Today's Law As Amended

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AB-1016 Rare Disease Advisory Council.(2019-2020)

As Amends the Law Today

 The Legislature finds and declares all of the following:
(a) Under federal law, including the Orphan Drug Act of 1983 and its amendments and the Rare Diseases Act of 2002, a rare disease is generally defined as a disease that affects fewer than 200,000 persons in the United States. Rare diseases are sometimes called orphan diseases. There are about 7,000 known rare diseases affecting approximately 30 million men, women, and children in the United States.
(b) The exact cause for many rare diseases remains unknown. However, 80 percent of rare diseases are genetic in origin and can be linked to mutations in a single gene or in multiple genes. Those diseases are referred to as genetic diseases. A genetic disease can be passed down from generation to generation, explaining why certain rare diseases run in families. It is also estimated that about one-half of all rare diseases affect children.
(c) A person suffering from a rare disease in this state faces a wide range of challenges, including, but not limited to, delays in obtaining a diagnosis, a misdiagnosis, a shortage of medical specialists familiar with and who can provide treatment for rare diseases, the prohibitive costs of treatment, and the inability to access therapies and medication that are used by physicians to treat rare diseases but that have not been approved by the United States Food and Drug Administration 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.
(e) It is, therefore, an appropriate public policy for this state to establish an advisory body whose membership would be comprised of persons with suitable qualifications in the field of rare diseases, 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.

SEC. 2.

 Part 4.7 (commencing with Section 124970) is added to Division 106 of the Health and Safety Code, to read:

PART 4.7. Rare Disease Advisory Council

 (a) There is hereby established, within the California Health and Human Services Agency, the Rare Disease Advisory Council.
(b) For purposes of this part, the following terms have the following meanings:
(1) “Advisory council” means the Rare Disease Advisory Council, unless otherwise specified.
(2) “Rare disease” has the same meaning as provided in Section 360bb of Title 21 of the United States Code, or its successor.
 (a) The advisory council shall consist of the following members:
(1) Directors, or ex officio members, within the California Health and Human Services Agency and other state agencies concerned with the provision of care to persons with rare diseases, to be appointed by the Secretary of California Health and Human Services.
(2) Public members, to be appointed by the State Public Health Officer, who shall include all of the following:
(A) Two physicians and surgeons 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.
(B) A registered professional nurse licensed to practice in this state who has expertise in providing care to patients with rare diseases.
(C) Two representatives of hospitals in this state.
(D) A representative of the health care coverage industry.
(E) A representative of the biopharmaceutical industry.
(F) A representative of the scientific community who is engaged in rare disease research.
(G) Two parents of a child with a rare disease.
(H) Two persons with a rare disease.
(I) Two patient advocacy organizations that operate within this state.
(3) On or after the date on which the advisory council is first convened, the advisory council may advise the California Health and Human Services Agency on additional at-large appointments to the advisory council that may be necessary to carry out its duties. At-large appointments to the advisory council may serve on an ad hoc basis.
(b) Members of the advisory council shall serve without compensation. However, public members appointed pursuant to paragraph (2) of subdivision (a) may be reimbursed for travel and other miscellaneous expenses necessary to perform their duties within the limits of funds made available to the advisory 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 advisory council.
(d) The advisory council shall meet periodically, but at least three times annually. The advisory council may 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, if those employees elect to participate.
(e) The term of each member of the advisory council is three years, to be staggered so that approximately one-third of the appointments expire each year. Vacancies in the membership of the advisory council shall be filled in the same manner as provided for in the original appointments.
 (a) The following are the purposes of the advisory council:
(1) Coordinating statewide efforts for the study of the incidence of rare diseases within this state and the status of the rare disease community.
(2) Acting as the advisory body on rare diseases to the Legislature, and state departments, agencies, commissions, and authorities, and private agencies, that provide services to, or that are charged with the care of, persons with rare diseases.
(3) (A) Coordinating the performance of the advisory council’s duties with any other state entities acting as advisory bodies on rare diseases, community-based organizations, and other public or private organizations, for the purpose of ensuring greater cooperation between state and federal activities regarding the research, diagnosis, and treatment of rare diseases.
(B) Federal agencies with which the advisory council may seek to coordinate may include, but are not limited to, the National Institutes of Health and the United States Food and Drug Administration.
(C) The coordination described in subparagraph (A) shall require, when appropriate, both of the following:
(i) Disseminating the advisory council’s research, identified best practices, and policy recommendations.
(ii) Utilizing common research collection and dissemination procedures.
(b) The following are the duties of the advisory council:
(1) Research and determine the most appropriate method to collect data on rare diseases, and information concerning patients, as the advisory council deems necessary and appropriate by conducting thorough and complete surveys of rare diseases diagnosed in this state, subject to all applicable privacy laws and protections. The advisory council shall ensure that the duties described in this paragraph are carried out in a manner that is coordinated and interoperable with similar research being conducted at the state and federal levels.
(2) 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 this state, and develop policy recommendations on those issues.
(3) Identify best practices for rare disease care from other states and at the national level that will improve rare disease care in this state.
(4) Develop effective strategies to raise public awareness of rare diseases in this state.
 (a) Before any appointments to the advisory council are made pursuant to Section 124970.1, the California Health and Human Services Agency shall research and report to the Legislature on existing sources of funding that may be used to finance the formation and operation of the advisory council.
(b) The advisory council shall apply for, and accept, any grant of funds from the federal government, private foundations, or other sources, that may be available for programs related to rare diseases.
(c) The advisory council shall report to the California Health and Human Services Agency, and to the Legislature, every two years 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 provided to persons with rare diseases in this state.
(d) Any reports submitted to the Legislature pursuant to this section shall be submitted in compliance with Section 9795 of the Government Code.