[Congressional Bills 116th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4228 Introduced in House (IH)]

<DOC>






116th CONGRESS
  1st Session
                                H. R. 4228

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.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           September 6, 2019

    Mr. Carson of Indiana (for himself, Mr. Hudson, Ms. Brownley of 
California, Mr. Bucshon, Mr. DeFazio, Mr. Deutch, Mr. Fitzpatrick, Ms. 
Jackson Lee, Mr. Lujan, Mrs. Carolyn B. Maloney of New York, Ms. Moore, 
 Mrs. Murphy, Ms. Norton, Mr. Raskin, Mr. Rouda, Mr. Suozzi, and Mrs. 
  Walorski) 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.

    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 ``Rare disease Advancement, 
surveillance Research, and Education Act of 2019'' or the ``RARE Act of 
2019''.

SEC. 2. NIH RARE DISEASE REGIONAL CENTERS OF EXCELLENCE.

    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 are authorized to be appropriated such sums as may be 
necessary for each of fiscal years 2020 through 2024 for carrying out 
section 481A (relating to rare disease regional centers of 
excellence).''.

SEC. 3. 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:

``SEC. 317U. CDC SURVEILLANCE OF RARE DISEASES AND CONDITIONS.

    ``(a) In General.--The Secretary may, as appropriate--
            ``(1) enhance and expand infrastructure and activities to 
        track the epidemiology of up to 4 rare diseases and conditions 
        selected under subsection (c)(1); and
            ``(2) incorporate information obtained through such 
        activities into an integrated surveillance system 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 
provides information that facilitates further research on rare diseases 
and conditions.
    ``(c) Content.--In carrying out subsection (a), the Secretary--
            ``(1) shall select up to 4 rare diseases and conditions 
        that are determined by the Secretary to have--
                    ``(A) a high rate of mortality or morbidity; or
                    ``(B) potential for meaningful research and 
                treatment;
            ``(2) shall provide for the collection and storage of 
        information on the incidence and prevalence of such rare 
        diseases and conditions in the United States;
            ``(3) to the extent practicable, shall provide for the 
        collection and storage of other available information on such 
        rare diseases and conditions, including information related to 
        persons living with such a rare disease or condition who choose 
        to participate in surveillance activities, and including 
        information on--
                    ``(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 the 
                rare disease or condition, such as genetic and 
                environmental risk factors and other information, as 
                appropriate; and
                    ``(C) diagnosis and progression markers;
            ``(4) to the extent practicable, shall provide for the 
        collection and storage of information relevant to analysis on 
        such rare diseases and conditions, such as information 
        concerning--
                    ``(A) the natural history of the diseases and 
                conditions;
                    ``(B) the prevention of the disease or condition, 
                including secondary diseases and conditions;
                    ``(C) the detection, management, and treatment 
                approaches for the diseases and conditions; and
                    ``(D) the development of outcomes measures; and
            ``(5) may address issues identified during the consultation 
        process under subsection (d).
    ``(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;
            ``(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) Supplement Not Supplant.--The activities under this section 
may supplement, but shall not supplant, any activities with respect to 
spina bifida, muscular dystrophy, or fragile X syndrome that are 
ongoing as of the date of enactment of this section.
    ``(j) Reports.--
            ``(1) Report on information and analyses.--Not later than 2 
        years 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.
    ``(k) 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 having a prevalence of fewer than 200,000 
        individuals in the United States.
    ``(l) Authorization of Appropriations.--To carry out this section, 
there are authorized to be appropriated $10,000,000 for each of fiscal 
years 2020 through 2025.''.

SEC. 4. INCREASING HEALTH PROFESSIONALS' AWARENESS OF RARE DISEASES.

    (a) 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.
    (b) Definition.--In this section, the term ``rare diseases and 
conditions'', with respect to a disease or condition, means having a 
prevalence of fewer than 200,000 individuals in the United States.

SEC. 5. REPORT.

    (a) 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''.
    (b) Authorization of Appropriations.--To carry out this section, 
there is authorized to be appropriated $1,000,000.
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