Cavernous Angioma Research Resource Act of 2013

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Cavernous Angioma Research Resource 
Act of 2013''.

SEC. 2. FINDINGS.

    Congress makes the following findings:
            (1) Cavernous angioma, also termed ``cerebral cavernous 
        malformations'' or ``CCM'', affects an estimated 1,500,000 
        people in the United States.
            (2) Cavernous angioma is a devastating blood vessel disease 
        that is characterized by the presence of vascular lesions that 
        develop and grow within the brain and spinal cord.
            (3) Detection of cavernous angioma lesions is achieved 
        through costly and specialized medical imaging techniques. 
        These techniques are often not readily available where patients 
        live, and require sedation for children and disabled adults.
            (4) Cavernous angioma is a common type of vascular anomaly, 
        but individuals may not be aware that they have the disease 
        until the onset of serious clinical symptoms. In the genetic 
        forms, they may not be aware that it may be passed on to their 
        children.
            (5) Individuals diagnosed with cavernous angioma may 
        experience neurological deficits, seizure, stroke, or sudden 
        death.
            (6) Due to limited research with respect to cavernous 
        angioma, there is no treatment regimen for the disease other 
        than brain and spinal surgery.
            (7) Some individuals with cavernous angioma are not 
        candidates for brain surgery. No alternative treatment option 
        is available for such individuals.
            (8) There is a shortage of physicians who are familiar with 
        cavernous angioma and affected individuals may find it 
        difficult to receive timely diagnosis and appropriate care.
            (9) Due to the presence of a specific disease-causing 
        mutation, termed the ``common Hispanic mutation'' that has 
        passed through as many as 17 generations of Americans descended 
        from the original Spanish settlers of the Southwest in the 
        1590s, New Mexico has the highest population density of 
        cavernous angioma in the world. Cavernous angioma affects 
        thousands of individuals in New Mexico and with ancestry in New 
        Mexico.
            (10) Other States with high rates of cavernous angioma due 
        to the common Hispanic Mutation include Texas, Arizona, and 
        Colorado.
            (11) To address the public health threat posed by cavernous 
        angioma in New Mexico and throughout the United States, there 
        is a need to identify institutions capable of running clinical 
        trial for this debilitating brain disorder.

SEC. 3. CAVERNOUS ANGIOMA RESEARCH ACTIVITIES.

    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. CAVERNOUS ANGIOMA RESEARCH ACTIVITIES.

    ``(a) Expansion, Intensification, and Coordination of Activities.--
The Director of NIH, acting through the director of the National 
Institute of Neurological Disorders and Stroke, shall expand and 
intensify programs of the National Institutes of Health or may award 
grants and cooperative agreements to public or nonprofit private 
entities (including State health departments, political subdivisions of 
States, universities, and other educational entities) for research and 
related activities concerning cavernous angioma.
    ``(b) Activities.--In expanding and intensifying programs under 
subsection (a), the Director of NIH may carry out the following:
            ``(1) Basic, translational, and clinical research.--Conduct 
        or financially support basic, clinical, and translational 
        research on cavernous angioma, including research on the 
        following:
                    ``(A) Proteomic, pharmacological, and cell 
                biological analysis of the cerebral cavernous 
                malformations (referred to in this section as the 
                `CCM') molecules.
                    ``(B) Continued development and expansion of novel 
                animal models for cavernous angioma preclinical 
                research.
                    ``(C) Early detection, diagnosis, and treatment of 
                cavernous angioma.
                    ``(D) Biological mechanisms for lesion genesis, 
                development, and maturation.
                    ``(E) Biological mechanisms for lesion bleeding and 
                symptomology.
                    ``(F) Novel biomedical and pharmacological 
                interventions designed to prohibit new lesion 
                development, lesion growth, and lesion bleeding.
                    ``(G) Contributions of genetic variation to 
                clinical presentation as targets for therapy.
                    ``(H) Identification and development of biomarkers 
                to measure phenotypic variation.
                    ``(I) Research related to improving the quality of 
                life for individuals with cavernous angioma and their 
                families.
                    ``(J) Clinical training programs aimed at 
                increasing the number of scientists and clinicians who 
                are trained to treat patients and carry out these 
                research directions.
            ``(2) Facilitation of research resources; clinical trial 
        preparedness.--
                    ``(A) Coordination.--Identify and support the 
                development of a clinical and research coordinating 
                center with the potential of coordinating a multi-site 
                clinical drug trial for cavernous angioma. Such 
                coordinating center shall provide a model for 
                additional trial sites, facilitate medical research to 
                develop a cure for cavernous angioma, and enhance the 
                medical care of individuals with cavernous angioma 
                nationwide. Such coordinating center shall--
                            ``(i) have an institutional infrastructure 
                        that is capable of hosting a clinical trial 
                        site and facilitating translational projects 
                        and collaborations for clinical trials;
                            ``(ii) have the capacity to maintain 
                        programs dedicated to patient education, 
                        patient outreach, and awareness, including--
                                    ``(I) launching a national 
                                multimedia public awareness campaign;
                                    ``(II) creating and distributing 
                                patient education materials for 
                                distribution by national physician and 
                                surgeon offices;
                                    ``(III) establishing an education 
                                program for elementary and secondary 
                                school nurses to facilitate early 
                                detection and diagnosis of cavernous 
                                angioma in areas of high cavernous 
                                angioma population density;
                                    ``(IV) coordinating regular patient 
                                and family-oriented educational 
                                conferences; and
                                    ``(V) developing nationally 
                                relevant electronic health teaching and 
                                communication tools and a network of 
                                professional capacity and patient and 
                                family support;
                            ``(iii) have the capacity to establish and 
                        maintain communication with other major 
                        cavernous angioma research and care 
                        institutions internationally for information 
                        sharing and coordination of research 
                        activities;
                            ``(iv) have demonstrated clinical expertise 
                        in cavernous angioma management;
                            ``(v) have a sufficient number of eligible 
                        patients for participation with particular 
                        focus on unique subpopulations including Common 
                        Hispanic Mutation and CCM3 gene mutation 
                        carriers; and
                            ``(vi) have a telehealth infrastructure to 
                        support and to provide clinical consultation 
                        for remote and underserved communities.
                    ``(B) Participation.--Identify and support the 
                development of clinical and research participation 
                centers with the potential to participate in a multi-
                site clinical drug trial for cavernous angioma. Such 
                participation centers may facilitate medical research 
                to develop a cure for cavernous angioma and enhance the 
                medical care of individuals with cavernous angioma in 
                partnership with the coordinating center under 
                subparagraph (A) and other national and international 
                centers. Such participation centers shall--
                            ``(i) have an institutional infrastructure 
                        capable of hosting a clinical trial site and 
                        facilitating translational projects and 
                        collaborations for clinical trials;
                            ``(ii) have the capacity to maintain 
                        communication with other major cavernous 
                        angioma research and care institutions 
                        internationally for information sharing and 
                        coordination of research activities;
                            ``(iii) have demonstrated clinical 
                        expertise in cavernous angioma management; and
                            ``(iv) have a sufficient numbers of 
                        eligible patients for participation with 
                        particular focus on unique subpopulations 
                        including Common Hispanic Mutation and CCM3 
                        gene mutation carriers as these unique 
                        populations may provide insight to other 
                        genetic and non-genetic forms of the illness.
    ``(c) Training Program for Clinicians and Scientists.--
            ``(1) In general.--Eligible coordinating and participation 
        centers under this section shall establish or expand training 
        programs for medical and allied health clinicians and 
        scientists in clinical practice and research relevant to 
        cavernous angioma.
            ``(2) Research resources.--In carrying out this subsection, 
        the Director of NIH may--
                    ``(A) use information collected by the National 
                Institutes of Health pursuant to other provisions of 
                law or prior to the date of the enactment of this 
                section;
                    ``(B) take into consideration the availability of 
                other research resources;
                    ``(C) encourage the use of research resources for 
                research on, and development of, therapies and 
                treatments for individuals with cavernous angioma; and
                    ``(D) encourage the inclusion of individuals with 
                cavernous angioma in clinical trials conducted or 
                supported by the National Institutes of Health.
            ``(3) Cavernous angioma consortium.--The Director of NIH 
        may provide for the participation of agencies of the National 
        Institutes of Health in a consortium to facilitate the exchange 
        of information and to make the research effort on cavernous 
        angioma more efficient and effective by ensuring consistent 
        communication, minimizing duplication of effort, and 
        integrating the varied perspectives of partner agencies, 
        organizations, and individuals. Such consortium shall include 
        at least one national cavernous angioma patient advocacy 
        organization and may be the same consortium receiving a grant 
        or contract under subsection (b)(2)(A).''.

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

    ``(a) In General.--The Secretary, acting through the Director of 
the Centers for Disease Control and Prevention, may award grants and 
cooperative agreements to public or nonprofit private entities 
(including State health departments, political subdivisions of States, 
universities, and other educational entities) for the collection, 
analysis, and reporting of data on cavernous angioma. In making such 
awards, the Secretary may provide direct technical assistance, 
including personnel support, in lieu of cash.
    ``(b) National Cavernous Angioma Epidemiology Program.--
            ``(1) Grants.--The Secretary, acting through the Director 
        of the Centers for Disease Control and Prevention, may award 
        grants to public or nonprofit private entities (including State 
        health departments, political subdivisions of States, 
        universities, and other educational entities) for the purpose 
        of carrying out epidemiological activities regarding cavernous 
        angioma, including collecting and analyzing information on the 
        number, incidence, correlates, and symptoms of cases and the 
        clinical utility (including costs and benefits) of specific 
        practice patterns. In making such awards, the Secretary may 
        provide direct technical assistance, including personnel 
        support, in lieu of cash.
            ``(2) National surveillance program.--In carrying out 
        subsection (a), the Secretary shall--
                    ``(A) provide for a national surveillance program; 
                and
                    ``(B) where possible, ensure that the surveillance 
                program is coordinated with the data and sample 
                collection activities of the National Institutes of 
                Health under section 409K.''.

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

    (a) Investigational New Drug Application.--The Commissioner of Food 
and Drugs shall work with clinical centers, investigators, and 
advocates to support appropriate investigational new drug application 
under section 505(i) of the Federal Food, Drug, and Cosmetic Act in an 
effort to hasten the pace of clinical trials for cavernous angioma.
    (b) Orphan Product Development.--Where applicable in rare 
subpopulations of cavernous angioma requiring unique pharmacological 
intervention, including those with the Common Hispanic Mutation or CCM3 
gene mutations, the Commissioner of Food and Drugs shall support 
appropriate requests for designations of drugs as orphan drugs under 
section 526 of the Federal Food, Drug, and Cosmetic Act.

SEC. 6. REPORT TO CONGRESS.

    Not later than January 1, 2015, and each January 1 thereafter, the 
Secretary of Health and Human Services shall prepare and submit to the 
appropriate committees of the Congress a report concerning the 
implementation of this Act and the amendments made by this Act.

Summary: Cavernous Angioma Research Resource Act of 2013 - Amends the Public Health Service Act to require the Director of the National Institutes of Health (NIH), acting through the Director of the National Institute of Neurological Disorders and Stroke, to expand and intensify NIH programs regarding research and related activities concerning cavernous angioma. Authorizes grants and cooperative agreements to public or nonprofit private entities for such activities. Authorizes the Director of NIH to: (1) conduct basic, clinical, and translational research on cavernous angioma; (2) identify and support the development of a clinical and research coordinating center with the potential of coordinating a multi-site clinical drug trial for cavernous angioma; and (3) identify and support the development of clinical and research participation centers with the potential to participate in such a trial.  Requires coordinating and participation centers to expand training programs for medical and allied health clinicians and scientists in clinical practice and research relevant to cavernous angioma. Authorizes the Director to provide for the participation of NIH agencies in a consortium (to include at least one patient advocacy organization) to facilitate the exchange of information and increase the efficiency and effectiveness of the research effort. Authorizes the Secretary of Health and Human Services (HHS) to award grants and cooperative agreements, including technical assistance, to public or nonprofit private entities for: (1) the collection, analysis, and reporting of data on cavernous angioma; 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. Requires establishment of a national surveillance program as part of such activities. Requires the Commissioner of Food and Drugs (FDA) to: (1) work with clinical centers, investigators, and advocates to support appropriate investigational new drug applications under the Federal Food, Drug, and Cosmetic Act in order to hasten the pace of clinical trials for cavernous angioma; and (2) where applicable in rare subpopulations of cavernous angioma requiring unique pharmacological intervention, including those with the Common Hispanic Mutation or CCM3 gene mutations, support appropriate requests for designations of orphan drugs.