[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[S. 2010 Introduced in Senate (IS)]
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116th CONGRESS
1st Session
S. 2010
To increase research, education, and treatment for cerebral cavernous
malformations.
_______________________________________________________________________
IN THE SENATE OF THE UNITED STATES
June 27, 2019
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 2019'' 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 14 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.
(9) A second mutation (CCM2 Common Deletion) originating in
the Southeastern United States before 1800 has increased rates
of the illness in South Carolina, Georgia, Florida, Alabama,
Mississippi, Louisiana, Texas, Oklahoma, Kentucky, Kansas, and
northern California.
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 CCM pathogenic biology or response
to clinical intervention;
``(B) safety or efficacy for new or repurposed
currently approved drugs for CCM treatment;
``(C) research related to improving and measuring
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) preclinical and clinical research related to
repurposing currently approved drugs for CCM treatment;
``(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;
``(L) novel biomedical and pharmacological
interventions designed to inhibit new lesion
development, lesion growth, and lesion bleeding;
``(M) novel biomedical and pharmacological
interventions designed to target existing lesions to
reduce their size and clinical activity;
``(N) continued research related to understanding
better the natural history and clinical variation
associated with CCM, particularly as it relates to the
development of drug development tools and clinical
outcome assessments;
``(O) the gut-brain axis and the effects of
microbiome composition on clinical symptomology; and
``(P) the microbiome as a therapeutic target for
CCM treatment.
``(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 build upon the network created by the U01
Clinical Trial Readiness Research Project to
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, CCM2 Common Deletion,
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 build
upon the network created by the U01 Clinical Trial
Readiness Research Project to 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
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 1 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 build upon the
network created by the U01 Clinical Trial Readiness Research
Project to 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 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, CCM2 Common Deletion, 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.
``(d) Prioritize CCM Funding for Biotech.--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, shall
prioritize the provision of grant funding for small biotechnology
entities that are working to develop treatments for 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 encouraging
consistency in ICD-10 coding, 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 the qualification of newly developed patient
reported outcome measures for quality 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 peer review mechanisms
for including Orphan Drug Designation, Fast Track, Breakthrough Therapy
Designation, Priority Review or Accelerated Review, where appropriate,
in an effort to hasten the pace of clinical trials for cerebral
cavernous malformation.
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