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
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.
This Act may be cited as the
Rare disease Advancement, Research, and Education Act of 2018 or the
RARE Act of 2018.
Improving programs and activities of HHS concerning rare diseases and conditions
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).
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:
CDC surveillance of rare diseases and conditions
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—
enhance and expand infrastructure and activities to track the epidemiology of rare diseases and conditions; and
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.
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.
In carrying out subsection (a), the Secretary—
shall provide for the collection and storage of information on the incidence and prevalence of rare diseases and conditions in the United States;
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—
demographics, such as age, race, ethnicity, sex, geographic location, family history, and other information, as appropriate;
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
diagnosis and progression markers;
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—
the natural history of the diseases and conditions;
the prevention of the diseases and conditions;
the detection, management, and treatment approaches for the diseases and conditions; and
the development of outcomes measures;
may address issues identified during the consultation process under subsection (d); and
initially may address a limited number of rare diseases or conditions.
In carrying out this section, the Secretary shall consult with individuals with appropriate expertise, which may include—
epidemiologists with experience in disease surveillance or registries;
representatives of national voluntary health associations that—
focus on rare diseases or conditions; and
have demonstrated experience in research, care, or patient services;
health information technology experts or other information management specialists;
clinicians with expertise in rare diseases or conditions;
research scientists with expertise in rare diseases or conditions, or experience conducting translational research or utilizing surveillance systems for scientific research purposes; and
patients and caregivers of patients with rare diseases or conditions.
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.
Coordination with other Federal, State, and local agencies
Subject to subsection (h), the Secretary shall—
make information and analysis in the National Rare Disease or Condition Surveillance System available, as appropriate—
to Federal departments and agencies, such as the National Institutes of Health and the Department of Veterans Affairs; and
to State and local agencies; and
identify, build upon, leverage, and coordinate among existing data and surveillance systems, surveys, registries, and other Federal public health infrastructure, wherever practicable.
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.
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.
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.
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—
the development and maintenance of the National Rare Disease or Condition Surveillance System;
the type of information collected and stored in the surveillance system;
the use and availability of such information, including guidelines for such use; and
the use and coordination of databases that collect or maintain information on rare diseases or conditions.
In this section:
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.
The term rare, with respect to a disease or condition, means affecting fewer than 200,000 individuals in the United States.
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.
AHRQ health care provider awareness activities
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.
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.
National Academies report
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.
Authorization of appropriations
To carry out this subsection, there is authorized to be appropriated $1,000,000.