II Calendar No. 282 113th CONGRESS 1st Session S. 1417 IN THE SENATE OF THE UNITED STATES August 1, 2013 Mrs. Hagan (for herself, Mr. Hatch , Mr. Enzi , Mr. Casey , Ms. Collins , Mr. Schumer , Mr. Cochran , Mr. Bennet , Ms. Cantwell , Mr. Donnelly , Mr. Murphy , Mr. Schatz , Mr. Franken , Mr. Harkin , and Mr. Wicker ) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions December 19, 2013 Reported by Mr. Harkin , with an amendment Strike out all after the enacting clause and insert the part printed in italic A BILL To amend the Public Health Service Act to reauthorize programs under part A of title XI of such Act.

1. Short title; table of contents (a) Short title This Act may be cited as the Newborn Screening Saves Lives Reauthorization Act of 2013 . (b) Table of contents The table of contents of this Act is as follows: Sec. 1. Short title; table of contents. Sec. 2. Improved newborn and child screening and follow-up for heritable disorders. Sec. 3. Evaluating the effectiveness of newborn and child screening and follow-up programs. Sec. 4. Advisory committee on heritable disorders in newborns and children. Sec. 5. Clearinghouse of Newborn Screening Information. Sec. 6. Laboratory quality. Sec. 7. Interagency Coordinating Committee on Newborn and Child Screening. Sec. 8. National contingency plan for newborn screening. Sec. 9. Hunter Kelly Research Program. Sec. 10. Authorization of appropriations. 2. Improved newborn and child screening and follow-up for heritable disorders Section 1109 of the Public Health Service Act ( 42 U.S.C. 300b–8 ) is amended— (1) in subsection (a)— (A) in the matter preceding paragraph (1), by striking and in consultation with the Advisory Committee and inserting and taking into consideration the recommendations of the Advisory Committee ; (B) in paragraph (2), by striking screening and training and inserting screening, counseling, and training ; and (C) in paragraph (4), by striking treatment and inserting follow-up and treatment ; (2) in subsection (h), by striking subsection (c)(2) each place that such appears and inserting subsection (c) ; and (3) by striking subsection (j) (relating to authorization of appropriations). 3. Evaluating the effectiveness of newborn and child screening and follow-up programs Section 1110 of the Public Health Service Act ( 42 U.S.C. 300b–9 ) is amended— (1) in the section heading, by inserting and follow-up after child screening ; (2) in subsection (a), by inserting follow-up, after the effectiveness of screening, ; (3) in subsection (b)— (A) in paragraph (1), by inserting ascertainment, treatment, after the effectiveness of screening, counseling, ; (B) in paragraph (2)— (i) by inserting ascertainment, treatment, after the effectiveness of screening, counseling, ; and (ii) by striking or at the end; (C) in paragraph (3), by striking the period at the end and inserting a semicolon; and (D) by adding at the end the following: (4) the availability and effectiveness of follow-up care for newborns and their families after screening and diagnosis; (5) the costs and effectiveness of newborn screening, evaluation and intervention programs, and surveillance systems conducted by State-based programs in order to answer issues of importance to State and national policymakers; (6) the effectiveness of newborn screening follow-up and intervention programs through the assessment of the health and development of children at school age and as young adults; (7) the level of data sharing regarding newborn screening with State-based birth defects and developmental disabilities monitoring programs; (8) the coordination of surveillance activities, including— (A) standardized data collection and reporting; and (B) use of electronic health records; (9) the facilitation of quality improvement in treatment and disease management based on gaps in services or care identified by longitudinal tracking systems; and (10) the utilization of data from longitudinal tracking systems to support the development and evaluation of evidence-based guidelines for diagnosis, treatment, and disease management. ; and (4) by striking subsection (d) (relating to authorization of appropriations). 4. Advisory committee on heritable disorders in newborns and children Section 1111 of the Public Health Service Act ( 42 U.S.C. 300b–10 ) is amended— (1) in subsection (b)— (A) in paragraph (4), by striking public health impact and inserting public health impact, including the cost ; and (B) in paragraph (6)— (i) in subparagraph (A), by striking achieve rapid diagnosis and inserting achieve best practices in rapid diagnosis and appropriate treatment ; (ii) in subparagraph (D), by inserting before the semicolon , including information on cost and incidence ; (iii) in subparagraph (J), by striking and at the end; and (iv) in subparagraph (K), by striking the period and inserting ; and ; (2) in subsection (d)— (A) in paragraph (1), by striking 180 and inserting 60 ; (B) in paragraph (2), by striking Newborn Screening Saves Lives Act of 2008 and insert Newborn Screening Saves Lives Reauthorization Act of 2013 ; and (C) by adding at the end the following: (4) Priority review (A) In general For each nomination to the recommended uniform screening panel that meets the criteria of subparagraph (B), the Advisory Committee on Heritable Disorders in Newborns and Children shall provide a priority review as described in this paragraph and subject to the deadlines in subparagraph (C). (B) Criteria for priority review The Advisory Committee on Heritable Disorders in Newborns and Children shall provide a priority review with respect to a nomination to the recommended uniform screening panel that meets each of the following criteria: (i) A human drug application, as defined in section 735(1) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 379g(1) ), has been submitted to the Secretary for treatment of such disease or condition. (ii) The application described in clause (i) has been granted priority review or has received fast track or breakthrough therapy designations by the Secretary as described in section 529(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(a)(1)). (C) Deadlines for priority review The Advisory Committee on Heritable Disorders in Newborns and Children shall vote on the nominated condition that is under priority review within 8 months of the later of— (i) the date on which the Advisory Committee refers the nomination to the condition review workgroup; or (ii) the date of the approval, by the Food and Drug Administration, of the treatment associated with the nomination to the recommended uniform screening panel. (D) Failure to act If the Secretary fails to act on a recommendation within the time period provided for in paragraph (1), the Secretary shall adopt the recommendation and include the screening recommended by the Advisory Committee on Heritable Disorders in Newborns and Children in the recommended uniform screening panel. (E) Other reviews For each nomination to the recommended uniform screening panel that does not meet the criteria of subparagraph (B), the Advisory Committee on Heritable Disorders in Newborns and Children shall provide for a review and vote on the nominated condition that is under review within 10 months of the date on which the Advisory Committee referred the nomination to the condition review workgroup. ; (3) by redesignating subsections (f) and (g) as subsections (g) and (h), respectively; (4) by inserting after subsection (e) the following new subsection: (f) Meetings The Advisory Committee shall meet at least 4 times each calendar year with at least 2 meetings in person. ; and (5) by striking subsection (h) (relating to authorization of appropriations), as redesignated by paragraph (2). 5. Clearinghouse of Newborn Screening Information Section 1112 of the Public Health Service Act ( 42 U.S.C. 300b–11 ) is amended— (1) in subsection (a)— (A) in paragraph (2), by striking ; and and inserting a semicolon; (B) in paragraph (3), by striking the period at the end and inserting a semicolon; and (C) by adding at the end the following new paragraphs: (4) maintain current data on the number of conditions for which screening is conducted in each State; and (5) disseminate available evidence-informed guidelines related to diagnosis, counseling, and treatment with respect to conditions detected by newborn screening. ; (2) in subsection (b)(4)(D), by striking Newborn Screening Saves Lives Act of 2008 and inserting Newborn Screening Saves Lives Reauthorization Act of 2013 ; and (3) by striking subsection (d) (relating to authorization of appropriations). 6. Laboratory quality Section 1113 of the Public Health Service Act ( 42 U.S.C. 300b–12 ) is amended— (1) in subsection (a)— (A) by striking the subsection enumerator and heading; and (B) by striking and in consultation with the Advisory Committee and inserting and taking into consideration the recommendations of the Advisory Committee ; and (2) by striking subsection (b) (relating to authorization of appropriations). 7. Interagency Coordinating Committee on Newborn and Child Screening Section 1114 of the Public Health Service Act ( 42 U.S.C. 300b–13 ) is amended— (1) in subsection (c), by striking the Administrator, the Director of the Agency for Healthcare Research and Quality and inserting the Administrator of the Health Resources and Services Administration, the Director of the Agency for Healthcare Research and Quality, the Commissioner of Food and Drugs, ; and (2) by striking subsection (e) (relating to authorization of appropriations) and inserting the following: (e) Report to congress (1) In general The Secretary shall— (A) not later than 1 year after the date of enactment of the Newborn Screening Saves Lives Reauthorization Act of 2013, submit to the Health, Education, Labor, and Pensions Committee of the Senate and the Energy and Commerce Committee of the House of Representatives a report on activities related to— (i) newborn screening; and (ii) screening children who have or are at risk for heritable disorders; and (B) not less than every 2 years, shall submit to such committees an updated version of such report. (2) Contents The report submitted under subsection (a) shall contain a description of— (A) the implementation of sections 1111 through 1116B, including this section; and (B) the amounts expended on such implementation. . 8. National contingency plan for newborn screening Section 1115(a) of the Public Health Service Act ( 42 U.S.C. 300b–14(a) ) is amended by adding at the end the following: The plan shall be updated as needed and at least every five years. . 9. Hunter Kelly Research Program Section 1116(a)(1) of the Public Health Service Act ( 42 U.S.C. 300b–15(a)(1) ) is amended— (1) in subparagraph (B), by striking ; and and inserting a semicolon; (2) by redesignating subparagraph (C) as subparagraph (E); and (3) by inserting after subparagraph (B) the following: (C) providing research and data for newborn conditions under review by the Advisory Committee on Heritable Disorders in Newborns and Children to be added to the recommended uniform screening panel; (D) conducting pilot studies on conditions recommended by the Advisory Committee on Heritable Disorders in Newborns and Children to ensure that screenings are ready for nationwide implementation; and . 10. Authorization of appropriations Part A of title XI of the Public Health Service Act is amended by adding at the end, the following: 1117. Authorization of appropriations for newborn screening programs and activities There are authorized to be appropriated— (1) to carry out sections 1109, 1110, 1111, and 1112, $18,334,000 for each of fiscal years 2014 through 2018; and (2) to carry out section 1113, $7,500,000 for each of fiscal years 2014 through 2018. .

1. Short title; table of contents (a) Short title This Act may be cited as the Newborn Screening Saves Lives Reauthorization Act of 2013 . (b) Table of contents The table of contents of this Act is as follows: Sec. 1. Short title; table of contents. Sec. 2. Improved newborn and child screening and follow-up for heritable disorders. Sec. 3. Evaluating the effectiveness of newborn and child screening and follow-up programs. Sec. 4. Advisory Committee on Heritable Disorders in Newborns and Children. Sec. 5. Clearinghouse of Newborn Screening Information. Sec. 6. Laboratory quality and surveillance. Sec. 7. Interagency Coordinating Committee on Newborn and Child Screening. Sec. 8. National contingency plan for newborn screening. Sec. 9. Hunter Kelly Research Program. Sec. 10. Authorization of appropriations. Sec. 11. Reports to Congress 2. Improved newborn and child screening and follow-up for heritable disorders Section 1109 of the Public Health Service Act ( 42 U.S.C. 300b–8 ) is amended— (1) in subsection (a)— (A) in the matter preceding paragraph (1)— (i) by striking subsection (j) and inserting section 1117 ; and (ii) by striking and in consultation with the Advisory Committee and inserting and taking into consideration the expertise of the Advisory Committee ; (B) in paragraph (2), by striking screening and training and inserting screening, counseling, and training ; (C) in paragraph (3), by striking and at the end; (D) in paragraph (4)— (i) by striking treatment and inserting follow-up and treatment ; and (ii) by striking the period and inserting ; and ; and (E) by adding at the end the following: (5) to improve the timely collection, delivery, receipt, and screening of specimens, and the timely diagnosis of heritable disorders in newborns. ; (2) in subsection (h), by striking subsection (c)(2) each place that such appears and inserting subsection (c) ; and (3) by striking subsection (j) (relating to authorization of appropriations). 3. Evaluating the effectiveness of newborn and child screening and follow-up programs Section 1110 of the Public Health Service Act ( 42 U.S.C. 300b–9 ) is amended— (1) in the section heading, by inserting and follow-up after child screening ; (2) in subsection (a), by striking of screening, and inserting , including with respect to timeliness, of screening, follow-up, ; (3) in subsection (b)— (A) in paragraph (1)— (i) by striking counseling, testing and inserting treatment, counseling, testing, follow-up, ; and (ii) by inserting before the semicolon the following: , including, as appropriate, through the assessment of health and development outcomes for such children through adolescence ; (B) in paragraph (2)— (i) by striking counseling, testing and inserting treatment, counseling, testing, follow-up, ; and (ii) by striking or at the end; (C) in paragraph (3), by striking the period at the end and inserting a semicolon; and (D) by adding at the end the following: (4) methods that may be identified to improve quality in the diagnosis, treatment, and disease management of heritable disorders based on gaps in services or care; or (5) methods or best practices by which the eligible entities described in section 1109 can achieve the timely collection, delivery, receipt, and screening of newborn screening specimens, and the timely diagnosis of heritable disorders in newborns. ; and (4) by striking subsection (d) (relating to authorization of appropriations). 4. Advisory committee on heritable disorders in newborns and children Section 1111 of the Public Health Service Act ( 42 U.S.C. 300b–10 ) is amended— (1) in subsection (b)— (A) by redesignating paragraphs (4) through (6) as paragraphs (5) through (7), respectively; (B) by inserting after paragraph (3), the following: (4) provide technical assistance, as appropriate, to individuals and organizations regarding the submission of nominations to the uniform screening panel, including prior to the submission of such nominations; ; (C) in paragraph (5) (as so redesignated), by inserting , including the cost after public health impact ; and (D) in paragraph (7) (as so redesignated)— (i) in subparagraph (A), by striking achieve rapid diagnosis and inserting achieve best practices in rapid diagnosis and appropriate treatment ; (ii) in subparagraph (D), by inserting before the semicolon , including information on cost and incidence ; (iii) in subparagraph (J), by striking and at the end; (iv) in subparagraph (K), by striking the period and inserting ; and ; and (v) by adding at the end the following: (L) the timely collection, delivery, receipt, and screening of specimens to be tested for heritable disorders in newborns in order to ensure rapid diagnosis and follow-up. ; (2) in subsection (d)— (A) in paragraph (1)— (i) by striking 180 and inserting 120 ; and (ii) by adding at the end the following: If the Secretary is unable to make a determination to adopt or reject such recommendation within such 120-day period, the Secretary shall notify the Advisory Committee and the appropriate committees of Congress of such determination together with an explanation for why the Secretary was unable to comply within such 120-day period, as well as a plan of action for consideration of such pending recommendations. ; (B) by striking paragraph (2); (C) by redesignating paragraph (3) as paragraph (2); and (D) by adding at the end the following: (3) Deadline for review For each nomination to the recommended uniform screening panel, the Advisory Committee on Heritable Disorders in Newborns and Children shall review and vote on the nominated condition within 9 months of the date on which the Advisory Committee referred the nomination to the condition review workgroup. ; (3) by redesignating subsections (f) and (g) as subsections (g) and (h), respectively; (4) by inserting after subsection (e) the following new subsection: (f) Meetings The Advisory Committee shall meet at least 4 times each calendar year, or as subject to the discretion of the Designated Federal Officer in consultation with the Chair. ; (5) in subsection (g) (as so redesignated), by striking Newborn Screening Saves Lives Act of 2008 and inserting Newborn Screening Saves Lives Reauthorization Act of 2013 ; and (6) by striking subsection (h) (relating to authorization of appropriations), as redesignated by paragraph (3). 5. Clearinghouse of Newborn Screening Information Section 1112 of the Public Health Service Act ( 42 U.S.C. 300b–11 ) is amended— (1) in subsection (a)— (A) in paragraph (2), by striking ; and and inserting a semicolon; (B) in paragraph (3)— (i) by striking data and inserting information ; and (ii) by striking the period at the end and inserting a semicolon; and (C) by adding at the end the following new paragraphs: (4) maintain current information on the number of conditions for which screening is conducted in each State; and (5) disseminate available evidence-based guidelines related to diagnosis, counseling, and treatment with respect to conditions detected by newborn screening. ; (2) in subsection (b)(4)(D), by striking Newborn Screening Saves Lives Act of 2008 and inserting Newborn Screening Saves Lives Reauthorization Act of 2013 ; (3) in subsection (c)— (A) by striking developing the clearinghouse and inserting carrying out activities ; and (B) by striking clearinghouse minimizes and inserting activities minimize ; and (4) by striking subsection (d) (relating to authorization of appropriations). 6. Laboratory quality and surveillance Section 1113 of the Public Health Service Act ( 42 U.S.C. 300b–12 ) is amended— (1) in the section heading, by inserting and surveillance before the period; (2) in subsection (a)— (A) by striking the subsection enumerator and heading; (B) in the matter preceding paragraph (1), by striking and in consultation with the Advisory Committee and inserting and taking into consideration the expertise of the Advisory Committee ; (C) in paragraph (1)— (i) by inserting timeliness for processing such tests, after newborn screening tests ; and (ii) by striking and at the end; and (D) in paragraph (2), by striking the period and inserting ; and ; and (3) by striking subsection (b) (relating to authorization of appropriations) and inserting the following: (b) Surveillance activities The Secretary, acting through the Director of the Centers for Disease Control and Prevention, and taking into consideration the expertise of the Advisory Committee on Heritable Disorders in Newborns and Children established under section 1111, may provide, as appropriate, for the coordination of surveillance activities, including— (1) through standardized data collection and reporting, as well as the use of electronic health records; and (2) by promoting data sharing regarding newborn screening with State-based birth defects and developmental disabilities monitoring programs. . 7. Interagency Coordinating Committee on Newborn and Child Screening Section 1114 of the Public Health Service Act ( 42 U.S.C. 300b–13 ) is amended— (1) in subsection (c), by striking the Administrator, the Director of the Agency for Healthcare Research and Quality and inserting the Administrator of the Health Resources and Services Administration, the Director of the Agency for Healthcare Research and Quality, the Commissioner of Food and Drugs, ; and (2) by striking subsection (e) (relating to authorization of appropriations). 8. National contingency plan for newborn screening Section 1115(a) of the Public Health Service Act ( 42 U.S.C. 300b–14(a) ) is amended by adding at the end the following: The plan shall be updated as needed and at least every five years. . 9. Hunter Kelly Research Program Section 1116(a)(1) of the Public Health Service Act ( 42 U.S.C. 300b–15(a)(1) ) is amended— (1) in subparagraph (B), by striking ; and and inserting a semicolon; (2) by redesignating subparagraph (C) as subparagraph (E); and (3) by inserting after subparagraph (B) the following: (C) by providing research findings and data for newborn conditions under review by the Advisory Committee on Heritable Disorders in Newborns and Children to be added to the recommended uniform screening panel; (D) conducting pilot studies on conditions recommended by the Advisory Committee on Heritable Disorders in Newborns and Children to ensure that screenings are ready for nationwide implementation; and . 10. Authorization of appropriations Part A of title XI of the Public Health Service Act is amended by adding at the end, the following: 1117. Authorization of appropriations for newborn screening programs and activities There are authorized to be appropriated— (1) to carry out sections 1109, 1110, 1111, and 1112, $18,334,000 for each of fiscal years 2014 through 2018; and (2) to carry out section 1113, $7,500,000 for each of fiscal years 2014 through 2018. . 11. Reports to Congress (a) GAO report on timeliness of newborn screening (1) In general Not later than 2 years after the date of enactment of this Act, the Comptroller General of the United States shall submit a report to the Committee on Health, Education, Labor and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives concerning the timeliness of screening for heritable disorders in newborns. (2) Contents The report submitted under paragraph (1) shall include the following: (A) An analysis of information regarding the timeliness of newborn screening, which may include the time elapsed from birth to specimen collection, specimen collection to receipt by laboratory, specimen receipt to reporting, reporting to follow-up testing, and follow-up testing to confirmed diagnosis. (B) A summary of any guidelines, recommendations, or best practices available to States and health care providers intended to support a timely newborn screening system. (C) An analysis of any barriers to maintaining a timely newborn screening system which may exist and recommendations for addressing such barriers. (b) Report by Secretary (1) In general The Secretary of Health and Human Services shall— (A) not later than 1 year after the date of enactment of the Newborn Screening Saves Lives Reauthorization Act of 2013 , 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 on activities related to— (i) newborn screening; and (ii) screening children who have or are at risk for heritable disorders; and (B) not less than every 2 years, shall submit to such committees an updated version of such report. (2) Contents The report submitted under this subsection shall contain a description of— (A) the ongoing activities under sections 1109, 1110, and 1112 through 1115 of the Public Health Service Act; and (B) the amounts expended on such activities.

December 19, 2013 Reported with an amendment