text: 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.
target: 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.
feat_title: Cavernous Angioma Research Resource Act of 2013