[Congressional Bills 117th Congress]
[From the U.S. Government Publishing Office]
[H.R. 254 Introduced in House (IH)]
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117th CONGRESS
1st Session
H. R. 254
To amend the Public Health Service Act to create a National
Neuromyelitis Optica Spectrum Disorder Consortium to provide grants,
coordinate synergistic research, and targeted therapy with respect to
the causes of, and risk factors associated with, neuromyelitis optica
spectrum disorder, and for other purposes.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
January 11, 2021
Ms. Lee of California introduced the following bill; which was referred
to the Committee on Energy and Commerce
_______________________________________________________________________
A BILL
To amend the Public Health Service Act to create a National
Neuromyelitis Optica Spectrum Disorder Consortium to provide grants,
coordinate synergistic research, and targeted therapy with respect to
the causes of, and risk factors associated with, neuromyelitis optica
spectrum disorder, and for other purposes.
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Neuromyelitis Optica Spectrum
Disorder Consortium Act''.
SEC. 2. FINDINGS.
Congress finds the following:
(1) Neuromyelitis optica spectrum disorder (in this section
and section 3 referred to as ``NMOSD'') is a devastating
neurologic disease leading to blindness, paralysis, and
premature death.
(2) There are an estimated 16,000 to 17,000 people with
NMOSD in the United States and more than a quarter-million
patients worldwide.
(3) Women are affected up to 7 times more than men, and
Afro-Caribbeans and Latino persons are about 2.5 times more
predisposed to NMOSD than Caucasians. The reasons why Blacks
and Hispanics are disproportionately affected cannot be fully
understood without further studies. Furthermore, why NMOSD
disproportionately occurs in females is unknown.
(4) The average age at diagnosis is approximately 35 to 45
years, a peak window of time that further compounds the burden
of NMOSD on parenthood and careers of women and men. The age
range of NMOSD patients is broad and includes children as young
as 3 years of age and adults as old as 90.
(5) NMOSD imposes substantial costs for affected patients
and their families both in financial costs such those
associated with medical care, prescription medicines, and
emergency room visits, as well as in opportunity costs such as
its negative impact on maintaining gainful employment or
attending school or career development programs.
(6) The origins of NMOSD are unknown, but it is
hypothesized to be autoimmune in nature. Collectively,
autoimmune diseases currently affect approximately 1 in 10
Americans. Without a clear understanding of the causes of
NMOSD, development of cures that save and improve lives and
reduce the substantial associated health care costs will not be
possible.
(7) Despite the recent Food and Drug Administration
approval of three medications for NMOSD, there remains an unmet
need for more effective and safe therapies to spare these
patients from this recurrent disease with its accumulating
neurologic disability.
(8) Because of their relatively low overall incidence,
orphan diseases like NMOSD frequently do not receive sufficient
attention and research funding. Of special importance is the
opportunity for the remarkable progress made recently regarding
NMOSD to serve as--
(A) a model for solutions to rare and immunologic
diseases; and
(B) an exemplary therapeutic disease target for
immunosuppressive therapies and for determining
vaccination benefits and risks relative to COVID-19.
(9) No single institution has a sufficient number of
patients to independently conduct research that will adequately
address the cause, prevention, treatment, and potential cure of
NMOSD. Furthermore, there is a paucity of resources available
for regenerative medicine research in NMOSD that will be
required to repair optic nerve and spinal cord damage caused by
NMOSD and thus to restore health.
(10) There has been no comprehensive study analyzing all
relevant clinical, biological, and epidemiological aspects of
NMOSD to identify potential risk factors and biomarkers for
NMOSD.
(11) We can apply our understanding of NMOSD to the study
of other autoimmune diseases, including type 1 diabetes
mellitus, rheumatoid arthritis, psoriasis, multiple sclerosis,
systemic lupus erythematosus, and many others.
SEC. 3. SENSE OF CONGRESS.
It is the sense of Congress that--
(1) there is a need to establish and coordinate a
synergistic, multicenter research effort based on collaboration
between regional consortia and governmental and nongovernmental
entities in order to--
(A) comprehensively study the causes of NMOSD;
(B) identify potential biomarkers of disease
activity;
(C) leverage recent efforts in developing approved
therapies for NMOSD as a model for developing
breakthrough therapies for other autoimmune diseases;
and
(D) highlight NMOSD as a model disease to better
understand the potential benefits and risks of
immunosuppressive therapy and innovative vaccine
strategies targeting COVID-19;
(2) there is a need to encourage a collaborative effort
among academic medical centers comprising epidemiological study
groups capable of gathering comprehensive and detailed
information for each patient enrolled in those groups; and
(3) the effort referred to in paragraph (2) should
facilitate investigation of environmental, nutritional,
genetic, and treatment factors with respect to the pathological
and epidemiological characteristics of NMOSD.
SEC. 4. ESTABLISHMENT OF THE NATIONAL NEUROMYELITIS OPTICA SPECTRUM
DISORDER CONSORTIUM.
Part B of title IV of the Public Health Service Act (42 U.S.C. 284
et seq.) is amended by adding after section 409J the following new
section:
``SEC. 409K. NATIONAL NEUROMYELITIS OPTICA SPECTRUM DISORDER
CONSORTIUM.
``(a) Establishment of the National Neuromyelitis Optica Spectrum
Disorder Consortium.--
``(1) In general.--Not later than 1 year after the date of
the enactment of this section, the Secretary, acting through
the Director of NIH, and in coordination with the Director of
the National Institute on Minority Health and Health
Disparities, shall establish, administer, and coordinate a
National Neuromyelitis Optica Spectrum Disorder Consortium (in
this section referred to as the `NMOSD Consortium') for the
purposes described in paragraph (2).
``(2) Purposes.--The purposes of the NMOSD Consortium shall
be the following:
``(A) Providing grants of not less than 5-years'
duration to eligible consortia for the purpose of
conducting research with respect to the causes of, risk
factors and biomarkers associated with, and treatment
of and comorbidities associated with, NMOSD.
``(B) Assembling a panel of experts to provide,
with respect to research funded by the NMOSD
Consortium, ongoing guidance and recommendations for
the development of the following:
``(i) A standardized study design,
including adaptive clinical trial structures
that may quickly and efficiently evaluate
multiple treatment regimens to optimize
precision and effectively assess personalized
medicine in rare and immunologic diseases.
``(ii) Standard protocols, methods,
procedures, and assays for collecting from
individuals enrolled as study participants a
minimum dataset that includes the following:
``(I) Complete medical history,
including autoimmune and nonautoimmune
comorbidities.
``(II) Neurologic examination and
standardization of critical clinical
outcomes such as the definition and
adjudication of relapse in NMOSD.
``(III) Biospecimens, including
serum, blood cells, cerebrospinal
fluid, DNA, and RNA.
``(IV) Radiological data, including
magnetic resonance imaging (MRI) and
optical coherence tomography, among
other modalities.
``(iii) Specific analytical methods for
examining data, including bioinformatic and
computational modeling for deterministic as
well as predictive capabilities.
``(iv) Provisions for consensus review of
enrolled cases, including clinical trial data
as well as off-label drug use and epidemiologic
studies that would be offer greater insights if
considered in aggregate than alone.
``(v) An integrated data collection
network, including registry and other
activities that improve scientific and clinical
efficiencies in achieving the purposes outlined
in this paragraph.
``(C) Designating a consortium-dedicated laboratory
to collect, analyze, and aggregate data with respect to
research funded by the NMOSD Consortium and to make
such data and analysis available to researchers.
``(3) Eligible consortia.--To be eligible for a grant under
this section, a consortium shall demonstrate the following:
``(A) The consortium has the capability to enroll
as research participants a minimum of 25 individuals
with a diagnosis of NMO from the consortium's
designated catchment area.
``(B) The designated catchment area of the
consortium does not overlap with the designated
catchment area of another consortium already receiving
a grant under this section.
``(4) Report.--Not later than 1 year after the date of the
enactment of this section, and annually thereafter, the
Secretary, acting through the Director of NIH, shall submit to
Congress a report with respect to the NMOSD Consortium, to be
made publicly available, including a summary of research funded
by the NMOSD Consortium and a list of consortia receiving
grants through the NMOSD Consortium. At the discretion of the
Secretary, such report may be combined with other similar or
existing reports.
``(5) Authorization of appropriations.--
``(A) In general.--There is authorized to be
appropriated $25,000,000 for each of fiscal years 2021
through 2024, to remain available until expended, to
carry out this section.
``(B) Sense of congress.--It is the sense of
Congress that funds appropriated to carry out this
section should be in addition to funds otherwise
available or appropriated to carry out the activities
described in this section.
``(b) Definitions.--For purposes of this section:
``(1) Catchment area.--The term `catchment area' means a
defined area for which population data are available.
``(2) Consortium.--The term `consortium' means a
partnership of two or more universities, health care
organizations, or government agencies, or any combination of
such entities, serving a designated catchment area.''.
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