A2528

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. Such diseases are referred to as genetic diseases. Genetic disease can be passed down from generation to generation, explaining why certain rare diseases run in families. It is also estimated that about half of all rare diseases affect children;

c. Environmental factors such as diet, smoking, or exposure to chemicals also play a part in causing rare diseases. These factors may directly cause, or interact with, genetic factors that increase the severity of rare disease;

d. Rare diseases can be chronic, progressive, debilitating, disabling, severe, and life threatening. A person suffering with a rare disease faces a wide range of challenges, including, but not limited to: delays in obtaining a diagnosis; being misdiagnosed; a shortage of medical specialists who are familiar with, and can provide treatment for, rare diseases; the prohibitive cost of treatment; and the inability to access therapies and medication that are used by doctors to treat rare diseases but have not been approved by the Federal Food and Drug Administration for that specific purpose;

e. In recent years, researchers have made considerable progress in developing diagnostic tools and treatment protocols for, and in discovering ways to prevent a variety of, rare diseases. However, much more remains to be done in the areas of rare disease research and the search for new therapeutics; and

f. It is therefore an appropriate public policy for the State of New Jersey to establish an advisory body, whose membership would be comprised of persons with suitable qualifications for this purpose, to examine the issues that affect persons with rare diseases, to educate medical professionals, government agencies, and the public about 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.

a. The advisory council shall consist of 16 members as follows:

(1) the Commissioners of Health, Human Services, Children and Families, and Environmental Protection, or their designees, as ex officio members; and

(2) twelve 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 patient advocate employed by a general hospital in this State; two representatives of general hospitals in this State, one of whom shall represent a religiously-affiliated hospital; a representative of the New Jersey Office on Minority and Multicultural Health; a representative of the health insurance industry, a representative of the pharmaceutical industry; a representative of the scientific community who is engaged in rare disease research; a parent of a child with a rare disease; and a person with a rare disease.

b. Vacancies in the membership of the advisory council shall be filled in the same manner provided for the original appointments. 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 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 may meet at the call of its chair and 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. assist the Commissioner of Health in establishing and implementing a rare disease registry program to serve as a single repository of information on all rare diseases diagnosed in this State;

c. 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, and to develop policy recommendations on those issues;

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

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

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 of each year, 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 to, 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

This bill establishes the 16-member New Jersey Rare Disease Advisory Council in the Department of Health.

The membership of the advisory council will consist of: the Commissioners of Health, Human Services, Children and Families, and Environmental Protection, or their designees, as ex officio members; and twelve public members to be appointed by the Governor, including two physicians licensed to practice in this State who have expertise in treating patients with rare diseases, one of whom is a pediatrician providing 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; two representatives of general hospitals in this State, one of whom represents a religiously-affiliated hospital; a patient advocate employed by a general hospital in this State; a representative of the New Jersey Office on Minority and Multicultural Health, the health insurance industry, the pharmaceutical industry, and the scientific community who is engaged in rare disease research; a parent of a child with a rare disease; and a person 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; assist the Commissioner of Health in establishing and implementing a rare disease registry program in this State; conduct a thorough and comprehensive study of all issues relating to the quality and cost-effectiveness of, and access to treatment and services provided for, persons with rare diseases in this State, and to develop policy recommendations on those issues; identify effective research-based strategies that have been developed to help diagnose, treat, and prevent rare diseases; and develop effective strategies to raise public awareness of rare diseases in this State.

Finally, the bill requires the advisory council to annually report to the Governor and the Legislature on 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.

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.