I 

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. LUJA ́N, 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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Rare disease Advance5 ment, surveillance Research, and Education Act of 2019’’ 

6 or the ‘‘RARE Act of 2019’’. 


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1 SEC. 2. NIH RARE DISEASE REGIONAL CENTERS OF EXCEL2 LENCE. 

3 Paragraph (1) of section 402A(a) of the Public 

4 Health Service Act (42 U.S.C. 282a(a)) is amended by 

5 adding at the end the following: ‘‘In addition to the 

6 amounts authorized to be appropriated by the preceding 

7 sentence, there are authorized to be appropriated such 

8 sums as may be necessary for each of fiscal years 2020 

9 through 2024 for carrying out section 481A (relating to 

10 rare disease regional centers of excellence).’’. 

11 SEC. 3. CDC SURVEILLANCE OF RARE DISEASES AND CON12 DITIONS. 

13 Title III of the Public Health Service Act is amended 

14 by inserting after section 317T of such Act (42 U.S.C. 

15 247b–22) the following: 

16 ‘‘SEC. 317U. CDC SURVEILLANCE OF RARE DISEASES AND 

17 CONDITIONS. 

18 ‘‘(a) IN GENERAL.—The Secretary may, as appro19 priate— 

20 ‘‘(1) enhance and expand infrastructure and ac21 tivities to track the epidemiology of up to 4 rare dis22 eases and conditions selected under subsection 

23 (c)(1); and 

24 ‘‘(2) incorporate information obtained through 

25 such activities into an integrated surveillance system 


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1 to be known as the National Rare Disease or Condi2 tion Surveillance System. 

3 ‘‘(b) RESEARCH.—The Secretary shall ensure that 

4 the National Rare Disease or Condition Surveillance Sys5 tem is designed in a manner that provides information 

6 that facilitates further research on rare diseases and con7 ditions. 

8 ‘‘(c) CONTENT.—In carrying out subsection (a), the 

9 Secretary— 

10 ‘‘(1) shall select up to 4 rare diseases and con11 ditions that are determined by the Secretary to 

12 have— 

13 ‘‘(A) a high rate of mortality or morbidity; 

14 or 

15 ‘‘(B) potential for meaningful research and 

16 treatment; 

17 ‘‘(2) shall provide for the collection and storage 

18 of information on the incidence and prevalence of 

19 such rare diseases and conditions in the United 

20 States; 

21 ‘‘(3) to the extent practicable, shall provide for 

22 the collection and storage of other available informa23 tion on such rare diseases and conditions, including 

24 information related to persons living with such a 

25 rare disease or condition who choose to participate 


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1 in surveillance activities, and including information 

2 on— 

3 ‘‘(A) demographics, such as age, race, eth4 nicity, sex, geographic location, family history, 

5 and other information, as appropriate; 

6 ‘‘(B) risk factors that may be associated 

7 with the rare disease or condition, such as ge8 netic and environmental risk factors and other 

9 information, as appropriate; and 

10 ‘‘(C) diagnosis and progression markers; 

11 ‘‘(4) to the extent practicable, shall provide for 

12 the collection and storage of information relevant to 

13 analysis on such rare diseases and conditions, such 

14 as information concerning— 

15 ‘‘(A) the natural history of the diseases 

16 and conditions; 

17 ‘‘(B) the prevention of the disease or con18 dition, including secondary diseases and condi19 tions; 

20 ‘‘(C) the detection, management, and 

21 treatment approaches for the diseases and con22 ditions; and 

23 ‘‘(D) the development of outcomes meas24 ures; and 


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1 ‘‘(5) may address issues identified during the 

2 consultation process under subsection (d). 

3 ‘‘(d) CONSULTATION.—In carrying out this section, 

4 the Secretary shall consult with individuals with appro5 priate expertise, which may include— 

6 ‘‘(1) epidemiologists with experience in disease 

7 surveillance; 

8 ‘‘(2) representatives of national voluntary 

9 health associations that— 

10 ‘‘(A) focus on rare diseases or conditions; 

11 and 

12 ‘‘(B) have demonstrated experience in re13 search, care, or patient services; 

14 ‘‘(3) health information technology experts or 

15 other information management specialists; 

16 ‘‘(4) clinicians with expertise in rare diseases or 

17 conditions; 

18 ‘‘(5) research scientists with expertise in rare 

19 diseases or conditions, or experience conducting 

20 translational research or utilizing surveillance sys21 tems for scientific research purposes; and 

22 ‘‘(6) patients and caregivers of patients with 

23 rare diseases or conditions. 

24 ‘‘(e) GRANTS.—The Secretary may award grants to, 

25 or enter into contracts or cooperative agreements with, 


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1 public or private nonprofit entities to carry out activities 

2 under this section. 

3 ‘‘(f) COORDINATION WITH OTHER FEDERAL, STATE, 

4 AND LOCAL AGENCIES.—Subject to subsection (h), the 

5 Secretary shall— 

6 ‘‘(1) make information and analysis in the Na7 tional Rare Disease or Condition Surveillance Sys8 tem available, as appropriate— 

9 ‘‘(A) to Federal departments and agencies, 

10 such as the National Institutes of Health and 

11 the Department of Veterans Affairs; and 

12 ‘‘(B) to State and local agencies; and 

13 ‘‘(2) identify, build upon, leverage, and coordi14 nate among existing data and surveillance systems, 

15 surveys, registries, and other Federal public health 

16 infrastructure, wherever practicable. 

17 ‘‘(g) PUBLIC ACCESS.—Subject to subsection (h), the 

18 Secretary shall ensure that information and analysis in the 

19 National Rare Disease or Conditions Surveillance System 

20 are available, as appropriate, to the public, including re21 searchers. 

22 ‘‘(h) PRIVACY.—The Secretary shall ensure that in23 formation and analysis in the National Rare Disease or 

24 Condition Surveillance System are made available only to 

25 the extent permitted by applicable Federal and State law, 


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1 and in a manner that protects personal privacy, to the ex2 tent required by applicable Federal and State privacy law, 

3 at a minimum. 

4 ‘‘(i) SUPPLEMENT NOT SUPPLANT.—The activities 

5 under this section may supplement, but shall not supplant, 

6 any activities with respect to spina bifida, muscular dys7 trophy, or fragile X syndrome that are ongoing as of the 

8 date of enactment of this section. 

9 ‘‘(j) REPORTS.— 

10 ‘‘(1) REPORT ON INFORMATION AND ANAL11 YSES.—Not later than 2 years after the date on 

12 which any system is established under this section, 

13 the Secretary shall submit an interim report to the 

14 Committee on Health, Education, Labor, and Pen15 sions of the Senate and the Committee on Energy 

16 and Commerce of the House of Representatives re17 garding aggregate information collected pursuant to 

18 this section and epidemiological analyses, as appro19 priate. Such report shall be posted on the Internet 

20 website of the Department of Health and Human 

21 Services and shall be updated biennially. 

22 ‘‘(2) IMPLEMENTATION REPORT.—Not later 

23 than 4 years after the date of the enactment of this 

24 section, the Secretary shall submit a report to the 


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1 Congress concerning the implementation of this sec2 tion. Such report shall include information on— 

3 ‘‘(A) the development and maintenance of 

4 the National Rare Disease or Condition Surveil5 lance System; 

6 ‘‘(B) the type of information collected and 

7 stored in the surveillance system; 

8 ‘‘(C) the use and availability of such infor9 mation, including guidelines for such use; and 

10 ‘‘(D) the use and coordination of databases 

11 that collect or maintain information on rare dis12 eases or conditions. 

13 ‘‘(k) DEFINITIONS.—In this section: 

14 ‘‘(1) NATIONAL VOLUNTARY HEALTH ASSOCIA15 TION.—The term ‘national voluntary health associa16 tion’ means a national nonprofit organization with 

17 chapters, other affiliated organizations, or networks 

18 in States throughout the United States with experi19 ence serving the population of individuals with a 

20 rare disease or condition and have demonstrated ex21 perience in rare disease or condition research, care, 

22 and patient services. 

23 ‘‘(2) RARE.—The term ‘rare’, with respect to a 

24 disease or condition, means having a prevalence of 

25 fewer than 200,000 individuals in the United States. 


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1 ‘‘(l) AUTHORIZATION OF APPROPRIATIONS.—To 

2 carry out this section, there are authorized to be appro3 priated $10,000,000 for each of fiscal years 2020 through 

4 2025.’’. 

5 SEC. 4. INCREASING HEALTH PROFESSIONALS’ AWARENESS 

6 OF RARE DISEASES. 

7 (a) IN GENERAL.—The Director of the Agency for 

8 Healthcare Research and Quality shall expand and inten9 sify the activities of the Agency to increase the awareness 

10 and knowledge of health care providers about rare diseases 

11 and conditions. 

12 (b) DEFINITION.—In this section, the term ‘‘rare dis13 eases and conditions’’, with respect to a disease or condi14 tion, means having a prevalence of fewer than 200,000 

15 individuals in the United States. 

16 SEC. 5. REPORT. 

17 (a) IN GENERAL.—The Secretary of Health and 

18 Human Services shall seek to enter into an arrangement 

19 with the National Academies (or another appropriate enti20 ty if the National Academies decline) to update and repub21 lish, by not later than 3 years after the date of enactment 

22 of this Act, the 2010 report of the National Academies 

23 entitled ‘‘Rare Diseases and Orphan Products: Accel24 erating Research and Development’’. 


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1 (b) AUTHORIZATION OF APPROPRIATIONS.—To carry 

2 out this section, there is authorized to be appropriated 

3 $1,000,000. 

Æ 


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