Senate Bill 2463

(COMMITTEE SUBSTITUTE)

AN ACT TO ESTABLISH A MISSISSIPPI RARE DISEASE ADVISORY COUNCIL WITHIN THE STATE DEPARTMENT OF HEALTH TO EDUCATE MEDICAL PROFESSIONALS, GOVERNMENTAL AGENCIES AND THE PUBLIC ABOUT RARE DISEASES AND TO FUND RESEARCH IN THE DEVELOPMENT OF TREATMENTS FOR RARE DISEASES; TO PROVIDE FOR THE MEMBERSHIP OF THE COUNCIL AND PRESCRIBE ITS DUTIES AND RESPONSIBILITIES; TO PROVIDE THAT THE COUNCIL SHALL APPLY FOR AND EXPEND GRANTS FROM THE FEDERAL GOVERNMENT OR PRIVATE SOURCES FOR FUNDING THE ACTIVITIES OF THE COUNCIL; AND FOR RELATED PURPOSES.

BE IT ENACTED BY THE LEGISLATURE OF THE STATE OF MISSISSIPPI:

SECTION 1. Findings. The Legislature finds and declares:

(a) A rare disease is defined as a disease that affects fewer than two hundred thousand (200,000) people in the United States. Rare diseases are sometimes called orphan diseases. There are seven thousand (7,000) known rare diseases affecting approximately thirty million (30,000,000) men, women and children in the United States;

(b) The exact cause for many rare diseases remains unknown. However, eighty percent (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) A person suffering with a rare disease in Mississippi faces a wide range of challenges, including, but not limited to: delays in obtaining a diagnosis; misdiagnosis; shortage of medical specialists familiar with, and can provide treatment for, rare diseases; 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 (FDA) for that specific purpose;

(d) 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 and development of new therapeutics; and

(e) It is, therefore, an appropriate public policy for Mississippi to establish an advisory body, whose membership would be comprised of persons with suitable qualifications for this purpose, including persons living 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.

SECTION 2. Establishment of a Rare Disease Advisory Council. (1) There is established the Mississippi Rare Disease Advisory Council (council) in the Mississippi Department of Health.

(2) The advisory council shall consist of members as follows:

(a) Employees of the Mississippi Department of Health and other state agencies concerned with the provision of care to persons with rare diseases appointed by the Executive Officer of the State Department of Health; and

(b) Public members to be appointed by the Executive Director of the State Department of Health, who shall include:

(i) Two (2) physicians licensed to practice in Mississippi who have expertise in treating patients with rare diseases, one of whom shall be a pediatrician who provides care to children with rare diseases;

(ii) A registered professional nurse licensed in Mississippi who has expertise in providing care to patients with rare diseases;

(iii) Two (2) representatives of hospitals in Mississippi;

(iv) A representative of the health insurance industry;

(v) A representative of the biopharmaceutical industry;

(vi) A representative of the scientific community who is engaged in rare disease research;

(vii) Two (2) parents of a child with a rare disease;

(viii) Two (2) persons with a rare disease;

(ix) Two (2) patient organizations that operate within Mississippi.

(3) Upon or after the Advisory Council is first convened, the council may advise the Executive Director of the State Department of Health on additional at-large appointments to the council that may be necessary to carry out its duties. At-large appointments to the council may serve on an ad-hoc basis.

(4) Members shall serve for four-year terms and may be reappointed. 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 travel and other miscellaneous expenses necessary to perform their duties within the limits of funds made available to the council for its purposes.

(5) The 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.

(6) The council shall meet periodically, but at least three (3) times annually. 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.

(7) The State Department of Health may provide staff services to the advisory council.

SECTION 3. Purpose and duties. (1) The purpose of the Council shall be to:

(a) Coordinate statewide efforts for the study of the incidence of rare disease within Mississippi and the status of the rare disease community;

(b) 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; and

(c) Coordinate the performance of the council's duties with other state rare disease advisory bodies, community-based organizations, and other public and private organizations for the purpose of ensuring greater cooperation between state and federal activities regarding the research, diagnosis, and treatment of rare diseases. Federal agencies may include, but are not exclusive to, the U.S. National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA). Such coordination shall require, when appropriate:

(i) Disseminating the council's research, identified best practices, and policy recommendations; and

(ii) The utilization of common research collection and dissemination procedures.

(2) The duties of the council shall be to:

(a) Research and determine the most appropriate method to collect rare disease data, and such information concerning these patients as the council deems necessary and appropriate to conduct thorough and complete surveys of rare disease diagnosed in Mississippi, subject to all applicable privacy laws and protection.

(b) The council shall ensure that the duties described in paragraph (a) are carried out in a manner that is coordinated and interoperable with similar research being conducted at the state and federal level.

(c) Research and identify priorities relating to the quality and cost-effectiveness of, and access to, treatment and services provided to persons with rare diseases in Mississippi, and develop policy recommendations on those issues.

(d) Identify best practices for rare disease care from other states and at the national level that will improve rare disease care in Mississippi.

(e) Develop effective strategies to raise public awareness of rare diseases in Mississippi.

SECTION 4. Funding and reporting. (1) Prior to appointing members of the council pursuant to Section 2 of this act, the State Department of Health shall research and report to the Legislature on existing sources of funding that may be used to finance the formation and operation of the council.

(2) 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.

(3) The council shall report to the State Department of Health and to the Legislature biennially 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 Mississippi.

SECTION 5. This act shall take effect and be in force from and after July 1, 2018.