents and provide and coordinate public health services and programs for Arizona. Responsibilities of DHS include but are not limited to: 1) promoting the development, maintenance, efficiency and effectiveness of local health departments or districts; 2) collecting and tabulating all information required in reference to births; 3) conducting a statewide health education program; 4) coordinating local programs concerning control of preventable diseases, maternal and child health, nutrition and dental health; 5) establishing and maintaining laboratories; 6) conducting continual evaluations of state, local and district public health programs; and 7) licensing and regulating health care institutions (A.R.S. ยง 36-132).
A rare disease is a disease, disorder, illness or condition that affects fewer than 200,000 Americans. There are over 10,000 known rare diseases that altogether affect more than 30,000,000 Americans. Rare diseases may include genetic disorders, metabolic disorders, neurological disorders, infectious diseases, and bone and tissue disorders. RDACs have been established in 30 states with the intent of helping states strategically identify and address barriers to adequate and effective treatment for individuals living with a rare disease (National Organization for Rare Disorders).
If there is a cost associated with establishing and operating an RDAC within DHS, there may be a fiscal impact to the state General Fund.
Provisions
1. Establishes the RDAC in DHS to provide guidance and recommendations to educate the public, the Legislature and other government agencies and departments, as appropriate, on the needs of individuals with a rare disease who live in Arizona, consisting of the following members appointed by the Governor:
a) one person who represents an Arizona academic research institution that receives grant funding for rate disease research;
b) one representative of DHS;
c) one representative of the Arizona Health Care Cost Containment System (AHCCCS);
d) one representative from the Department of Insurance and Financial Institutions;
e) one registered nurse or advanced practice registered nurse with experience treating rare diseases who is licensed and practicing in Arizona;
f) two physicians practicing in Arizona who have experience treating patients with rare diseases, one of whom has experience treating pediatric populations;
g) one geneticist or genetic counselor;
h) one hospital administrator, or the administrator's designee, from an Arizona hospital that provides care to persons with rare diseases;
i) at least one patient with a rare disease;
j) at least one caregiver of a person with a rare disease;
k) one person representing an Arizona rare disease patient organization;
l) one pharmacist with experience dispensing drugs used to treat rare diseases;
m) one representative of the biopharma industry;
n) one representative of a health insurer;
o) one member of the scientific community who is a medical researcher with experience conducting research on rare diseases; and
p) one mental health provider with experience treating patients with rare diseases in Arizona.
2. Requires the RDAC appointment process to be conducted transparently to provide interested individuals an opportunity to apply for membership.
3. Requires all RDAC members to be full-time residents of the state, if practicable.
4. Requires RDAC membership to include a diverse set of stakeholders who represent the geographic and population diversity of Arizona.
5. Requires the initial meeting of the RDAC to occur within 90 days after the effective date and to continue meeting at least once per month.
6. Allows the RDAC to meet in person or via an online meeting platform.
7. Requires the RDAC to provide opportunities for the public to hear updates on its work and provide input.
8. Requires the RDAC to develop and maintain a public website for posting meeting minutes and notices and accepting public comments.
9. Specifies that RDAC members serve three-year terms and are not eligible to receive compensation but are eligible for reimbursement of expenses.
10. Allows the RDAC to conduct the following activities to benefit those impacted by rare diseases in Arizona:
a) convene
public hearings, make inquiries and solicit public comments to assist with a
first-year landscape or survey of the unmet needs of rare disease patients,
caregivers and providers;
b) provide testimony and comments on pending legislation and rules that impact the Arizona rare disease community;
c) consult with experts on rare diseases to develop policy recommendations that improve patient access to, and quality of, rare disease specialists, affordable and comprehensive health care coverage, relevant diagnostics, timely treatment and other needed services;
d) research and make recommendations to state agencies and health insurers that provide services to persons with rare diseases regarding the impact of orphan drug pricing, prior authorization, cost-sharing or other barriers to providing treatment and care for patients;
e) evaluate and make recommendations to improve AHCCCS and state-regulated private health insurance coverage of drugs for rare disease patients, including engaging with the Pharmacy and Therapeutics Committee, to improve coverage of diagnostics and facilitate access to necessary health care providers with expertise in treating rare diseases; and
f) identify and distribute educational resources for health care providers to foster recognition and optimize treatment of rare diseases.
11. Requires the RDAC, by December 1 of each year, to submit a report to the Governor, Chairpersons and ranking members of the Health and Human Services Committees of the Senate and House of Representatives, or their successor committees, that:
a) describes the activities and progress of the RDAC; and
b) provides recommendations to the Governor and the Legislature on ways to address the needs of people living with rare diseases in Arizona.
12. Requires, before submission, a draft of the RDAC annual report to be made available for public comment and discussed at an open public meeting.
13. Allows the RDAC to solicit gifts, grants and donations for operations, activities and initiatives.
14. Establishes the initial terms of RDAC membership as follows:
a) five terms ending January 1, 2028;
b) six terms ending January 1, 2029; and
c) six terms ending January 1, 2030.
15. Requires, after the initial terms of membership, the Governor to make all subsequent appointments as prescribed.
16. Contains a statement of legislative findings.
17. Becomes effective on the general effective date.
House Action
HHS 2/3/25 DP 12-0-0-0
GOV 2/20/25 DP 5-1-1-0
3rd Read 2/26/25 46-12-2
Prepared by Senate Research
March 10, 2025
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