,i #aacth ,,congress #ast ,session ,h4 ,r4 #beba ,,in ,,the ,,house ,,of ,,representatives ,june #bf1 #bjac ,mr4 ,ben ,ray ,lujn of ,new ,mexico 7for himself1 ,ms4 ,michelle ,lujan ,grisham of ,new ,mexico1 and ,mr4 ,pearce7 introduced the following bill2 which was referred to the ,committee on ,energy and ,commerce ,a ,,bill ,to amend the ,public ,health ,service ,act to expand and intensify programs of the ,national ,institutes of ,health and the ,centers for ,disease ,control and ,prevention with respect to translational research and related activities concerning cavernous angioma1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,cavernous ,angioma ,research ,resource ,act of #bjac4 #b4 ,findings ,congress makes the following findings3 7#a7 ,cavernous angioma1 also termed cerebral cavernous malformations or ,,ccm1 affects an estimated #a1ejj1jjj people in the ,united ,states4 7#b7 ,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 cord4 7#c7 ,detection of cavernous angioma lesions is achieved through costly and specialized medical imaging techniques4 ,these techniques are often not readily available where patients live1 and require sedation for children and disabled adults4 7#d7 ,cavernous angioma is a common type of vascular anomaly1 but individuals may not be aware that they have the disease until the onset of serious clinical symptoms4 ,in the genetic forms1 they may not be aware that it may be passed on to their children4 7#e7 ,individuals diagnosed with cavernous angioma may experience neurological deficits1 seizure1 stroke1 or sudden death4 7#f7 ,due to limited research with respect to cavernous angioma1 there is no treatment regimen for the disease other than brain and spinal surgery4 7#g7 ,some individuals with cavernous angioma are not candidates for brain surgery4 ,no alternative treatment option is available for such individuals4 7#h7 ,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 care4 7#i7 ,due to the presence of a specific disease-causing mutation1 termed the common ,hispanic mutation that has passed through as many as #ag generations of ,americans descended from the original ,spanish settlers of the ,southwest in the #aeijs1 ,new ,mexico has the highest population density of cavernous angioma in the world4 ,cavernous angioma affects thousands of individuals in ,new ,mexico and with ancestry in ,new ,mexico4 7#aj7 ,other ,states with high rates of cavernous angioma due to the common ,hispanic ,mutation include ,texas1 ,arizona1 and ,colorado4 7#aa7 ,to address the public health threat posed by cavernous angioma in ,new ,mexico and throughout the ,united ,states1 there is a need to identify institutions capable of running clinical trial for this debilitating brain disorder4 #c4 ,cavernous angioma research activities ,part ,b of title ,,iv of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bhd et seq47 is amended by adding at the end the following3 #dji,k4 ,cavernous angioma research activities 7a7 ,expansion1 ,intensification1 and ,coordination of ,activities ,the ,director of ,,nih1 acting through the director of the ,national ,institute of ,neurological ,disorders and ,stroke1 shall expand and intensify programs of the ,national ,institutes of ,health or may award grants and cooperative agreements to public or nonprofit private entities 7including ,state health departments1 political subdivisions of ,states1 universities1 and other educational entities7 for research and related activities concerning cavernous angioma4 7b7 ,activities ,in expanding and intensifying programs under subsection 7a71 the ,director of ,,nih may carry out the following3 7#a7 ,basic1 translational1 and clinical research ,conduct or financially support basic1 clinical1 and translational research on cavernous angioma1 including research on the following3 7,a7 ,proteomic1 pharmacological1 and cell biological analysis of the cerebral cavernous malformations 7referred to in this section as the ,,ccm7 molecules4 7,b7 ,continued development and expansion of novel animal models for cavernous angioma preclinical research4 7,c7 ,early detection1 diagnosis1 and treatment of cavernous angioma4 7,d7 ,biological mechanisms for lesion genesis1 development1 and maturation4 7,e7 ,biological mechanisms for lesion bleeding and symptomology4 7,f7 ,novel biomedical and pharmacological interventions designed to prohibit new lesion development1 lesion growth1 and lesion bleeding4 7,g7 ,contributions of genetic variation to clinical presentation as targets for therapy4 7,h7 ,identification and development of biomarkers to measure phenotypic variation4 7,i7 ,research related to improving the quality of life for individuals with cavernous angioma and their families4 7,j7 ,clinical training programs aimed at increasing the number of scientists and clinicians who are trained to treat patients and carry out these research directions4 7#b7 ,facilitation of research resources2 clinical trial preparedness 7,a7 ,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 angioma4 ,such coordinating center shall provide a model for additional trial sites1 facilitate medical research to develop a cure for cavernous angioma1 and enhance the medical care of individuals with cavernous angioma nationwide4 ,such coordinating center shall-- 7i7 have an institutional infrastructure that is capable of hosting a clinical trial site and facilitating translational projects and collaborations for clinical trials2 7ii7 have the capacity to maintain programs dedicated to patient education1 patient outreach1 and awareness1 including-- 7,i7 launching a national multimedia public awareness campaign2 7,,ii7 creating and distributing patient education materials for distribution by national physician and surgeon offices2 7,,iii7 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 density2 7,,iv7 coordinating regular patient and family-oriented educational conferences2 and 7,v7 developing nationally relevant electronic health teaching and communication tools and a network of professional capacity and patient and family support2 7iii7 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 activities2 7iv7 have demonstrated clinical expertise in cavernous angioma management2 7v7 have a sufficient number of eligible patients for participation with particular focus on unique subpopulations including ,common ,hispanic ,mutation and ,,ccm#c gene mutation carriers2 and 7vi7 have a telehealth infrastructure to support and to provide clinical consultation for remote and underserved communities4 7,b7 ,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 angioma4 ,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 7,a7 and other national and international centers4 ,such participation centers shall-- 7i7 have an institutional infrastructure capable of hosting a clinical trial site and facilitating translational projects and collaborations for clinical trials2 7ii7 have the capacity to maintain communication with other major cavernous angioma research and care institutions internationally for information sharing and coordination of research activities2 7iii7 have demonstrated clinical expertise in cavernous angioma management2 and 7iv7 have a sufficient numbers of eligible patients for participation with particular focus on unique subpopulations including ,common ,hispanic ,mutation and ,,ccm#c gene mutation carriers as these unique populations may provide insight to other genetic and non-genetic forms of the illness4 7c7 ,training program for clinicians and scientists 7#a7 ,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 angioma4 7#b7 ,research resources ,in carrying out this subsection1 the ,director of ,,nih may-- 7,a7 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 section2 7,b7 take into consideration the availability of other research resources2 7,c7 encourage the use of research resources for research on1 and development of1 therapies and treatments for individuals with cavernous angioma2 and 7,d7 encourage the inclusion of individuals with cavernous angioma in clinical trials conducted or supported by the ,national ,institutes of ,health4 7#c7 ,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 communication1 minimizing duplication of effort1 and integrating the varied perspectives of partner agencies1 organizations1 and individuals4 ,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 7b77#b77;,a74 4 #d4 ,centers for ,disease ,control and ,prevention cavernous angioma surveillance and research programs ,part ,b of title ,,iii of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bdc et seq47 is amended by inserting after section #cag,t the following3 #cag,u4 ,cavernous angioma surveillance and research programs 7a7 ,in general ,the ,secretary1 acting through the ,director of the ,centers for ,disease ,control and ,prevention1 may award grants and cooperative agreements to public or nonprofit private entities 7including ,state health departments1 political subdivisions of ,states1 universities1 and other educational entities7 for the collection1 analysis1 and reporting of data on cavernous angioma4 ,in making such awards1 the ,secretary may provide direct technical assistance1 including personnel support1 in lieu of cash4 7b7 ,national ,cavernous ,angioma ,epidemiology ,program 7#a7 ,grants ,the ,secretary1 acting through the ,director of the ,centers for ,disease ,control and ,prevention1 may award grants to public or nonprofit private entities 7including ,state health departments1 political subdivisions of ,states1 universities1 and other educational entities7 for the purpose of carrying out epidemiological activities regarding cavernous angioma1 including collecting and analyzing information on the number1 incidence1 correlates1 and symptoms of cases and the clinical utility 7including costs and benefits7 of specific practice patterns4 ,in making such awards1 the ,secretary may provide direct technical assistance1 including personnel support1 in lieu of cash4 7#b7 ,national surveillance program ,in carrying out subsection 7a71 the ,secretary shall-- 7,a7 provide for a national surveillance program2 and 7,b7 where possible1 ensure that the surveillance program is coordinated with the data and sample collection activities of the ,national ,institutes of ,health under section #dji,k4 4 #e4 ,food and ,drug ,administration cavernous angioma clinical trial preparedness and support program 7a7 ,investigational new drug application ,the ,commissioner of ,food and ,drugs shall work with clinical centers1 investigators1 and advocates to support appropriate investigational new drug application under section #eje7;i7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act in an effort to hasten the pace of clinical trials for cavernous angioma4 7b7 ,orphan product development ,where applicable in rare subpopulations of cavernous angioma requiring unique pharmacological intervention1 including those with the ,common ,hispanic ,mutation or ,,ccm#c gene mutations1 the ,commissioner of ,food and ,drugs shall support appropriate requests for designations of drugs as orphan drugs under section #ebf of the ,federal ,food1 ,drug1 and ,cosmetic ,act4 #f4 ,report to congress ,not later than ,january #a1 #bjae1 and each ,january #a thereafter1 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 ,act4