Neuromyelitis Optica Spectrum Disorder Consortium Act

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 syn- ergistic 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 tar- geted 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 Representa- 1 tives of the United States of America in Congress assembled, 2 SECTION 1. SHORT TITLE. 3 This Act may be cited as the ‘‘Neuromyelitis Optica 4 Spectrum Disorder Consortium Act’’. 5 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 2 •HR 254 IH SEC. 2. FINDINGS. 1 Congress finds the following: 2 (1) Neuromyelitis optica spectrum disorder (in 3 this section and section 3 referred to as ‘‘NMOSD’’) 4 is a devastating neurologic disease leading to blind- 5 ness, paralysis, and premature death. 6 (2) There are an estimated 16,000 to 17,000 7 people with NMOSD in the United States and more 8 than a quarter-million patients worldwide. 9 (3) Women are affected up to 7 times more 10 than men, and Afro-Caribbeans and Latino persons 11 are about 2.5 times more predisposed to NMOSD 12 than Caucasians. The reasons why Blacks and His- 13 panics are disproportionately affected cannot be fully 14 understood without further studies. Furthermore, 15 why NMOSD disproportionately occurs in females is 16 unknown. 17 (4) The average age at diagnosis is approxi- 18 mately 35 to 45 years, a peak window of time that 19 further compounds the burden of NMOSD on par- 20 enthood and careers of women and men. The age 21 range of NMOSD patients is broad and includes 22 children as young as 3 years of age and adults as 23 old as 90. 24 (5) NMOSD imposes substantial costs for af- 25 fected patients and their families both in financial 26 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 3 •HR 254 IH costs such those associated with medical care, pre- 1 scription medicines, and emergency room visits, as 2 well as in opportunity costs such as its negative im- 3 pact on maintaining gainful employment or attend- 4 ing school or career development programs. 5 (6) The origins of NMOSD are unknown, but 6 it is hypothesized to be autoimmune in nature. Col- 7 lectively, autoimmune diseases currently affect ap- 8 proximately 1 in 10 Americans. Without a clear un- 9 derstanding of the causes of NMOSD, development 10 of cures that save and improve lives and reduce the 11 substantial associated health care costs will not be 12 possible. 13 (7) Despite the recent Food and Drug Adminis- 14 tration approval of three medications for NMOSD, 15 there remains an unmet need for more effective and 16 safe therapies to spare these patients from this re- 17 current disease with its accumulating neurologic dis- 18 ability. 19 (8) Because of their relatively low overall inci- 20 dence, orphan diseases like NMOSD frequently do 21 not receive sufficient attention and research funding. 22 Of special importance is the opportunity for the re- 23 markable progress made recently regarding NMOSD 24 to serve as— 25 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 4 •HR 254 IH (A) a model for solutions to rare and 1 immunologic diseases; and 2 (B) an exemplary therapeutic disease tar- 3 get for immunosuppressive therapies and for 4 determining vaccination benefits and risks rel- 5 ative to COVID–19. 6 (9) No single institution has a sufficient num- 7 ber of patients to independently conduct research 8 that will adequately address the cause, prevention, 9 treatment, and potential cure of NMOSD. Further- 10 more, there is a paucity of resources available for re- 11 generative medicine research in NMOSD that will be 12 required to repair optic nerve and spinal cord dam- 13 age caused by NMOSD and thus to restore health. 14 (10) There has been no comprehensive study 15 analyzing all relevant clinical, biological, and epide- 16 miological aspects of NMOSD to identify potential 17 risk factors and biomarkers for NMOSD. 18 (11) We can apply our understanding of 19 NMOSD to the study of other autoimmune diseases, 20 including type 1 diabetes mellitus, rheumatoid ar- 21 thritis, psoriasis, multiple sclerosis, systemic lupus 22 erythematosus, and many others. 23 SEC. 3. SENSE OF CONGRESS. 24 It is the sense of Congress that— 25 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 5 •HR 254 IH (1) there is a need to establish and coordinate 1 a synergistic, multicenter research effort based on 2 collaboration between regional consortia and govern- 3 mental and nongovernmental entities in order to— 4 (A) comprehensively study the causes of 5 NMOSD; 6 (B) identify potential biomarkers of disease 7 activity; 8 (C) leverage recent efforts in developing 9 approved therapies for NMOSD as a model for 10 developing breakthrough therapies for other 11 autoimmune diseases; and 12 (D) highlight NMOSD as a model disease 13 to better understand the potential benefits and 14 risks of immunosuppressive therapy and innova- 15 tive vaccine strategies targeting COVID–19; 16 (2) there is a need to encourage a collaborative 17 effort among academic medical centers comprising 18 epidemiological study groups capable of gathering 19 comprehensive and detailed information for each pa- 20 tient enrolled in those groups; and 21 (3) the effort referred to in paragraph (2) 22 should facilitate investigation of environmental, nu- 23 tritional, genetic, and treatment factors with respect 24 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 6 •HR 254 IH to the pathological and epidemiological characteris- 1 tics of NMOSD. 2 SEC. 4. ESTABLISHMENT OF THE NATIONAL 3 NEUROMYELITIS OPTICA SPECTRUM DIS- 4 ORDER CONSORTIUM. 5 Part B of title IV of the Public Health Service Act 6 (42 U.S.C. 284 et seq.) is amended by adding after section 7 409J the following new section: 8 ‘‘SEC. 409K. NATIONAL NEUROMYELITIS OPTICA SPECTRUM 9 DISORDER CONSORTIUM. 10 ‘‘(a) ESTABLISHMENT OF THE NATIONAL 11 NEUROMYELITIS OPTICA SPECTRUM DISORDER CONSOR- 12 TIUM.— 13 ‘‘(1) IN GENERAL.—Not later than 1 year after 14 the date of the enactment of this section, the Sec- 15 retary, acting through the Director of NIH, and in 16 coordination with the Director of the National Insti- 17 tute on Minority Health and Health Disparities, 18 shall establish, administer, and coordinate a Na- 19 tional Neuromyelitis Optica Spectrum Disorder Con- 20 sortium (in this section referred to as the ‘NMOSD 21 Consortium’) for the purposes described in para- 22 graph (2). 23 ‘‘(2) PURPOSES.—The purposes of the NMOSD 24 Consortium shall be the following: 25 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 7 •HR 254 IH ‘‘(A) Providing grants of not less than 5- 1 years’ duration to eligible consortia for the pur- 2 pose of conducting research with respect to the 3 causes of, risk factors and biomarkers associ- 4 ated with, and treatment of and comorbidities 5 associated with, NMOSD. 6 ‘‘(B) Assembling a panel of experts to pro- 7 vide, with respect to research funded by the 8 NMOSD Consortium, ongoing guidance and 9 recommendations for the development of the 10 following: 11 ‘‘(i) A standardized study design, in- 12 cluding adaptive clinical trial structures 13 that may quickly and efficiently evaluate 14 multiple treatment regimens to optimize 15 precision and effectively assess personal- 16 ized medicine in rare and immunologic dis- 17 eases. 18 ‘‘(ii) Standard protocols, methods, 19 procedures, and assays for collecting from 20 individuals enrolled as study participants a 21 minimum dataset that includes the fol- 22 lowing: 23 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 8 •HR 254 IH ‘‘(I) Complete medical history, 1 including autoimmune and nonauto- 2 immune comorbidities. 3 ‘‘(II) Neurologic examination and 4 standardization of critical clinical out- 5 comes such as the definition and adju- 6 dication of relapse in NMOSD. 7 ‘‘(III) Biospecimens, including 8 serum, blood cells, cerebrospinal fluid, 9 DNA, and RNA. 10 ‘‘(IV) Radiological data, includ- 11 ing magnetic resonance imaging 12 (MRI) and optical coherence tomog- 13 raphy, among other modalities. 14 ‘‘(iii) Specific analytical methods for 15 examining data, including bioinformatic 16 and computational modeling for deter- 17 ministic as well as predictive capabilities. 18 ‘‘(iv) Provisions for consensus review 19 of enrolled cases, including clinical trial 20 data as well as off-label drug use and epi- 21 demiologic studies that would be offer 22 greater insights if considered in aggregate 23 than alone. 24 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 9 •HR 254 IH ‘‘(v) An integrated data collection net- 1 work, including registry and other activi- 2 ties that improve scientific and clinical effi- 3 ciencies in achieving the purposes outlined 4 in this paragraph. 5 ‘‘(C) Designating a consortium-dedicated 6 laboratory to collect, analyze, and aggregate 7 data with respect to research funded by the 8 NMOSD Consortium and to make such data 9 and analysis available to researchers. 10 ‘‘(3) ELIGIBLE CONSORTIA.—To be eligible for 11 a grant under this section, a consortium shall dem- 12 onstrate the following: 13 ‘‘(A) The consortium has the capability to 14 enroll as research participants a minimum of 25 15 individuals with a diagnosis of NMO from the 16 consortium’s designated catchment area. 17 ‘‘(B) The designated catchment area of the 18 consortium does not overlap with the designated 19 catchment area of another consortium already 20 receiving a grant under this section. 21 ‘‘(4) REPORT.—Not later than 1 year after the 22 date of the enactment of this section, and annually 23 thereafter, the Secretary, acting through the Direc- 24 tor of NIH, shall submit to Congress a report with 25 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 10 •HR 254 IH respect to the NMOSD Consortium, to be made pub- 1 licly available, including a summary of research 2 funded by the NMOSD Consortium and a list of 3 consortia receiving grants through the NMOSD Con- 4 sortium. At the discretion of the Secretary, such re- 5 port may be combined with other similar or existing 6 reports. 7 ‘‘(5) AUTHORIZATION OF APPROPRIATIONS.— 8 ‘‘(A) IN GENERAL.—There is authorized to 9 be appropriated $25,000,000 for each of fiscal 10 years 2021 through 2024, to remain available 11 until expended, to carry out this section. 12 ‘‘(B) SENSE OF CONGRESS.—It is the 13 sense of Congress that funds appropriated to 14 carry out this section should be in addition to 15 funds otherwise available or appropriated to 16 carry out the activities described in this section. 17 ‘‘(b) DEFINITIONS.—For purposes of this section: 18 ‘‘(1) CATCHMENT AREA.—The term ‘catchment 19 area’ means a defined area for which population 20 data are available. 21 ‘‘(2) CONSORTIUM.—The term ‘consortium’ 22 means a partnership of two or more universities, 23 health care organizations, or government agencies, 24 VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS 11 •HR 254 IH or any combination of such entities, serving a des- 1 ignated catchment area.’’. 2 Æ VerDate Sep 11 2014 01:01 Feb 05, 2021 Jkt 019200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6301 E:\BILLS\H254.IH H254 kjohnson on DSK79L0C42PROD with BILLS