Neuromyelitis Optica Spectrum Disorder Consortium Act
Bill: H.R.254 | Sponsor: Rep. Lee, Barbara (D-CA)
Policy area: Health

Summary:
Neuromyelitis Optica Spectrum Disorder Consortium Act
This bill requires the National Institutes of Health to establish a national consortium to coordinate and support research on neuromyelitis optica spectrum disorder, including by awarding grants. This is a neurological disease that can cause blindness, paralysis, and premature death.

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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