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PRINTER'S NO. 261
THE GENERAL ASSEMBLY OF PENNSYLVANIA
HOUSE BILL
No.
239
Session of
2017
INTRODUCED BY TOEPEL, LONGIETTI, BAKER, V. BROWN, D. COSTA,
HEFFLEY, MURT, O'BRIEN, SCHLOSSBERG, SIMMONS, TOPPER AND
WARD, FEBRUARY 1, 2017
REFERRED TO COMMITTEE ON HEALTH, FEBRUARY 1, 2017
AN ACT
Establishing the Rare Disease Advisory Council and providing for
its powers and duties; and providing for duties of the
Department of Health, the Insurance Department, the
Department of Human Services and the Department of Education.
The General Assembly of the Commonwealth of Pennsylvania
hereby enacts as follows:
Section 1. Short title.
This act shall be known and may be cited as the Rare Disease
Advisory Council Act.
Section 2. Definitions.
The following words and phrases when used in this act shall
have the meanings given to them in this section unless the
context clearly indicates otherwise:
"Advisory council." The Rare Disease Advisory Council
established in section 3.
"Department." The Department of Health of the Commonwealth.
"Rare disease." A disease or condition that affects fewer
than 200,000 individuals living in the United States.
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"Secretary." The Secretary of Health of the Commonwealth.
Section 3. Establishment and membership.
(a) Establishment.--The Rare Disease Advisory Council is
established.
(b) Members.--The advisory council shall consist of the
following members:
(1) The secretary or a designee authorized to act on
behalf of the secretary.
(2) The heads of State agencies that may be concerned
with the provision of care to persons with rare diseases or
designees authorized to act on behalf of the agency heads. At
a minimum, members under this paragraph shall include the
Secretary of Human Services and the Secretary of Education or
their respective designees.
(3) The Insurance Commissioner or a designee authorized
to act on behalf of the Insurance Commissioner.
(4) Public members to be appointed by the secretary, who
shall include:
(i) Three physicians licensed to practice in this
Commonwealth who have expertise in the diagnosis and
management of patients with rare diseases, one of whom
shall be a pediatrician who provides care to children
with rare diseases.
(ii) Two registered professional nurses or nurse
practitioners licensed in this Commonwealth who have
expertise in providing care to patients with rare
diseases.
(iii) An epidemiologist licensed in this
Commonwealth who has expertise in the incidence,
prevalence and control of rare diseases.
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(iv) Two representatives of hospitals in this
Commonwealth, one of which must be a research hospital.
(v) Two representatives of the health insurance
industry.
(vi) Two representatives involved in the
biopharmaceutical industry.
(vii) Two representatives of the scientific
community who are engaged in rare disease research.
(viii) Two parents, each of whom has a child with a
rare disease, not of the same family.
(ix) Two individuals with rare diseases.
(x) Representatives of two rare disease-specific
patient organizations that operate within this
Commonwealth.
(c) Additional members.--The advisory council may at any
time advise the department on additional at-large appointments
to the advisory council that may be necessary to carry out its
duties. At-large appointees to the advisory council may serve on
an ad hoc basis.
(d) Vacancies.--Vacancies in the membership of the advisory
council shall be filled in the same manner as provided for the
original appointments.
(e) Appointment of public members and chairperson.--The
secretary shall appoint the public members of the advisory
council and a chairperson of the advisory council within 60 days
of the effective date of this section.
(f) Compensation.--The public members of the advisory
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
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advisory council for its purposes. Reimbursement for expenses
incurred by public members of the advisory council shall be
provided by the department according to standard criteria
currently in existence.
(g) Meetings.--The advisory council shall meet periodically,
at a minimum of three times each year. A member may participate
in a meeting by teleconference. The initial meeting of the
advisory council shall be held within 90 days of the effective
date of this section.
(h) Assistance of other government agencies.--The advisory
council shall be entitled to call to its assistance and avail
itself of the services of the employees of any State or local
government agency as it may require and as may be available to
it for its purposes.
(i) Staff.--The department may provide staff services to the
advisory council, including providing a secretary to the
advisory council.
Section 4. Purpose and duties.
(a) Purpose.--The purpose of the advisory council is to:
(1) Coordinate Statewide efforts for the study of the
incidence and prevalence of rare diseases within this
Commonwealth and the status of the rare disease community.
(2) Act as the advisory body on rare diseases to the
General Assembly and to all relevant State and private
agencies that provide services to, or are charged with the
care of, individuals with rare diseases.
(3) Coordinate the performance of the advisory council's
duties with those of other rare disease advisory bodies and
community-based organizations and other public and private
organizations within this Commonwealth for the purpose of
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ensuring greater cooperation between the entities within this
Commonwealth and Federal agencies regarding the research,
diagnosis and treatment of rare diseases. For purposes of
this paragraph, Federal agencies may include, but are not
limited to, the National Institutes of Health and the Food
and Drug Administration. The coordination shall require, when
appropriate:
(i) Disseminating the outcomes of the advisory
council's research, identified best practices and policy
recommendations.
(ii) Utilizing common research collection and
dissemination procedures.
(b) Duties.--The duties of the advisory council are to:
(1) Research and determine the most appropriate method
to collect rare disease data, and information concerning
individuals with rare diseases, as the advisory council deems
necessary and appropriate to conduct comprehensive surveys of
rare diseases diagnosed in this Commonwealth, subject to all
applicable privacy laws and protections.
(2) Ensure that the duties described in subsection (a)
(1) are carried out in a manner that is coordinated and
interoperable with other similar research being conducted at
the Federal and State level.
(3) Research and identify priorities relating to the
quality and cost effectiveness of and access to treatment and
other services provided to individuals with rare diseases in
this Commonwealth and develop policy recommendations on those
issues.
(4) Identify best practices in other states and at the
national level for rare disease management, monitoring and
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surveillance, education, detection, diagnosis, information
and care to improve these functions and capabilities relative
to rare diseases within this Commonwealth.
(5) Develop effective strategies to raise public
awareness of rare diseases within this Commonwealth.
(6) Coordinate the development of a task force,
comprised of members of the advisory council and other
individuals as may be necessary and appropriate to facilitate
the development and publication of the report specified in
section 6(b).
(c) Construction.--Nothing in this act shall be construed to
prohibit the advisory council from issuing interim reports or
taking interim actions should these seem appropriate.
Section 5. Funding.
(a) Report to General Assembly.--Prior to appointing members
of the advisory council, the department shall research and
report to the General Assembly within 30 days of the effective
date of this section existing sources of funding that may be
used to finance the formation and operation of the advisory
council.
(b) Grant and other funding.--The advisory council shall
apply for, and is authorized to accept, a grant of money from
the Federal Government, a private foundation or any other source
which may be available for programs related to rare diseases.
Section 6. Reports.
(a) Preliminary report.--Within 12 months of the effective
date of this section, the advisory council shall deliver to the
Public Health and Welfare Committee of the Senate and the Health
Committee of the House of Representatives a preliminary report
on the work of the advisory council. The report shall be made
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available to the public. The report shall include a summary of
the accomplishments of the advisory council since its formation
and the status of its work in preparation of the report required
under subsection (b).
(b) Report.--Within two years of the effective date of this
section, the advisory council shall deliver to the Public Health
and Welfare Committee of the Senate and the Health Committee of
the House of Representatives a comprehensive report. The report
shall be made available to the public. The report shall include
the following:
(1) The incidence and prevalence of rare diseases within
this Commonwealth.
(2) The needs of the rare disease community within this
Commonwealth and the actions necessary and feasible to
address those needs.
(c) Biennial reports.--The advisory council shall report
biennially to the department, the Public Health and Welfare
Committee of the Senate and the Health Committee of the House of
Representatives on the activities of the advisory council and
its findings and recommendations on issues relating to the
management, monitoring and surveillance, education, detection,
diagnosis, information and care regarding rare diseases in this
Commonwealth, including the quality and cost effectiveness of
care, access to treatment and other relevant services for
individuals affected by rare diseases.
Section 7. Expiration.
This act shall expire June 30, 2025.
Section 8. Effective date.
This act shall take effect immediately.
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