I 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Neuromyelitis Optica 

5 Spectrum Disorder Consortium Act’’. 


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1 SEC. 2. FINDINGS. 

2 Congress finds the following: 

3 (1) Neuromyelitis optica spectrum disorder (in 

4 this section and section 3 referred to as ‘‘NMOSD’’) 

5 is a devastating neurologic disease leading to blind6 ness, paralysis, and premature death. 

7 (2) There are an estimated 16,000 to 17,000 

8 people with NMOSD in the United States and more 

9 than a quarter-million patients worldwide. 

10 (3) Women are affected up to 7 times more 

11 than men, and Afro-Caribbeans and Latino persons 

12 are about 2.5 times more predisposed to NMOSD 

13 than Caucasians. The reasons why Blacks and His14 panics are disproportionately affected cannot be fully 

15 understood without further studies. Furthermore, 

16 why NMOSD disproportionately occurs in females is 

17 unknown. 

18 (4) The average age at diagnosis is approxi19 mately 35 to 45 years, a peak window of time that 

20 further compounds the burden of NMOSD on par21 enthood and careers of women and men. The age 

22 range of NMOSD patients is broad and includes 

23 children as young as 3 years of age and adults as 

24 old as 90. 

25 (5) NMOSD imposes substantial costs for af26 fected patients and their families both in financial 


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1 costs such those associated with medical care, pre2 scription medicines, and emergency room visits, as 

3 well as in opportunity costs such as its negative im4 pact on maintaining gainful employment or attend5 ing school or career development programs. 

6 (6) The origins of NMOSD are unknown, but 

7 it is hypothesized to be autoimmune in nature. Col8 lectively, autoimmune diseases currently affect ap9 proximately 1 in 10 Americans. Without a clear un10 derstanding of the causes of NMOSD, development 

11 of cures that save and improve lives and reduce the 

12 substantial associated health care costs will not be 

13 possible. 

14 (7) Despite the recent Food and Drug Adminis15 tration approval of three medications for NMOSD, 

16 there remains an unmet need for more effective and 

17 safe therapies to spare these patients from this re18 current disease with its accumulating neurologic dis19 ability. 

20 (8) Because of their relatively low overall inci21 dence, orphan diseases like NMOSD frequently do 

22 not receive sufficient attention and research funding. 

23 Of special importance is the opportunity for the re24 markable progress made recently regarding NMOSD 

25 to serve as— 


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1 (A) a model for solutions to rare and 

2 immunologic diseases; and 

3 (B) an exemplary therapeutic disease tar4 get for immunosuppressive therapies and for 

5 determining vaccination benefits and risks rel6 ative to COVID–19. 

7 (9) No single institution has a sufficient num8 ber of patients to independently conduct research 

9 that will adequately address the cause, prevention, 

10 treatment, and potential cure of NMOSD. Further11 more, there is a paucity of resources available for re12 generative medicine research in NMOSD that will be 

13 required to repair optic nerve and spinal cord dam14 age caused by NMOSD and thus to restore health. 

15 (10) There has been no comprehensive study 

16 analyzing all relevant clinical, biological, and epide17 miological aspects of NMOSD to identify potential 

18 risk factors and biomarkers for NMOSD. 

19 (11) We can apply our understanding of 

20 NMOSD to the study of other autoimmune diseases, 

21 including type 1 diabetes mellitus, rheumatoid ar22 thritis, psoriasis, multiple sclerosis, systemic lupus 

23 erythematosus, and many others. 

24 SEC. 3. SENSE OF CONGRESS. 

25 It is the sense of Congress that— 


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1 (1) there is a need to establish and coordinate 

2 a synergistic, multicenter research effort based on 

3 collaboration between regional consortia and govern4 mental and nongovernmental entities in order to— 

5 (A) comprehensively study the causes of 

6 NMOSD; 

7 (B) identify potential biomarkers of disease 

8 activity; 

9 (C) leverage recent efforts in developing 

10 approved therapies for NMOSD as a model for 

11 developing breakthrough therapies for other 

12 autoimmune diseases; and 

13 (D) highlight NMOSD as a model disease 

14 to better understand the potential benefits and 

15 risks of immunosuppressive therapy and innova16 tive vaccine strategies targeting COVID–19; 

17 (2) there is a need to encourage a collaborative 

18 effort among academic medical centers comprising 

19 epidemiological study groups capable of gathering 

20 comprehensive and detailed information for each pa21 tient enrolled in those groups; and 

22 (3) the effort referred to in paragraph (2) 

23 should facilitate investigation of environmental, nu24 tritional, genetic, and treatment factors with respect 


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1 to the pathological and epidemiological characteris2 tics of NMOSD. 

3 SEC. 4. ESTABLISHMENT OF THE NATIONAL 

4 NEUROMYELITIS OPTICA SPECTRUM DIS5 ORDER CONSORTIUM. 

6 Part B of title IV of the Public Health Service Act 

7 (42 U.S.C. 284 et seq.) is amended by adding after section 

8 409J the following new section: 

9 ‘‘SEC. 409K. NATIONAL NEUROMYELITIS OPTICA SPECTRUM 

10 DISORDER CONSORTIUM. 

11 ‘‘(a) ESTABLISHMENT OF THE NATIONAL

12 NEUROMYELITIS OPTICA SPECTRUM DISORDER CONSOR13 TIUM.— 

14 ‘‘(1) IN GENERAL.—Not later than 1 year after 

15 the date of the enactment of this section, the Sec16 retary, acting through the Director of NIH, and in 

17 coordination with the Director of the National Insti18 tute on Minority Health and Health Disparities, 

19 shall establish, administer, and coordinate a Na20 tional Neuromyelitis Optica Spectrum Disorder Con21 sortium (in this section referred to as the ‘NMOSD 

22 Consortium’) for the purposes described in para23 graph (2). 

24 ‘‘(2) PURPOSES.—The purposes of the NMOSD 

25 Consortium shall be the following: 


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1 ‘‘(A) Providing grants of not less than 5- 

2 years’ duration to eligible consortia for the pur3 pose of conducting research with respect to the 

4 causes of, risk factors and biomarkers associ5 ated with, and treatment of and comorbidities 

6 associated with, NMOSD. 

7 ‘‘(B) Assembling a panel of experts to pro8 vide, with respect to research funded by the 

9 NMOSD Consortium, ongoing guidance and 

10 recommendations for the development of the 

11 following: 

12 ‘‘(i) A standardized study design, in13 cluding adaptive clinical trial structures 

14 that may quickly and efficiently evaluate 

15 multiple treatment regimens to optimize 

16 precision and effectively assess personal17 ized medicine in rare and immunologic dis18 eases. 

19 ‘‘(ii) Standard protocols, methods, 

20 procedures, and assays for collecting from 

21 individuals enrolled as study participants a 

22 minimum dataset that includes the fol23 lowing: 


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1 ‘‘(I) Complete medical history, 

2 including autoimmune and nonauto3 immune comorbidities. 

4 ‘‘(II) Neurologic examination and 

5 standardization of critical clinical out6 comes such as the definition and adju7 dication of relapse in NMOSD. 

8 ‘‘(III) Biospecimens, including 

9 serum, blood cells, cerebrospinal fluid, 

10 DNA, and RNA. 

11 ‘‘(IV) Radiological data, includ12 ing magnetic resonance imaging 

13 (MRI) and optical coherence tomog14 raphy, among other modalities. 

15 ‘‘(iii) Specific analytical methods for 

16 examining data, including bioinformatic 

17 and computational modeling for deter18 ministic as well as predictive capabilities. 

19 ‘‘(iv) Provisions for consensus review 

20 of enrolled cases, including clinical trial 

21 data as well as off-label drug use and epi22 demiologic studies that would be offer 

23 greater insights if considered in aggregate 

24 than alone. 


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1 ‘‘(v) An integrated data collection net2 work, including registry and other activi3 ties that improve scientific and clinical effi4 ciencies in achieving the purposes outlined 

5 in this paragraph. 

6 ‘‘(C) Designating a consortium-dedicated 

7 laboratory to collect, analyze, and aggregate 

8 data with respect to research funded by the 

9 NMOSD Consortium and to make such data 

10 and analysis available to researchers. 

11 ‘‘(3) ELIGIBLE CONSORTIA.—To be eligible for 

12 a grant under this section, a consortium shall dem13 onstrate the following: 

14 ‘‘(A) The consortium has the capability to 

15 enroll as research participants a minimum of 25 

16 individuals with a diagnosis of NMO from the 

17 consortium’s designated catchment area. 

18 ‘‘(B) The designated catchment area of the 

19 consortium does not overlap with the designated 

20 catchment area of another consortium already 

21 receiving a grant under this section. 

22 ‘‘(4) REPORT.—Not later than 1 year after the 

23 date of the enactment of this section, and annually 

24 thereafter, the Secretary, acting through the Direc25 tor of NIH, shall submit to Congress a report with 


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1 respect to the NMOSD Consortium, to be made pub2 licly available, including a summary of research 

3 funded by the NMOSD Consortium and a list of 

4 consortia receiving grants through the NMOSD Con5 sortium. At the discretion of the Secretary, such re6 port may be combined with other similar or existing 

7 reports. 

8 ‘‘(5) AUTHORIZATION OF APPROPRIATIONS.— 

9 ‘‘(A) IN GENERAL.—There is authorized to 

10 be appropriated $25,000,000 for each of fiscal 

11 years 2021 through 2024, to remain available 

12 until expended, to carry out this section. 

13 ‘‘(B) SENSE OF CONGRESS.—It is the 

14 sense of Congress that funds appropriated to 

15 carry out this section should be in addition to 

16 funds otherwise available or appropriated to 

17 carry out the activities described in this section. 

18 ‘‘(b) DEFINITIONS.—For purposes of this section: 

19 ‘‘(1) CATCHMENT AREA.—The term ‘catchment 

20 area’ means a defined area for which population 

21 data are available. 

22 ‘‘(2) CONSORTIUM.—The term ‘consortium’ 

23 means a partnership of two or more universities, 

24 health care organizations, or government agencies, 


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1 or any combination of such entities, serving a des2 ignated catchment area.’’. 

Æ 


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