Title: CCM-CARE Act
Policy area: Health

Summary:
Cerebral Cavernous Malformations Clinical Awareness, Research, and Education Act of 2017 or the CCM-CARE Act
This bill amends the Public Health Service Act to require the National Institutes of Health (NIH) to strengthen and coordinate its efforts concerning cerebral cavernous malformation (CCM). (CCM is a condition in which blood vessels in the brain and spinal cord become enlarged, which can lead to seizures, paralysis, hearing or vision loss, or bleeding in the brain.) The NIH may award grants and enter into cooperative agreements for CCM research.
The NIH must: (1) award grants and contracts to plan and provide support for a network of CCM Clinical Research Centers; (2) identify and support additional centers to facilitate medical research to develop a cure for CCM and enhance medical care for CCM; and (3) convene a Cerebral Cavernous Malformations Research Consortium to develop programs for clinicians, scientists, and patients.
The Centers for Disease Control and Prevention may award grants and enter into cooperative agreements for the collection, analysis, and reporting of data on CCM.
The Department of Health and Human Services must award grants and enter into cooperative agreements for epidemiological activities related to CCM and must provide for a national CCM surveillance program.
The Food and Drug Administration must, in order to hasten clinical trials for CCM, coordinate with clinical centers, investigators, and advocates to support: (1) qualification of biomarkers, (2) qualification of patient reported outcome measures for quality of life, (3) investigational new drug applications, and (4) adaptive trial designs for rare disease research and expedited review of medications.

[Congressional Bills 115th Congress]
[From the U.S. Government Publishing Office]
[S. 475 Introduced in Senate (IS)]

<DOC>






115th CONGRESS
  1st Session
                                 S. 475

 To increase research, education, and treatment for cerebral cavernous 
                             malformations.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                           February 28, 2017

Mr. Udall (for himself and Mr. Heinrich) introduced the following bill; 
     which was read twice and referred to the Committee on Health, 
                     Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL


 
 To increase research, education, and treatment for cerebral cavernous 
                             malformations.

    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 ``Cerebral Cavernous Malformations 
Clinical Awareness, Research, and Education Act of 2017'' or the ``CCM-
CARE Act''.

SEC. 2. FINDINGS.

    Congress finds as follows:
            (1) Cerebral cavernous malformations (referred to in this 
        section as ``CCM''), also known as cavernous angioma, or 
        cavernoma, is a devastating blood vessel disease characterized 
        by vascular lesions that develop and grow within the brain and 
        spinal cord.
            (2) Detection of CCM lesions is achieved through costly and 
        specialized medical imaging techniques, often not accessible or 
        convenient to patients who need them.
            (3) While CCM is a common type of vascular anomaly, many 
        individuals are not aware they have the disease until the onset 
        of serious clinical symptoms. CCM is often inherited 
        unknowingly.
            (4) CCM affects an estimated 600,000 people in the United 
        States.
            (5) Individuals diagnosed with CCM may experience 
        neurological deficits, seizure, stroke, or sudden death.
            (6) Due to limited research, there is currently no 
        treatment for CCM other than brain and spinal surgery, and only 
        for certain patients.
            (7) There is also a shortage of trained physicians to 
        provide skilled and timely diagnosis and appropriate treatment 
        for CCM.
            (8) While the hereditary form of CCM may occur among any 
        ethnicity, the presence of a mutation called the ``common 
        Hispanic mutation'', has passed through 17 or more generations 
        of American descendants from the original Spanish settlers of 
        the Southwest in the 1590s. New Mexico has the highest 
        population density of CCM in the world; Texas, Arizona, and 
        Colorado also have high rates of CCM due to the common Hispanic 
        mutation.

SEC. 3. EXPANSION AND COORDINATION OF ACTIVITIES OF NATIONAL INSTITUTES 
              OF HEALTH WITH RESPECT TO CEREBRAL CAVERNOUS 
              MALFORMATIONS RESEARCH.

    Part B of title IV of the Public Health Service Act (42 U.S.C. 284 
et seq.) is amended by adding at the end the following:

``SEC. 409K. CEREBRAL CAVERNOUS MALFORMATIONS RESEARCH ACTIVITIES.

    ``(a) Expansion and Coordination of Activities.--The Director of 
NIH, in coordination with the directors of the National Institute of 
Neurological Disorders and Stroke, the National Center for Advancing 
Translational Sciences, the National Heart, Lung, and Blood Institute, 
and other national research institutes, as appropriate, for the purpose 
of conducting research and related activities concerning cerebral 
cavernous malformations (referred to in this section as `CCM')--
            ``(1) shall strengthen and coordinate efforts of the 
        National Institutes of Health; and
            ``(2) may award grants and cooperative agreements to public 
        or nonprofit private entities (including State health 
        departments, political subdivisions of States, universities, 
        and other medical or educational entities).
    ``(b) Activities.--The research and related activities described in 
subsection (a) shall include the following:
            ``(1) Clinical, translational, and basic research.--The 
        Director of NIH shall conduct or support, through funding 
        opportunity announcements, grants, or cooperative agreements, 
        basic, clinical, and translational research on CCM, including 
        research on--
                    ``(A) the identification and development of 
                biomarkers that fulfill the requirement of the Food and 
                Drug Administration for biomarker qualification as 
                proper measures of phenotypic variation;
                    ``(B) safety or efficacy for new or repurposed 
                currently approved drugs for CCM treatment;
                    ``(C) research related to improving the quality of 
                life for individuals with CCM and their families;
                    ``(D) contributions of genetic variation to 
                clinical presentation as targets for therapy;
                    ``(E) early detection, diagnosis, and treatment of 
                CCM;
                    ``(F) clinical training programs aimed at 
                increasing the number of scientists and clinicians who 
                are trained to treat patients and carry out the 
                research described in this paragraph;
                    ``(G) continued development and expansion of novel 
                animal models for preclinical research relating to CCM;
                    ``(H) pre-clinical and clinical research related to 
                repurposing currently approved drugs for treatment of 
                CCM;
                    ``(I) proteomic, pharmacological, and cell 
                biological analysis of CCM molecules;
                    ``(J) biological mechanisms for lesion genesis, 
                development, and maturation;
                    ``(K) biological mechanisms for lesion bleeding and 
                symptomology; and
                    ``(L) novel biomedical and pharmacological 
                interventions designed to inhibit new lesion 
                development, lesion growth, and lesion bleeding.
            ``(2) Facilitation of research resources; clinical trial 
        preparedness.--
                    ``(A) In general.--The Director of NIH shall award 
                grants and contracts to public or nonprofit private 
                entities to fund all or part of the cost of planning, 
                establishing, and providing basic operating support for 
                a network of CCM Clinical Research Centers, including 
                Coordinating and Participating centers regarding 
                research on various forms of CCM.
                    ``(B) Clinical and research coordination centers.--
                            ``(i) In general.--The Director of NIH 
                        shall identify and support the development of 2 
                        geographically distributed national clinical 
                        and research coordinating centers with unique 
                        clinical expertise and the potential for 
                        coordinating multi-site clinical drug trials 
                        with respect to CCM.
                            ``(ii) Duties.--The coordinating centers 
                        identified under clause (i) shall provide a 
                        model for the participation centers described 
                        in paragraph (3), facilitate medical research 
                        to develop a cure for CCM, and enhance the 
                        medical care of individuals with CCM 
                        nationwide, including by--
                                    ``(I) maintaining an institutional 
                                infrastructure capable of hosting 
                                clinical trials and facilitating 
                                translational research projects and 
                                collaborations for clinical trials;
                                    ``(II) implementing the programs 
                                dedicated to patient education, patient 
                                outreach, and awareness developed by 
                                the Cerebral Cavernous Malformations 
                                Consortium under subsection (c)(3)(B);
                                    ``(III) developing the capacity to 
                                establish and maintain communication 
                                with other major CCM research and care 
                                institutions internationally for 
                                information sharing and coordination of 
                                research activities;
                                    ``(IV) demonstrating clinical 
                                expertise in the management of CCM and 
                                appointing a director and support 
                                staff, including a trainee and patient 
                                representative, for CCM research 
                                programming;
                                    ``(V) treating a sufficient number 
                                of eligible patients for participation 
                                with particular focus on unique 
                                subpopulations, such as patients with 
                                the common Hispanic mutation, Ashkenazi 
                                Jewish mutation, or CCM3 gene mutation 
                                carriers; and
                                    ``(VI) maintaining a telehealth 
                                infrastructure to support and provide 
                                clinical consultation for remote and 
                                underserved communities.
            ``(3) Participation centers.--
                    ``(A) In general.--The Director of NIH shall 
                identify and support the development of approximately 6 
                to 10 clinical and research participation centers to 
                facilitate medical research to develop a cure for CCM 
                and enhance the medical care of individuals with CCM, 
                in partnership with the coordinating centers under 
                paragraph (2) and other national and international 
                entities, as appropriate.
                    ``(B) Eligibility.--To qualify for selection as a 
                participation center under subparagraph (A), an entity 
                shall--
                            ``(i) at the time of selection--
                                    ``(I) be affiliated with an 
                                established research network of the 
                                National Institutes of Health; and
                                    ``(II) have the potential to 
                                participate in a multisite clinical 
                                drug trial with respect to CCM;
                            ``(ii) demonstrate--
                                    ``(I) an institutional 
                                infrastructure capable of hosting a 
                                clinical trial site and facilitating 
                                translational projects and 
                                collaborations for clinical trials;
                                    ``(II) the capacity to maintain 
                                communication with other major CCM 
                                research and care institutions 
                                internationally for information sharing 
                                and coordination of research 
                                activities, especially through health 
                                information technology; and
                                    ``(III) clinical expertise in CCM 
                                disease management or complete the CCM 
                                clinical training program under 
                                subsection (c)(4); and
                            ``(iii) have a sufficient number of 
                        eligible patients with CCM.
                    ``(C) Duration of support.--The Director of NIH may 
                provide support for participation centers under this 
                section for a period not to exceed 5 years. The 
                Director of NIH may extend the period of support for a 
                center for one or more additional periods, not to 
                exceed an additional 5 years, if the operations of such 
                center have been reviewed by an appropriate technical 
                and scientific peer review group established by the 
                Director of NIH and if such group has recommended to 
                the Director that such period should be extended.
    ``(c) Cerebral Cavernous Malformations Consortium.--
            ``(1) In general.--The Director of NIH shall convene a 
        Cerebral Cavernous Malformations Research Consortium (referred 
        to in this section as the `consortium').
            ``(2) Membership.--The consortium--
                    ``(A) shall include representatives of--
                            ``(i) the coordinating centers selected 
                        under subsection (b)(2); and
                            ``(ii) at least 1 national CCM patient 
                        advocacy organization, which may be an entity 
                        that receives a grant or contract under 
                        subsection (b)(2)(A); and
                    ``(B) may include representatives of the National 
                Institutes of Health or the Food and Drug 
                Administration, in an advisory or ex officio role.
            ``(3) Responsibilities.--Through a consensus based 
        decisionmaking model, the consortium shall divide assignments 
        and be responsible for--
                    ``(A) developing and implementing training programs 
                for clinicians and scientists in accordance with 
                paragraph (4);
                    ``(B) developing patient education, outreach, and 
                awareness programs and materials, which may be tailored 
                for specific regional needs at coordinating centers, 
                including--
                            ``(i) a regional multimedia public 
                        awareness campaign;
                            ``(ii) patient education materials for 
                        distribution by regional physician and surgeon 
                        offices;
                            ``(iii) an education program for elementary 
                        and secondary school nurses to facilitate early 
                        detection and diagnosis of CCM in areas in 
                        which there is a high density of cases of CCM;
                            ``(iv) regular regional patient and family-
                        oriented educational conferences; and
                            ``(v) nationally relevant electronic health 
                        teaching and communication tools and a network 
                        of professional capacity and patient and family 
                        support; and
                    ``(C) preparing a biannual report to Congress, in 
                accordance with paragraph (5).
            ``(4) Training program for clinicians and scientists.--
                    ``(A) In general.--The consortium, in cooperation 
                with the coordinating centers, shall establish or 
                expand a physician training program, including 
                information and education on advances in the diagnosis 
                and treatment of CCM, and training and continuing 
                education through programs for scientists, physicians, 
                medical students, and other health professionals and 
                care coordinators who provide care for patients with 
                CCM, telehealth, and research relevant to CCM, for the 
                purpose of supporting the development of new 
                participation centers through educational programming 
                to gain the expertise needed to become clinical and 
                research participation centers with the potential to 
                participate in clinical drug trials.
                    ``(B) Stipends.--The Director of NIH may provide 
                stipends for health professionals who are enrolled in 
                the training programs described in subparagraph (A).
                    ``(C) Eligibility.--To be eligible to participate 
                in the training program, an individual shall be 
                affiliated with an entity that is in an existing 
                clinical research network of the National Institutes of 
                Health.
            ``(5) Report to congress.--The Director of NIH, on behalf 
        of the consortium, shall biennially submit to the Committee on 
        Health, Education, Labor, and Pensions of the Senate and the 
        Committee on Energy and Commerce of the House of 
        Representatives a report that describes the research, 
        education, and other activities on CCM conducted or supported 
        through the Department of Health and Human Services. Each such 
        report shall include--
                    ``(A) a research plan;
                    ``(B) provisions specifying the amounts expended by 
                the Department of Health and Human Services with 
                respect to various forms of CCM, including those 
                affected by the common Hispanic Mutation, Ashkenazi 
                Jewish mutation, CCM3 gene mutations, and other 
                familial and sporadic forms of cerebral cavernous 
                malformation; and
                    ``(C) recommendations for particular projects or 
                types of projects that the national research institutes 
                or other entities in the field of research should 
                conduct on inherited or non-inherited forms of CCM.''.

SEC. 4. CENTERS FOR DISEASE CONTROL AND PREVENTION CEREBRAL CAVERNOUS 
              MALFORMATIONS SURVEILLANCE AND RESEARCH PROGRAMS.

    Part B of title III of the Public Health Service Act (42 U.S.C. 243 
et seq.) is amended by inserting after section 317T the following:

``SEC. 317U. CEREBRAL CAVERNOUS MALFORMATIONS SURVEILLANCE AND RESEARCH 
              PROGRAMS.

    ``(a) In General.--The Secretary, acting through the Director of 
the Centers for Disease Control and Prevention, may award grants in 
such sums as may be necessary and cooperative agreements to public or 
nonprofit private entities (including State health departments, 
political subdivisions of States, universities, and other medical or 
educational entities) for the collection, analysis, and reporting of 
data on cerebral cavernous malformations (referred to in this section 
as `CCM').
    ``(b) National Cerebral Cavernous Malformations Epidemiology 
Program.--The Secretary shall award grants and cooperative agreements, 
including technical assistance, to public or nonprofit private entities 
for--
            ``(1) the collection, analysis, and reporting of data on 
        CCM; and
            ``(2) epidemiological activities, including collecting and 
        analyzing information on the number, incidence, correlates, and 
        symptoms of cases and the clinical utility of specific practice 
        patterns.
    ``(c) National Surveillance Program.--The Secretary shall--
            ``(1) provide for a national surveillance program for the 
        purpose of carrying out epidemiological activities regarding 
        CCM, including collecting and analyzing information on the 
        number, incidence, correlates, and symptoms of cases of CCM and 
        the clinical utility (including costs and benefits) of specific 
        practice patterns; and
            ``(2) wherever possible, ensure that the surveillance 
        program is coordinated with the data and sample collection 
        activities of the National Institutes of Health under section 
        409K.
    ``(d) Technical Assistance.--In making awards under this section, 
the Secretary may provide direct technical assistance, including 
personnel support.
    ``(e) Coordination With Clinical Centers.--The Secretary shall 
ensure that epidemiological information is made available to clinical 
centers as supported by the Director of the National Institutes of 
Health under section 409K.
    ``(f) Authorization of Appropriations.--There are authorized to be 
appropriated such sums as may be necessary to carry out this 
section.''.

SEC. 5. FOOD AND DRUG ADMINISTRATION CEREBRAL CAVERNOUS MALFORMATIONS 
              CLINICAL TRIAL PREPAREDNESS AND SUPPORT PROGRAM.

    (a) Biomarker Qualification Program.--The Secretary of Health and 
Human Services, acting through the Commissioner of Food and Drugs, 
shall coordinate with clinical centers, investigators, and advocates to 
support the qualification of appropriate surrogate biomarkers in an 
effort to hasten the pace of clinical trials for cerebral cavernous 
malformation.
    (b) Clinical Outcome Assessment Qualification.--The Secretary of 
Health and Human Services, acting through the Commissioner of Food and 
Drugs, shall coordinate with clinical centers, investigators, and 
advocates to support qualification of newly developed patient reported 
outcome measures for qualify of life as a clinical outcome in an effort 
to hasten the pace of clinical trials for cerebral cavernous 
malformation.
    (c) Investigational New Drug Application.--The Secretary of Health 
and Human Services, acting through the Commissioner of Food and Drugs, 
shall coordinate with clinical centers, investigators, and advocates to 
support appropriate investigational new drug applications under section 
505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) 
in an effort to hasten the pace of clinical trials for cerebral 
cavernous malformation.
    (d) Adaptive Trial Design and Expedited Review Pathways.--The 
Secretary of Health and Human Services, acting through the Commissioner 
of Food and Drugs, shall coordinate with clinical centers, 
investigators, and advocates to support appropriate adaptive trial 
designs for rare disease research and expedited review mechanisms for 
including Fast Track, Breakthrough Therapy Designation, Priority and/or 
Accelerated Review, where appropriate, in an effort to hasten the pace 
of clinical trials for cerebral cavernous malformation.
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