2025 -- H 5023

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LC000507

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     STATE OF RHODE ISLAND

IN GENERAL ASSEMBLY

JANUARY SESSION, A.D. 2025

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A N   A C T

RELATING TO HEALTH AND SAFETY -- RARE DISEASE ADVISORY COUNCIL

     Introduced By: Representatives Kennedy, Spears, Diaz, Donovan, Cotter, Carson,
Azzinaro, Kazarian, Edwards, and Serpa

     Date Introduced: January 10, 2025

     Referred To: House Health & Human Services

     It is enacted by the General Assembly as follows:

     SECTION 1. Legislative findings.

     The general assembly hereby finds and declares:

     (1) A rare disease, sometimes called an orphan disease, is defined as a disease that affects

fewer than 200,000 people in the United States.

     (2) There are more than 7,000 known rare diseases affecting approximately 25-30 million

Americans, more than half of which are children.

     (3) More than 90% of rare diseases do not have a Food and Drug Administration (FDA)

approved treatment.

     (4) While the exact cause for many rare diseases remains unknown, many rare diseases are

genetic in origin and can be linked to mutations in a single gene or in multiple genes, which can be

passed down from generation to generation.

     (5) People with rare diseases face many challenges, including delays in obtaining an

accurate diagnosis, finding a health care provider with expertise in their condition, and a lack of

affordable access to therapies and medication used to treat rare diseases.

     (6) A state-based advisory council composed of qualified professionals and persons living

with rare diseases and their caregivers could educate medical professionals, government agencies,

legislators, and the public about rare diseases as an important public health issue and encourage

research into the development of new ways to diagnose and treat rare diseases.

     SECTION 2. Title 23 of the General Laws entitled "HEALTH AND SAFETY" is hereby

amended by adding thereto the following chapter:

CHAPTER 87.1

RARE DISEASE ADVISORY COUNCIL

     23-87.1-1. Short title.

     This chapter shall be known and may be cited as the "Rare Disease Advisory Council."

     23-87.1-2. Establishment.

     (a) There is established in the department of health the office of rare disease advisory

council.

     (b) The purpose of the council is to provide guidance and recommendations to educate the

public, legislature, as well as other government agencies, on the needs of individuals with rare

diseases living in Rhode Island.

     (c) The council shall conduct the following activities to benefit those impacted by rare

diseases in Rhode Island. The duties of the council shall include:

     (1) Convene public hearings, make inquiries, and solicit comments from the general public

in Rhode Island to assist the council with a first-year landscape or survey of the needs of rare

disease patients, caregivers, and providers in the state.

     (2) Provide testimony and comments on pending legislation and regulations before the

legislature and other state agencies that impact Rhode Island's rare disease community.

     (3) 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.

     (4) Research and make recommendations to state agencies and insurers that provide

services to persons with a rare disease on the impact of prior authorization, cost-sharing, tiering, or

other utilization management procedures on the provision of treatment and care for patients.

     (5) Establish best practices and protocols to include in-state planning related to natural

disasters, public health emergencies or other emergency declarations to enable continuity of care

for rare disease patients and ensure safeguards against discrimination for rare disease patients are

in place.

     (6) Evaluate and make recommendations to implement improvements to newborn infant

screening programs.

     (7) Evaluate and make recommendations to improve Medicaid coverage of drugs for rare

disease patients, including engagement with the drug utilization review board, to improve coverage

of diagnostics, and facilitate access to necessary healthcare providers with expertise in the treatment

of rare diseases.

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     (8) Publish a list of existing, publicly accessible resources on research, diagnosis,

treatment, and education relating to the rare diseases on the council's website. For purposes of this

chapter, "rare disease" or "orphan disease" means a disease that affects fewer than two hundred

thousand (200,000) people in the United States.

     (9) Identify areas of unmet need for research and opportunities for collaboration across

stakeholders that can inform future studies and reports by the council.

     (10) Identify and distribute educational resources for health care providers to foster

recognition and optimize treatment of rare diseases.

     (11) Research and identify best practices to reduce health disparities and achieve health

equity in the research, diagnosis and treatment of rare diseases.

     (12) Research and identify best practices to ensure continuity of care for rare disease

patients transitioning from child/youth services to adult care.

     23-87.1-3. Membership of council.

     (a) All members of the council shall be full-time residents of Rhode Island and membership

shall include a diverse set of stakeholders.

     (b) The governor shall appoint all members and shall designate a chair of the council within

thirty (30) days of enactment. The chair shall not hold any position within the government of Rhode

Island and shall serve for a term of three (3) years.

     (c) The governor shall appoint the following members:

     (1) One representative from academic research institutions in the state that receives any

grant funding for rare disease research;

     (2) One representative from the Rhode Island department of health's office of minority

health;

     (3) One representative from the Rhode Island Medicaid agency;

     (4) One representative from the Rhode Island department of business regulation insurance

division;

     (5) One registered nurse or advanced practice registered nurse licensed and practicing in

Rhode Island with experience treating rare diseases;

     (6) One physician practicing in Rhode Island with experience treating rare diseases;

     (7) One hospital administrator, or designee, from a hospital in Rhode Island that provides

care to persons diagnosed with a rare disease;

     (8) At least two (2) patients who have a rare disease;

     (9) At least one caregiver of a person with a rare disease;

     (10) One representative of a rare disease patient organization that operates in Rhode Island;

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     (11) A pharmacist with experience dispensing drugs used to treat rare diseases;

     (12) A representative of the biopharma industry;

     (13) A representative of a health plan company; and

     (14) A member of the scientific community who is engaged in rare disease research,

including, but not limited to, a medical researcher with experience conducting research on rare

diseases.

     23-87.1-4. Terms and vacancies for council members.

     (a) Term of members. Council members shall serve no longer than three (3) years, except

that, to facilitate a staggered rotation of members to retain continuity and knowledge transfer,

during the initial five (5) years after the establishment of the council, members may serve up to a

four (4) year term.

     (b) Vacancy. If a vacancy occurs, the vacancy shall be filled in a like manner as required

pursuant to this section.

     23-87.1-5. Reporting requirements.

     (a) The council shall submit a report to the governor, the speaker of the house and the

president of the senate, and the chairs of the house and senate health and finance committees within

one year of enactment of the council and annually thereafter. Prior to submission, a draft of the

annual report shall be made available for public comment and discussed at an open public meeting.

     (b) Annual reports shall:

     (1) Describe the activities and progress of the council under this section;

     (2) Provide an update on the status of funding of the council; and

     (3) Provide recommendations to the governor and legislature on ways to address the needs

of people living with rare diseases in the state.

     23-87.1-6. Funding for council.

     (a) The council members may solicit funds on behalf of the council.

     (b) The council shall establish a restricted receipt account and all funds designated or raised

by or for the advisory council shall be placed in the restricted receipt account to support the

activities of the council necessary to carry out the provisions of this chapter.

     (c) The council shall report funding sources in the annual reports outlined in § 23-87.1-

5(b).

     23-87.1-7. Meeting requirements.

     (a) Frequency.

     (1) The initial meeting of the council shall occur within the first ninety (90) days after

enactment.

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     (2) During the first twelve (12) months after the initial meeting, the council shall meet each

month.

     (3) Thereafter, the council shall meet once per quarter in person or via online meeting

platform as determined by the chair.

     (b) Notice.

     (1) The council shall, in compliance with chapter 46 of title 42 ("open meetings"):

     (i) Provide opportunities for the public to hear updates and provide input into their work;

and

     (ii) Create and maintain a public website where meeting minutes, notices of upcoming

meetings, and public comments can be submitted.

     SECTION 3. This act shall take effect upon passage.

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EXPLANATION

BY THE LEGISLATIVE COUNCIL

OF

A N   A C T

RELATING TO HEALTH AND SAFETY -- RARE DISEASE ADVISORY COUNCIL

***

     This act would establish a rare disease advisory council within the department of health.

The council would provide guidance and recommendations to educate health care providers and

the citizens of the state.

     This act would take effect upon passage.

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