I 115th CONGRESS 2d Session H. R. 5115 IN THE HOUSE OF REPRESENTATIVES February 27, 2018 Mr. Carson of Indiana (for himself and Mr. Costello of Pennsylvania ) introduced the following bill; which was referred to the Committee on Energy and Commerce A BILL To expand and improve the programs and activities of the Department of Health and Human Services for awareness, education, research, surveillance, diagnosis, and treatment concerning rare diseases and conditions.

1. Short title This Act may be cited as the Rare disease Advancement, Research, and Education Act of 2018 or the RARE Act of 2018 . 2. Improving programs and activities of HHS concerning rare diseases and conditions (a) NIH Paragraph (1) of section 402A(a) of the Public Health Service Act ( 42 U.S.C. 282a(a) ) is amended by adding at the end the following: In addition to the amounts authorized to be appropriated by the preceding sentence, there is authorized to be appropriated $10,000,000 for each of fiscal years 2019 through 2023 for carrying out section 481A (relating to rare disease regional centers of excellence). . (b) CDC surveillance of rare diseases and conditions Title III of the Public Health Service Act is amended by inserting after section 317T of such Act ( 42 U.S.C. 247b–22 ) the following: 317U CDC surveillance of rare diseases and conditions (a) In General The Secretary, acting through the Director of the Centers for Disease Control and Prevention and in coordination with other agencies as the Secretary determines, shall, as appropriate— (1) enhance and expand infrastructure and activities to track the epidemiology of rare diseases and conditions; and (2) incorporate information obtained through such activities into an integrated surveillance system, which may consist of or include a registry, to be known as the National Rare Disease or Condition Surveillance System. (b) Research The Secretary shall ensure that the National Rare Disease or Condition Surveillance System is designed in a manner that facilitates further research on rare diseases and conditions. (c) Content In carrying out subsection (a), the Secretary— (1) shall provide for the collection and storage of information on the incidence and prevalence of rare diseases and conditions in the United States; (2) to the extent practicable, shall provide for the collection and storage of other available information on rare diseases and conditions, including information related to persons living with a rare disease or condition who choose to participate, such as— (A) demographics, such as age, race, ethnicity, sex, geographic location, family history, and other information, as appropriate; (B) risk factors that may be associated with a rare disease or condition, such as genetic and environmental risk factors and other information, as appropriate; and (C) diagnosis and progression markers; (3) to the extent practicable, shall provide for the collection and storage of information relevant to analysis on rare diseases and conditions, such as information concerning— (A) the natural history of the diseases and conditions; (B) the prevention of the diseases and conditions; (C) the detection, management, and treatment approaches for the diseases and conditions; and (D) the development of outcomes measures; (4) may address issues identified during the consultation process under subsection (d); and (5) initially may address a limited number of rare diseases or conditions. (d) Consultation In carrying out this section, the Secretary shall consult with individuals with appropriate expertise, which may include— (1) epidemiologists with experience in disease surveillance or registries; (2) representatives of national voluntary health associations that— (A) focus on rare diseases or conditions; and (B) have demonstrated experience in research, care, or patient services; (3) health information technology experts or other information management specialists; (4) clinicians with expertise in rare diseases or conditions; (5) research scientists with expertise in rare diseases or conditions, or experience conducting translational research or utilizing surveillance systems for scientific research purposes; and (6) patients and caregivers of patients with rare diseases or conditions. (e) Grants The Secretary may award grants to, or enter into contracts or cooperative agreements with, public or private nonprofit entities to carry out activities under this section. (f) Coordination with other Federal, State, and local agencies Subject to subsection (h), the Secretary shall— (1) make information and analysis in the National Rare Disease or Condition Surveillance System available, as appropriate— (A) to Federal departments and agencies, such as the National Institutes of Health and the Department of Veterans Affairs; and (B) to State and local agencies; and (2) identify, build upon, leverage, and coordinate among existing data and surveillance systems, surveys, registries, and other Federal public health infrastructure, wherever practicable. (g) Public access Subject to subsection (h), the Secretary shall ensure that information and analysis in the National Rare Disease or Conditions Surveillance System are available, as appropriate, to the public, including researchers. (h) Privacy The Secretary shall ensure that information and analysis in the National Rare Disease or Condition Surveillance System are made available only to the extent permitted by applicable Federal and State law, and in a manner that protects personal privacy, to the extent required by applicable Federal and State privacy law, at a minimum. (i) Reports (1) Report on information and analyses Not later than 1 year after the date on which any system is established under this section, the Secretary shall submit an interim 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 regarding aggregate information collected pursuant to this section and epidemiological analyses, as appropriate. Such report shall be posted on the Internet website of the Department of Health and Human Services and shall be updated biennially. (2) Implementation report Not later than 4 years after the date of the enactment of this section, the Secretary shall submit a report to the Congress concerning the implementation of this section. Such report shall include information on— (A) the development and maintenance of the National Rare Disease or Condition Surveillance System; (B) the type of information collected and stored in the surveillance system; (C) the use and availability of such information, including guidelines for such use; and (D) the use and coordination of databases that collect or maintain information on rare diseases or conditions. (j) Definitions In this section: (1) National voluntary health association The term national voluntary health association means a national nonprofit organization with chapters, other affiliated organizations, or networks in States throughout the United States with experience serving the population of individuals with a rare disease or condition and have demonstrated experience in rare disease or condition research, care, and patient services. (2) Rare The term rare , with respect to a disease or condition, means affecting fewer than 200,000 individuals in the United States. (k) Authorization of Appropriations To carry out this section, there are authorized to be appropriated $5,000,000 for each of fiscal years 2018 through 2023. . (c) AHRQ health care provider awareness activities (1) In general The Director of the Agency for Healthcare Research and Quality shall expand and intensify the activities of the Agency to increase the awareness and knowledge of health care providers about rare diseases and conditions. (2) Definition In this subsection, the term rare diseases and conditions refers to any disease or condition affecting fewer than 200,000 individuals in the United States. (d) National Academies report (1) In general The Secretary of Health and Human Services shall seek to enter into an arrangement with the National Academies (or another appropriate entity if the National Academies decline) to update and republish, by not later than 3 years after the date of enactment of this Act, the 2010 report of the National Academies entitled Rare Diseases and Orphan Products: Accelerating Research and Development . (2) Authorization of appropriations To carry out this subsection, there is authorized to be appropriated $1,000,000.