USC Subpart 1

in effect

42 U.S.C. § 287 National Center for Advancing Translational Sciences

(a) PurposeThe purpose of the National Center for Advancing Translational Sciences (in this subpart referred to as the “Center”) is to advance translational sciences, including by—(1) coordinating and developing resources that leverage basic research in support of translational science; and (2) developing partnerships and working cooperatively to foster synergy in ways that do not create duplication, redundancy, and competition with industry activities. (b) Clinical trial activities(1) In generalThe Center may develop and provide infrastructure and resources for all phases of clinical trials research. Except as provided in paragraph (2), the Center may support clinical trials only through the end of phase IIB. (2) ExceptionThe Center may support clinical trial activities through the end of phase III for a treatment for a rare disease or condition (as defined in section 360bb of title 21) so long as—(A) the Center gives public notice for a period of at least 120 days of the Center’s intention to support the clinical trial activities in phase III; (B) no public or private organization provides credible written intent to the Center that the organization has timely plans to further the clinical trial activities or conduct clinical trials of a similar nature beyond phase IIB; and (C) the Center ensures that support of the clinical trial activities in phase III will not increase the Federal Government’s liability beyond the award value of the Center’s support. (c) Biennial reportThe Center shall publish a report on a biennial basis that, with respect to all research supported by the Center, includes a complete list of—(1) the molecules being studied; (2) clinical trial activities being conducted; (3) the methods and tools in development; (4) ongoing partnerships, including—(A) the rationale for each partnership; (B) the status of each partnership; (C) the funding provided by the Center to other entities pursuant to each partnership, and (D) the activities which have been transferred to industry pursuant to each partnership; (5) known research activity of other entities that is or will expand upon research activity of the Center; (6) the methods and tools, if any, that have been developed since the last biennial report was prepared; and (7) the methods and tools, if any, that have been developed and are being utilized by the Food and Drug Administration to support medical product reviews. (d) Inclusion of listThe first biennial report submitted under this section after December 13, 2016, shall include a complete list of all of the methods and tools, if any, which have been developed by research supported by the Center. (e) Rule of constructionNothing in this section shall be construed as authorizing the Secretary to disclose any information that is a trade secret, or other privileged or confidential information subject to section 552(b)(4) of title 5 or section 1905 of title 18.

42 U.S.C. § 287a Cures Acceleration Network

(a) DefinitionsIn this section:(1) Biological productThe term “biological product” has the meaning given such term in section 262 of this title. (2) Drug; deviceThe terms “drug” and “device” have the meanings given such terms in section 321 of title 21. (3) High need cureThe term “high need cure” means a drug (as that term is defined by section 321(g)(1) of title 21,11 So in original. A closing parenthesis probably should precede the comma. biological product (as that term is defined by section 262(i) 22 See References in Text note below. of this title), or device (as that term is defined by section 321(h) of title 21) that, in the determination of the Director of the Center—(A) is a priority to diagnose, mitigate, prevent, or treat harm from any disease or condition; and (B) for which the incentives of the commercial market are unlikely to result in its adequate or timely development. (4) Medical productThe term “medical product” means a drug, device, biological product, or product that is a combination of drugs, devices, and biological products. (b) Establishment of the Cures Acceleration NetworkSubject to the appropriation of funds as described in subsection (g), there is established within the Center a program to be known as the Cures Acceleration Network (referred to in this section as “CAN”), which shall—(1) be under the direction of the Director of the Center, taking into account the recommendations of a CAN Review Board (referred to in this section as the “Board”), described in subsection (d); and (2) award grants and contracts to eligible entities, as described in subsection (e), to accelerate the development of high need cures, including through the development of medical products and behavioral therapies. (c) FunctionsThe functions of the CAN are to—(1) conduct and support revolutionary advances in basic research, translating scientific discoveries from bench to bedside; (2) award grants and contracts to eligible entities to accelerate the development of high need cures; (3) provide the resources necessary for government agencies, independent investigators, research organizations, biotechnology companies, academic research institutions, and other entities to develop high need cures; (4) reduce the barriers between laboratory discoveries and clinical trials for new therapies; and (5) facilitate review in the Food and Drug Administration for the high need cures funded by the CAN, through activities that may include—(A) the facilitation of regular and ongoing communication with the Food and Drug Administration regarding the status of activities conducted under this section; (B) ensuring that such activities are coordinated with the approval requirements of the Food and Drug Administration, with the goal of expediting the development and approval of countermeasures and products; and (C) connecting interested persons with additional technical assistance made available under section 360bbb–4 of title 21. (d) CAN Board(1) EstablishmentThere is established a Cures Acceleration Network Review Board (referred to in this section as the “Board”), which shall advise the Director of the Center on the conduct of the activities of the Cures Acceleration Network. (2) Membership(A) In general(i) AppointmentThe Board shall be comprised of 24 members who are appointed by the Secretary and who serve at the pleasure of the Secretary. (ii) Chairperson and Vice ChairpersonThe Secretary shall designate, from among the 24 members appointed under clause (i), one Chairperson of the Board (referred to in this section as the “Chairperson”) and one Vice Chairperson. (B) Terms(i) In generalEach member shall be appointed to serve a 4-year term, except that any member appointed to fill a vacancy occurring prior to the expiration of the term for which the member’s predecessor was appointed shall be appointed for the remainder of such term. (ii) Consecutive appointments; maximum termsA member may be appointed to serve not more than 3 terms on the Board, and may not serve more than 2 such terms consecutively. (C) Qualifications(i) In generalThe Secretary shall appoint individuals to the Board based solely upon the individual’s established record of distinguished service in one of the areas of expertise described in clause (ii). Each individual appointed to the Board shall be of distinguished achievement and have a broad range of disciplinary interests. (ii) ExpertiseThe Secretary shall select individuals based upon the following requirements:(I) For each of the fields of—(aa) basic research; (bb) medicine; (cc) biopharmaceuticals; (dd) discovery and delivery of medical products; (ee) bioinformatics and gene therapy; (ff) medical instrumentation; and (gg) regulatory review and approval of medical products, the Secretary shall select at least 1 individual who is eminent in such fields. (II) At least 4 individuals shall be recognized leaders in professional venture capital or private equity organizations and have demonstrated experience in private equity investing. (III) At least 8 individuals shall represent disease advocacy organizations. (3) Ex-officio members(A) AppointmentIn addition to the 24 Board members described in paragraph (2), the Secretary shall appoint as ex-officio members of the Board—(i) a representative of the National Institutes of Health, recommended by the Secretary of the Department of Health and Human Services; (ii) a representative of the Office of the Assistant Secretary of Defense for Health Affairs, recommended by the Secretary of Defense; (iii) a representative of the Office of the Under Secretary for Health for the Veterans Health Administration, recommended by the Secretary of Veterans Affairs; (iv) a representative of the National Science Foundation, recommended by the Chair of the National Science Board; and (v) a representative of the Food and Drug Administration, recommended by the Commissioner of Food and Drugs. (B) TermsEach ex-officio member shall serve a 3-year term on the Board, except that the Chairperson may adjust the terms of the initial ex-officio members in order to provide for a staggered term of appointment for all such members. (4) Responsibilities of the Board and the Director of the Center(A) Responsibilities of the Board(i) In generalThe Board shall advise, and provide recommendations to, the Director of the Center with respect to—(I) policies, programs, and procedures for carrying out the duties of the Director of the Center under this section; and (II) significant barriers to successful translation of basic science into clinical application (including issues under the purview of other agencies and departments). (ii) ReportIn the case that the Board identifies a significant barrier, as described in clause (i)(II), the Board shall submit to the Secretary a report regarding such barrier. (B) Responsibilities of the Director of the CenterWith respect to each recommendation provided by the Board under subparagraph (A)(i), the Director of the Center shall respond in writing to the Board, indicating whether such Director will implement such recommendation. In the case that the Director of the Center indicates a recommendation of the Board will not be implemented, such Director shall provide an explanation of the reasons for not implementing such recommendation. (5) Meetings(A) In generalThe Board shall meet 4 times per calendar year, at the call of the Chairperson. (B) Quorum; requirements; limitations(i) QuorumA quorum shall consist of a total of 13 members of the Board, excluding ex-officio members, with diverse representation as described in clause (iii). (ii) Chairperson or Vice ChairpersonEach meeting of the Board shall be attended by either the Chairperson or the Vice Chairperson. (iii) Diverse representationAt each meeting of the Board, there shall be not less than one scientist, one representative of a disease advocacy organization, and one representative of a professional venture capital or private equity organization. (6) Compensation and travel expenses(A) CompensationMembers shall receive compensation at a rate to be fixed by the Chairperson but not to exceed a rate equal to the daily equivalent of the annual rate of basic pay prescribed for level IV of the Executive Schedule under section 5315 of title 5 for each day (including travel time) during which the member is engaged in the performance of the duties of the Board. All members of the Board who are officers or employees of the United States shall serve without compensation in addition to that received for their services as officers or employees of the United States. (B) Travel expensesMembers of the Board shall be allowed travel expenses, including per diem in lieu of subsistence, at rates authorized for persons employed intermittently by the Federal Government under section 5703(b) 33 So in original. Section 5703 of title 5 does not contain a subsec. (b). of title 5, while away from their homes or regular places of business in the performance of services for the Board. (e) Grant program(1) Supporting innovationTo carry out the purposes described in this section, the Director of the Center shall award contracts, grants, or cooperative agreements to the entities described in paragraph (2), to—(A) promote innovation in technologies supporting the advanced research and development and production of high need cures, including through the development of medical products and behavioral therapies. (B) accelerate the development of high need cures, including through the development of medical products, behavioral therapies, and biomarkers that demonstrate the safety or effectiveness of medical products; or (C) help the award recipient establish protocols that comply with Food and Drug Administration standards and otherwise permit the recipient to meet regulatory requirements at all stages of development, manufacturing, review, approval, and safety surveillance of a medical product. (2) Eligible entitiesTo receive assistance under paragraph (1), an entity shall—(A) be a public or private entity, which may include a private or public research institution, an institution of higher education, a medical center, a biotechnology company, a pharmaceutical company, a disease advocacy organization, a patient advocacy organization, or an academic research institution; (B) submit an application containing—(i) a detailed description of the project for which the entity seeks such grant or contract; (ii) a timetable for such project; (iii) an assurance that the entity will submit—(I) interim reports describing the entity’s—(aa) progress in carrying out the project; and (bb) compliance with all provisions of this section and conditions of receipt of such grant or contract; and (II) a final report at the conclusion of the grant period, describing the outcomes of the project; and (iv) a description of the protocols the entity will follow to comply with Food and Drug Administration standards and regulatory requirements at all stages of development, manufacturing, review, approval, and safety surveillance of a medical product; and (C) provide such additional information as the Director of the Center may require. (3) Awards(A) The cures acceleration partnership awards(i) Initial award amountEach award under this subparagraph shall be not more than $15,000,000 per project for the first fiscal year for which the project is funded, which shall be payable in one payment. (ii) Funding in subsequent fiscal yearsAn eligible entity receiving an award under clause (i) may apply for additional funding for such project by submitting to the Director of the Center the information required under subparagraphs (B) and (C) of paragraph (2). The Director may fund a project of such eligible entity in an amount not to exceed $15,000,000 for a fiscal year subsequent to the initial award under clause (i). (iii) Matching fundsAs a condition for receiving an award under this subsection, an eligible entity shall contribute to the project non-Federal funds in the amount of $1 for every $3 awarded under clauses (i) and (ii), except that the Director of the Center may waive or modify such matching requirement in any case where the Director determines that the goals and objectives of this section cannot adequately be carried out unless such requirement is waived. (B) The cures acceleration grant awards(i) Initial award amountEach award under this subparagraph shall be not more than $15,000,000 per project for the first fiscal year for which the project is funded, which shall be payable in one payment. (ii) Funding in subsequent fiscal yearsAn eligible entity receiving an award under clause (i) may apply for additional funding for such project by submitting to the Board the information required under subparagraphs (B) and (C) of paragraph (2). The Director of the Center may fund a project of such eligible entity in an amount not to exceed $15,000,000 for a fiscal year subsequent to the initial award under clause (i). (C) The cures acceleration flexible research awardsIf the Director of the Center determines that the goals and objectives of this section cannot adequately be carried out through a contract, grant, or cooperative agreement, the Director of the Center shall have flexible research authority to use other transactions to fund projects in accordance with the terms and conditions of this section. Awards made under such flexible research authority for a fiscal year shall not exceed 20 percent of the total funds appropriated under subsection (g)(1) for such fiscal year. (4) Suspension of awards for defaults, noncompliance with provisions and plans, and diversion of funds; repayment of fundsThe Director of the Center may suspend the award to any entity upon noncompliance by such entity with provisions and plans under this section or diversion of funds. (5) AuditsThe Director of the Center may enter into agreements with other entities to conduct periodic audits of the projects funded by grants or contracts awarded under this subsection. (6) Closeout proceduresAt the end of a grant or contract period, a recipient shall follow the closeout procedures under section 74.71 of title 45, Code of Federal Regulations (or any successor regulation). (7) ReviewA determination by the Director of the Center as to whether a drug, device, or biological product is a high need cure (for purposes of subsection (a)(3)) shall not be subject to judicial review. (f) Competitive basis of awardsAny grant, cooperative agreement, or contract awarded under this section shall be awarded on a competitive basis. (g) Authorization of appropriations(1) In generalFor purposes of carrying out this section, there are authorized to be appropriated $500,000,000 for fiscal year 2010, and such sums as may be necessary for subsequent fiscal years. Funds appropriated under this section shall be available until expended. (2) Limitation on use of funds otherwise appropriatedNo funds appropriated under this chapter, other than funds appropriated under paragraph (1), may be allocated to the Cures Acceleration Network.

42 U.S.C. § 287a–1 Office of Rare Diseases

(a) EstablishmentThere is established within the Center an office to be known as the Office of Rare Diseases (in this section referred to as the “Office”), which shall be headed by a Director (in this section referred to as the “Director”), appointed by the Director of the Center. (b) Duties(1) In generalThe Director of the Office shall carry out the following:(A) The Director shall recommend an agenda for conducting and supporting research on rare diseases through the national research institutes and centers. The agenda shall provide for a broad range of research and education activities, including scientific workshops and symposia to identify research opportunities for rare diseases. (B) The Director shall, with respect to rare diseases, promote coordination and cooperation among the national research institutes and centers and entities whose research is supported by such institutes. (C) The Director, in collaboration with the directors of the other relevant institutes and centers of the National Institutes of Health, may enter into cooperative agreements with and make grants for regional centers of excellence on rare diseases in accordance with section 287a–2 of this title. (D) The Director shall promote the sufficient allocation of the resources of the National Institutes of Health for conducting and supporting research on rare diseases. (E) The Director shall promote and encourage the establishment of a centralized clearinghouse for rare and genetic disease information that will provide understandable information about these diseases to the public, medical professionals, patients and families. (2) Principal advisor regarding orphan diseasesWith respect to rare diseases, the Director shall serve as the principal advisor to the Director of NIH and shall provide advice to other relevant agencies. The Director shall provide liaison with national and international patient, health and scientific organizations concerned with rare diseases. (c) DefinitionFor purposes of this section, the term “rare disease” means any disease or condition that affects less than 200,000 persons in the United States.

42 U.S.C. § 287a–2 Rare disease regional centers of excellence

(a) Cooperative agreements and grants(1) In generalThe Director of the Office of Rare Diseases (in this section referred to as the “Director”), in collaboration with the directors of the other relevant institutes and centers of the National Institutes of Health, may enter into cooperative agreements with and make grants to public or private nonprofit entities to pay all or part of the cost of planning, establishing, or strengthening, and providing basic operating support for regional centers of excellence for clinical research into, training in, and demonstration of diagnostic, prevention, control, and treatment methods for rare diseases. (2) PoliciesA cooperative agreement or grant under paragraph (1) shall be entered into in accordance with policies established by the Director of NIH. (b) Coordination with other institutesThe Director shall coordinate the activities under this section with similar activities conducted by other national research institutes, centers and agencies of the National Institutes of Health and by the Food and Drug Administration to the extent that such institutes, centers and agencies have responsibilities that are related to rare diseases. (c) Uses for Federal payments under cooperative agreements or grantsFederal payments made under a cooperative agreement or grant under subsection (a) may be used for—(1) staffing, administrative, and other basic operating costs, including such patient care costs as are required for research; (2) clinical training, including training for allied health professionals, continuing education for health professionals and allied health professions personnel, and information programs for the public with respect to rare diseases; and (3) clinical research and demonstration programs. (d) Period of support; additional periodsSupport of a center under subsection (a) may be for a period of not to exceed 5 years. Such period may be extended by the Director for additional periods of not more than 5 years if the operations of such center have been reviewed by an appropriate technical and scientific peer review group established by the Director and if such group has recommended to the Director that such period should be extended.

42 U.S.C. § 287a–4 General clinical research centers

(a) GrantsThe Director of the Center shall award grants for the establishment of general clinical research centers to provide the infrastructure for clinical research including clinical research training and career enhancement. Such centers shall support clinical studies and career development in all settings of the hospital or academic medical center involved. (b) ActivitiesIn carrying out subsection (a), the Director of National Institutes of Health shall expand the activities of the general clinical research centers through the increased use of telecommunications and telemedicine initiatives.