A4016

An Act establishing the New Jersey Rare Disease Advisory Council and supplementing Title 26 of the Revised Statutes.

Be It Enacted by the Senate and General Assembly of the State of New Jersey:

1. The Legislature finds and declares:

a. A rare disease is defined as a disease that affects fewer than 20,000 people. Rare diseases are sometimes called orphan diseases. There are 7,000 rare diseases affecting approximately 25 to 30 million Americans;

b. The exact cause for many rare diseases remains unknown. However, 80% of 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;

c. A person suffering with a rare disease faces a wide range of challenges, including delays in obtaining a diagnosis; being misdiagnosed; shortages of medical specialists who can provide treatment for rare diseases; and the lack of therapies and medication that are used by doctors to treat rare diseases;

d. There is a significant link between rare disease and the coronavirus 2019 (COVID-19) pandemic which heightens the importance of the establishment of an advisory council to examine the issues that affect persons with rare diseases in the State. Many people who live with rare diseases are immunosuppressed and have respiratory and neurologic issues that make the consequences of the virus much more severe for them;

e. Additionally, those with rare diseases rely on the health care system much more than those who do not have chronic and rare conditions. In as much as they go to their physicians’ offices, medical testing sites, and hospitals more often than most, their risk of exposure to the COVID-19 virus is much greater;

f. People who live with rare diseases are impacted by potential shortages of life-saving medications and supplies due to changes in supply chain resulting from the COVID-19 pandemic. People with rare diseases also rely on the support of their families and home health aides, and because of the social distancing and quarantining, the pandemic puts them at risk of not having the support they need in accessing services and treatment;

g. Researchers have made considerable progress in developing diagnostic tools and treatment protocols for rare diseases and discovering methods of prevention. However, much more remains to be done in the search for new therapeutics and in understanding the link between rare disease and pandemics, and

h. It is therefore an appropriate public policy for the State of New Jersey to establish an advisory body, whose membership would be comprised of qualified professionals and persons living with rare diseases, that would be tasked to educate medical professionals, government agencies, and the public about the importance of rare diseases as an important public health issue, and to encourage and fund research in the development of new treatments for rare diseases.

2. There is established the New Jersey Rare Disease Advisory Council in the Department of Health which shall advise the Legislature, State departments, agencies, commissions, and authorities, and private agencies providing services for persons diagnosed with a rare disease.

a. The advisory council shall consist of 30 members as follows:

(1) the Commissioners of Banking and Insurance, Children and Families, Environmental Protection, Health, Human Services, and the Executive Director of the New Jersey Office on Minority and Multicultural Health, or their designees, as ex officio members;

(2) two members of the Senate, one of whom is appointed by the President of the Senate, and one of whom is appointed by the Minority Leader of the Senate;

(3) two members of the General Assembly, one of whom is appointed by the Speaker of the General Assembly, and one of whom is appointed by the Minority Leader of the General Assembly; and

(4) 20 public members to be appointed by the Governor, who shall include: two physicians licensed to practice in this State who have expertise in treating patients with rare diseases, one of whom shall be a pediatrician who provides care to children with rare diseases; a registered professional nurse licensed in this State who has expertise in providing care to patients with rare diseases; a representative of general hospital or hospital system in this State recommended by the New Jersey Hospital Association; a representative of federally qualified health center in this State recommended by the New Jersey Primary Care Association; a geneticist licensed to practice in this State; a genetic counselor who has experience in providing services to persons diagnosed with a rare disease, their families, or their caregivers or care partners; a representative of the health insurance industry recommended by the New Jersey Association of Health Plans; a pharmacist licensed to practice in this State who has experience with persons with a rare disease; a representative of the pharmaceutical industry who has expertise in rare diseases recommended by the HealthCare Institute of New Jersey; a representative of the biotechnology industry who is has expertise in rare diseases recommended by BIO NJ; a representative of the medical technology industry who has expertise in rare diseases; a representative of the Rutgers Biomedical and Health Sciences who is engaged in rare disease research; a representative of the Rowan University Graduate School of Biomedical Sciences who is engaged in rare disease research; two representatives of rare disease patient advocacy organizations; two persons, age 18 years or older, who have a rare disease; and two caregivers or care partners for a patient or partner with a rare disease; one of who shall be a caregiver for a child with a rare disease.

b. Public members of the advisory council shall serve for a term of three years, except that of the initial appointments, seven public members shall serve for one year, seven public members shall serve for two years, and six public members shall serve for three years. Vacancies in the membership of the council shall be filled in the same manner as the original appointments were made. The public members of the council shall serve without compensation but may be reimbursed for traveling and other miscellaneous expenses necessary to perform their duties within the limits of funds made available to the council for its purposes.

c. The advisory council shall organize as soon as practicable after the appointment of its members, and the Governor shall select a chairperson and vice-chairperson from among its members. The chairperson shall appoint a secretary who need not be a member of the council.

d. The advisory council shall meet a minimum of three times a year but may meet more often at the call of its chair. The council may hold hearings at the times and in the places it deems appropriate and necessary to fulfill its charge. The council shall be entitled 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 to it for its purposes.

e. The Department of Health shall provide staff services to the advisory council.

3. The purpose of the advisory council shall be to:

a. act as the advisory body on rare diseases to the Legislature and State departments, agencies, commissions, authorities, and private agencies that provide services to, or are charged with the care of, persons with rare diseases;

b. conduct a thorough and comprehensive study of all issues relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases in this State, including the link between rare diseases and the COVID-19 pandemic, and to develop policy recommendations on those issues;

c establish and implement a repository of best practice standards to share with health care providers that will ensure they are adequately informed of the most effective strategies for recognizing and treating rare diseases in New Jersey;

d. identify effective research-based strategies that have been developed to help diagnose, treat, and prevent rare diseases;

e. develop effective strategies to raise public awareness of rare diseases in this State;

f. evaluate and make recommendations to improve:

(1) the State’s Newborn Screening Program in the Department of Health; and

(2) State Medicaid coverage for approved treatments and medications for patients with a rare disease;

g. research and make policy recommendations to the Legislature on access to health insurance specialists and other needed services for patients with a rare disease; and

h. identify, with assistance from the public, additional research topics on rare disease to inform future studies the council may conduct.

4. The advisory council shall apply for, and accept, any grant of money from the federal government, private foundations, or other sources, which may be available for programs related to rare diseases.

5. The advisory council shall report to the Governor and, pursuant to section 2 of P.L.1991, c.164 (C.52:14-19.1), to the Legislature, no later than December 31st, on a biennial basis, starting in the second year next following the enactment of this act, on the activities of the advisory council and its findings and recommendations on issues relating to the quality of, and access to, treatment and services for persons with rare diseases in this State.

6. The Commissioner of Health, pursuant to the "Administrative Procedures Act," P.L.1968, c.410 (C.52:14B-1 et seq.) shall adopt rules and regulations necessary to effectuate the purposes of this act.

7. This act shall take effect immediately.

STATEMENT

There is a significant link between rare disease and the coronavirus 2019 (COVID-19) pandemic. Many people who live with rare diseases are immunosuppressed and have respiratory and neurologic issues that make the consequences of the virus much more severe for them. This link heightens the importance of the establishment of an advisory council to examine the issues that affect persons with rare diseases in the State.

Therefore, this bill establishes the 30-member New Jersey Rare Disease Advisory Council in the Department of Health to advise the Legislature, State departments, agencies, commissions, and authorities, and private agencies providing services for persons diagnosed with a rare disease.

The membership of the advisory council will consist of: the Commissioners of Banking and Insurance, Children and Families, Environmental Protection, Health, Human Services, and the Executive Director of the New Jersey Office on Minority and Multicultural Health or their designees, as ex officio members; two members of the Senate, one appointed by the President of the Senate, and one appointed by the Minority Leader of the Senate; two members of the General Assembly, one appointed by the Speaker of the General Assembly, and one appointed by the Minority Leader of the General Assembly; and 20 public members appointed by the Governor, including two physicians licensed to practice in this State who have expertise in treating patients with rare diseases, one who would be a pediatrician who provides care to children with rare diseases; a registered professional nurse licensed in this State who has expertise in providing care to patients with rare diseases; a representative of general hospital or hospital system in this State recommended by the New Jersey Hospital Association; a representative of federally qualified health center in this State recommended by the New Jersey Primary Care Association; a geneticist licensed to practice in this State; a genetic counselor who has experience in providing services to persons diagnosed with a rare disease, their families, or their caregivers or care partners; a representative of the health insurance industry recommended by the New Jersey Association of Health Plans; a pharmacist licensed to practice in this State who has experience with persons with a rare disease; a representative of the pharmaceutical industry who has expertise in rare diseases recommended by the HealthCare Institute of New Jersey; a representative of the biotechnology industry who has expertise in rare diseases recommended by BIO NJ; a representative of the medical technology industry who has expertise in rare diseases; a representative of the Rutgers Biomedical and Health Sciences who is engaged in rare disease research; a representative of the Rowan University Graduate School of Biomedical Sciences who is engaged in rare disease research; two representatives of rare disease patient advocacy organizations; two persons, age 18 years or older, who have a rare disease; and two caregivers or care partners for a patient or partner with a rare disease; one who would be a caregiver for a child with a rare disease.

The purpose of the advisory council will be to: act as the advisory body on rare diseases to the Legislature and State departments, agencies, commissions, authorities, and private agencies that provide services to, or are charged with the care of, persons with rare diseases; conduct a thorough and comprehensive study of all issues relating to the quality of, and access to, treatment and services provided to persons with rare diseases in this State, including the link between rare diseases and the COVID-19 pandemic, and to develop policy recommendations on those issues; establish and implement a repository of best practice to share with health care providers that will ensure they are adequately informed of the most effective strategies for recognizing and treating rare diseases in New Jersey; identify effective research-based strategies that have been developed to help diagnose, treat, and prevent rare diseases; develop effective strategies to raise public awareness of rare diseases in this State; evaluate and make recommendations to improve the State’s Newborn Screening Program in the Department of Health and State Medicaid coverage for approved treatments and medications for patients with a rare disease; research and make policy recommendations to the Legislature on access to health insurance specialists and other needed services for patients with a rare disease; and identify, with assistance from the public, additional research topics on rare disease to inform future studies the council may conduct.

Finally, the bill requires the advisory council to report biennially, to the Governor and the Legislature on its findings and recommendations on issues relating to the quality of, and access to, treatment and services provided to persons with rare diseases in this State.

This bill is similar to H-7094, the "Rare Disease Community Support, Resource Coordination and Quality of Life Act of 2014," that was adopted by the state of Rhode Island earlier this year.