CCM–CARE Act

I 116TH CONGRESS 1ST SESSION H. R. 3573 To increase research, education, and treatment for cerebral cavernous malformations. IN THE HOUSE OF REPRESENTATIVES JUNE 27, 2019 Mr. LUJA´N (for himself, Ms. HAALAND, and Ms. TORRES SMALL of New Mex- ico) introduced the following bill; which was referred to the Committee on Energy and Commerce A BILL To increase research, education, and treatment for cerebral cavernous malformations. 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 ‘‘Cerebral Cavernous 4 Malformations Clinical Awareness, Research, and Edu- 5 cation Act of 2019’’ or the ‘‘CCM–CARE Act’’. 6 SEC. 2. FINDINGS. 7 Congress finds as follows: 8 (1) Cerebral cavernous malformations (referred 9 to in this section as ‘‘CCM’’), also known as cav- 10 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 2 •HR 3573 IH ernous angioma, or cavernoma, is a devastating 1 blood vessel disease characterized by vascular lesions 2 that develop and grow within the brain and spinal 3 cord. 4 (2) Detection of CCM lesions is achieved 5 through costly and specialized medical imaging tech- 6 niques, often not accessible or convenient to patients 7 who need them. 8 (3) While CCM is a common type of vascular 9 anomaly, many individuals are not aware they have 10 the disease until the onset of serious clinical symp- 11 toms. CCM is often inherited unknowingly. 12 (4) CCM affects an estimated 600,000 people 13 in the United States. 14 (5) Individuals diagnosed with CCM may expe- 15 rience neurological deficits, seizure, stroke, or sud- 16 den death. 17 (6) Due to limited research, there is currently 18 no treatment for CCM other than brain and spinal 19 surgery, and only for certain patients. 20 (7) There is also a shortage of trained physi- 21 cians to provide skilled and timely diagnosis and ap- 22 propriate treatment for CCM. 23 (8) While the hereditary form of CCM may 24 occur among any ethnicity, the presence of a muta- 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 3 •HR 3573 IH tion called the ‘‘common Hispanic mutation’’, has 1 passed through 14 or more generations of American 2 descendants from the original Spanish settlers of the 3 Southwest in the 1590s. New Mexico has the highest 4 population density of CCM in the world; Texas, Ari- 5 zona, and Colorado also have high rates of CCM due 6 to the common Hispanic mutation. 7 (9) A second mutation (CCM2 Common Dele- 8 tion) originating in the Southeastern United States 9 before 1800 has increased rates of the illness in 10 South Carolina, Georgia, Florida, Alabama, Mis- 11 sissippi, Louisiana, Texas, Oklahoma, Kentucky, 12 Kansas, and northern California. 13 SEC. 3. EXPANSION AND COORDINATION OF ACTIVITIES OF 14 NATIONAL INSTITUTES OF HEALTH WITH RE- 15 SPECT TO CEREBRAL CAVERNOUS MAL- 16 FORMATIONS RESEARCH. 17 Part B of title IV of the Public Health Service Act 18 (42 U.S.C. 284 et seq.) is amended by adding at the end 19 the following: 20 ‘‘SEC. 409K. CEREBRAL CAVERNOUS MALFORMATIONS RE- 21 SEARCH ACTIVITIES. 22 ‘‘(a) EXPANSION AND COORDINATION OF ACTIVI- 23 TIES.—The Director of NIH, in coordination with the di- 24 rectors of the National Institute of Neurological Disorders 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 4 •HR 3573 IH and Stroke, the National Center for Advancing Transla- 1 tional Sciences, the National Heart, Lung, and Blood In- 2 stitute, and other national research institutes, as appro- 3 priate, for the purpose of conducting research and related 4 activities concerning cerebral cavernous malformations 5 (referred to in this section as ‘CCM’)— 6 ‘‘(1) shall strengthen and coordinate efforts of 7 the National Institutes of Health; and 8 ‘‘(2) may award grants and cooperative agree- 9 ments to public or nonprofit private entities (includ- 10 ing State health departments, political subdivisions 11 of States, universities, and other medical or edu- 12 cational entities). 13 ‘‘(b) ACTIVITIES.—The research and related activi- 14 ties described in subsection (a) shall include the following: 15 ‘‘(1) CLINICAL, TRANSLATIONAL, AND BASIC 16 RESEARCH.—The Director of NIH shall conduct or 17 support, through funding opportunity announce- 18 ments, grants, or cooperative agreements, basic, clin- 19 ical, and translational research on CCM, including 20 research on— 21 ‘‘(A) the identification and development of 22 biomarkers that fulfill the requirement of the 23 Food and Drug Administration for biomarker 24 qualification as proper measures of CCM patho- 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 5 •HR 3573 IH genic biology or response to clinical interven- 1 tion; 2 ‘‘(B) safety or efficacy for new or 3 repurposed currently approved drugs for CCM 4 treatment; 5 ‘‘(C) research related to improving and 6 measuring the quality of life for individuals 7 with CCM and their families; 8 ‘‘(D) contributions of genetic variation to 9 clinical presentation as targets for therapy; 10 ‘‘(E) early detection, diagnosis, and treat- 11 ment of CCM; 12 ‘‘(F) clinical training programs aimed at 13 increasing the number of scientists and clini- 14 cians who are trained to treat patients and 15 carry out the research described in this para- 16 graph; 17 ‘‘(G) continued development and expansion 18 of novel animal models for preclinical research 19 relating to CCM; 20 ‘‘(H) pre-clinical and clinical research re- 21 lated to repurposing currently approved drugs 22 for CCM treatment; 23 ‘‘(I) proteomic, pharmacological, and cell 24 biological analysis of CCM molecules; 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 6 •HR 3573 IH ‘‘(J) biological mechanisms for lesion gen- 1 esis, development, and maturation; 2 ‘‘(K) biological mechanisms for lesion 3 bleeding and symptomology; 4 ‘‘(L) novel biomedical and pharmacological 5 interventions designed to inhibit new lesion de- 6 velopment, lesion growth, and lesion bleeding; 7 ‘‘(M) novel biomedical and pharmacological 8 interventions designed to target existing lesions 9 to reduce their size and clinical activity; 10 ‘‘(N) continued research related to under- 11 standing better the natural history and clinical 12 variation associated with CCM, particularly as 13 it relates to the development of drug develop- 14 ment tools and clinical outcome assessments; 15 ‘‘(O) the gut-brain axis and the effects of 16 microbiome composition on clinical symptomolo- 17 gy; and 18 ‘‘(P) the microbiome as a therapeutic tar- 19 get for CCM treatment. 20 ‘‘(2) FACILITATION OF RESEARCH RESOURCES; 21 CLINICAL TRIAL PREPAREDNESS.— 22 ‘‘(A) IN GENERAL.—The Director of NIH 23 shall award grants and contracts to public or 24 nonprofit private entities to fund all or part of 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 7 •HR 3573 IH the cost of planning, establishing, and providing 1 basic operating support for a network of CCM 2 Clinical Research Centers, including Coordi- 3 nating and Participating centers regarding re- 4 search on various forms of CCM. 5 ‘‘(B) CLINICAL AND RESEARCH COORDINA- 6 TION CENTERS.— 7 ‘‘(i) IN GENERAL.—The Director of 8 NIH shall build upon the network created 9 by the U01 Clinical Trial Readiness Re- 10 search Project to identify and support the 11 development of 2 geographically distributed 12 national clinical and research coordinating 13 centers with unique clinical expertise and 14 the potential for coordinating multi-site 15 clinical drug trials with respect to CCM. 16 ‘‘(ii) DUTIES.—The coordinating cen- 17 ters identified under clause (i) shall pro- 18 vide a model for the participation centers 19 described in paragraph (3), facilitate med- 20 ical research to develop a cure for CCM, 21 and enhance the medical care of individ- 22 uals with CCM nationwide, including by— 23 ‘‘(I) maintaining an institutional 24 infrastructure capable of hosting clin- 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 8 •HR 3573 IH ical trials and facilitating translational 1 research projects and collaborations 2 for clinical trials; 3 ‘‘(II) implementing the programs 4 dedicated to patient education, patient 5 outreach, and awareness developed by 6 the Cerebral Cavernous Malformations 7 Consortium under subsection 8 (c)(3)(B); 9 ‘‘(III) developing the capacity to 10 establish and maintain communication 11 with other major CCM research and 12 care institutions internationally for in- 13 formation sharing and coordination of 14 research activities; 15 ‘‘(IV) demonstrating clinical ex- 16 pertise in the management of CCM 17 and appointing a director and support 18 staff, including a trainee and patient 19 representative, for CCM research pro- 20 gramming; 21 ‘‘(V) treating a sufficient number 22 of eligible patients for participation 23 with particular focus on unique sub- 24 populations, such as patients with the 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00008 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 9 •HR 3573 IH common Hispanic mutation, Ash- 1 kenazi Jewish mutation, CCM2 Com- 2 mon Deletion, or CCM3 gene muta- 3 tion carriers; and 4 ‘‘(VI) maintaining a telehealth 5 infrastructure to support and provide 6 clinical consultation for remote and 7 underserved communities. 8 ‘‘(3) PARTICIPATION CENTERS.— 9 ‘‘(A) IN GENERAL.—The Director of NIH 10 shall build upon the network created by the 11 U01 Clinical Trial Readiness Research Project 12 to identify and support the development of ap- 13 proximately 6 to 10 clinical and research par- 14 ticipation centers to facilitate medical research 15 to develop a cure for CCM and enhance the 16 medical care of individuals with CCM, in part- 17 nership with the coordinating centers under 18 paragraph (2) and other national and inter- 19 national entities, as appropriate. 20 ‘‘(B) ELIGIBILITY.—To qualify for selec- 21 tion as a participation center under subpara- 22 graph (A), an entity shall— 23 ‘‘(i) at the time of selection— 24 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00009 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 10 •HR 3573 IH ‘‘(I) be affiliated with an estab- 1 lished research network of the Na- 2 tional Institutes of Health; and 3 ‘‘(II) have the potential to par- 4 ticipate in a multisite clinical drug 5 trial with respect to CCM; 6 ‘‘(ii) demonstrate— 7 ‘‘(I) an institutional infrastruc- 8 ture capable of hosting a clinical trial 9 site and facilitating translational 10 projects and collaborations for clinical 11 trials; 12 ‘‘(II) the capacity to maintain 13 communication with other major CCM 14 research and care institutions inter- 15 nationally for information sharing and 16 coordination of research activities, es- 17 pecially through health information 18 technology; and 19 ‘‘(III) clinical expertise in CCM 20 management or complete the CCM 21 clinical training program under sub- 22 section (c)(4); and 23 ‘‘(iii) have a sufficient number of eli- 24 gible patients with CCM. 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 11 •HR 3573 IH ‘‘(C) DURATION OF SUPPORT.—The Direc- 1 tor of NIH may provide support for participa- 2 tion centers under this section for a period not 3 to exceed 5 years. The Director of NIH may ex- 4 tend the period of support for a center for one 5 or more additional periods, not to exceed an ad- 6 ditional 5 years, if the operations of such center 7 have been reviewed by an appropriate technical 8 and scientific peer review group established by 9 the Director of NIH and if such group has rec- 10 ommended to the Director that such period 11 should be extended. 12 ‘‘(c) CEREBRAL CAVERNOUS MALFORMATIONS CON- 13 SORTIUM.— 14 ‘‘(1) IN GENERAL.—The Director of NIH shall 15 build upon the network created by the U01 Clinical 16 Trial Readiness Research Project to convene a Cere- 17 bral Cavernous Malformations Research Consortium 18 (referred to in this section as the ‘consortium’). 19 ‘‘(2) MEMBERSHIP.—The consortium— 20 ‘‘(A) shall include representatives of— 21 ‘‘(i) the coordinating centers selected 22 under subsection (b)(2); and 23 ‘‘(ii) at least 1 national CCM patient 24 advocacy organization, which may be an 25 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 12 •HR 3573 IH entity that receives a grant or contract 1 under subsection (b)(2)(A); and 2 ‘‘(B) may include representatives of the 3 National Institutes of Health or the Food and 4 Drug Administration, in an advisory or ex offi- 5 cio role. 6 ‘‘(3) RESPONSIBILITIES.—Through a consensus 7 based decisionmaking model, the consortium shall 8 divide assignments and be responsible for— 9 ‘‘(A) developing and implementing training 10 programs for clinicians and scientists in accord- 11 ance with paragraph (4); 12 ‘‘(B) developing patient education, out- 13 reach, and awareness programs and materials, 14 which may be tailored for specific regional 15 needs at coordinating centers, including— 16 ‘‘(i) a regional multimedia public 17 awareness campaign; 18 ‘‘(ii) patient education materials for 19 distribution by regional physician and sur- 20 geon offices; 21 ‘‘(iii) an education program for ele- 22 mentary and secondary school nurses to fa- 23 cilitate early detection and diagnosis of 24 VerDate Sep 11 2014 23:48 Jul 09, 2019 Jkt 089200 PO 00000 Frm 00012 Fmt 6652 Sfmt 6201 E:\BILLS\H3573.IH H3573 pamtmann on DSKBFK8HB2PROD with BILLS 13 •HR 3573 IH CCM in areas in which there is a high den- 1 sity of cases of CCM; 2 ‘‘(iv) regular regional patient and 3 family oriented educational conferences; 4 and 5 ‘‘(v) nationally relevant electronic 6 health teaching and communication tools 7 and a network of professional capacity and 8 patient and family support; and 9 ‘‘(C) preparing a biannual report to Con- 10 gress, in accorda [Text truncated for display. 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