I

115TH CONGRESS
2D SESSION

H. R. 5115

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
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

Be it enacted by the Senate and House of Representa-

2 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 Advance-

5 ment, Research, and Education Act of 2018’’ or the
lotter on DSK3G9T082PROD with BILLS

6 ‘‘RARE Act of 2018’’.

VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00001

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 2
1

SEC. 2. IMPROVING PROGRAMS AND ACTIVITIES OF HHS

2

CONCERNING RARE DISEASES AND CONDI-

3

TIONS.

4

(a) NIH.—Paragraph (1) of section 402A(a) of the

5 Public Health Service Act (42 U.S.C. 282a(a)) is amended
6 by adding at the end the following:
7

‘‘In addition to the amounts authorized to be appro-

8

priated by the preceding sentence, there is author-

9

ized to be appropriated $10,000,000 for each of fis-

10

cal years 2019 through 2023 for carrying out sec-

11

tion 481A (relating to rare disease regional centers

12

of excellence).’’.

13

(b) CDC SURVEILLANCE

OF

RARE DISEASES

AND

14 CONDITIONS.—Title III of the Public Health Service Act
15 is amended by inserting after section 317T of such Act
16 (42 U.S.C. 247b–22) the following:
17

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

18
19

CONDITIONS.

‘‘(a) IN GENERAL.—The Secretary, acting through

20 the Director of the Centers for Disease Control and Pre21 vention and in coordination with other agencies as the Sec-

lotter on DSK3G9T082PROD with BILLS

22 retary determines, shall, as appropriate—
23

‘‘(1) enhance and expand infrastructure and ac-

24

tivities to track the epidemiology of rare diseases

25

and conditions; and

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00002

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 3
1

‘‘(2) incorporate information obtained through

2

such activities into an integrated surveillance sys-

3

tem, which may consist of or include a registry, to

4

be known as the National Rare Disease or Condition

5

Surveillance System.

6

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

7 the National Rare Disease or Condition Surveillance Sys8 tem is designed in a manner that facilitates further re9 search on rare diseases and conditions.
10

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

lotter on DSK3G9T082PROD with BILLS

11 Secretary—
12

‘‘(1) shall provide for the collection and storage

13

of information on the incidence and prevalence of

14

rare diseases and conditions in the United States;

15

‘‘(2) to the extent practicable, shall provide for

16

the collection and storage of other available informa-

17

tion on rare diseases and conditions, including infor-

18

mation related to persons living with a rare disease

19

or condition who choose to participate, such as—

20

‘‘(A) demographics, such as age, race, eth-

21

nicity, sex, geographic location, family history,

22

and other information, as appropriate;

23

‘‘(B) risk factors that may be associated

24

with a rare disease or condition, such as genetic

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00003

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 4
1

and environmental risk factors and other infor-

2

mation, as appropriate; and

3

‘‘(C) diagnosis and progression markers;

4

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

5

the collection and storage of information relevant to

6

analysis on rare diseases and conditions, such as in-

7

formation concerning—

8

‘‘(A) the natural history of the diseases

9

and conditions;

10

‘‘(B) the prevention of the diseases and

11

conditions;

12

‘‘(C) the detection, management, and

13

treatment approaches for the diseases and con-

14

ditions; and

15

‘‘(D) the development of outcomes meas-

16

ures;

17

‘‘(4) may address issues identified during the

18

consultation process under subsection (d); and

19

‘‘(5) initially may address a limited number of

20

rare diseases or conditions.

21

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

22 the Secretary shall consult with individuals with appro-

lotter on DSK3G9T082PROD with BILLS

23 priate expertise, which may include—
24
25

‘‘(1) epidemiologists with experience in disease
surveillance or registries;

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00004

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 5
1

‘‘(2)

2

representatives

of

national

voluntary

health associations that—

3

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

4

and

5

‘‘(B) have demonstrated experience in re-

6

search, care, or patient services;

7

‘‘(3) health information technology experts or

8

other information management specialists;

9

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

10

conditions;

11

‘‘(5) research scientists with expertise in rare

12

diseases or conditions, or experience conducting

13

translational research or utilizing surveillance sys-

14

tems for scientific research purposes; and

15

‘‘(6) patients and caregivers of patients with

16

rare diseases or conditions.

17

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

18 or enter into contracts or cooperative agreements with,
19 public or private nonprofit entities to carry out activities
20 under this section.
21
22

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

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

lotter on DSK3G9T082PROD with BILLS

23 Secretary shall—

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00005

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 6
1

‘‘(1) make information and analysis in the Na-

2

tional Rare Disease or Condition Surveillance Sys-

3

tem available, as appropriate—

4

‘‘(A) to Federal departments and agencies,

5

such as the National Institutes of Health and

6

the Department of Veterans Affairs; and

7

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

8

‘‘(2) identify, build upon, leverage, and coordi-

9

nate among existing data and surveillance systems,

10

surveys, registries, and other Federal public health

11

infrastructure, wherever practicable.

12

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

13 Secretary shall ensure that information and analysis in the
14 National Rare Disease or Conditions Surveillance System
15 are available, as appropriate, to the public, including re16 searchers.
17

‘‘(h) PRIVACY.—The Secretary shall ensure that in-

18 formation and analysis in the National Rare Disease or
19 Condition Surveillance System are made available only to
20 the extent permitted by applicable Federal and State law,
21 and in a manner that protects personal privacy, to the ex22 tent required by applicable Federal and State privacy law,

lotter on DSK3G9T082PROD with BILLS

23 at a minimum.
24

‘‘(i) REPORTS.—

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00006

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 7
1

‘‘(1) REPORT

2

YSES.—Not

3

which any system is established under this section,

4

the Secretary shall submit an interim report to the

5

Committee on Health, Education, Labor, and Pen-

6

sions of the Senate and the Committee on Energy

7

and Commerce of the House of Representatives re-

8

garding aggregate information collected pursuant to

9

this section and epidemiological analyses, as appro-

10

priate. Such report shall be posted on the Internet

11

website of the Department of Health and Human

12

Services and shall be updated biennially.

13

later than 1 year after the date on

‘‘(2) IMPLEMENTATION

REPORT.—Not

than 4 years after the date of the enactment of this

15

section, the Secretary shall submit a report to the

16

Congress concerning the implementation of this sec-

17

tion. Such report shall include information on—

18

‘‘(A) the development and maintenance of

19

the National Rare Disease or Condition Surveil-

20

lance System;
‘‘(B) the type of information collected and

22

stored in the surveillance system;

23

‘‘(C) the use and availability of such infor-

24

mation, including guidelines for such use; and

•HR 5115 IH
VerDate Sep 11 2014

later

14

21

lotter on DSK3G9T082PROD with BILLS

ON INFORMATION AND ANAL-

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00007

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 8
1

‘‘(D) the use and coordination of databases

2

that collect or maintain information on rare dis-

3

eases or conditions.

4

‘‘(j) DEFINITIONS.—In this section:

5

‘‘(1) NATIONAL

VOLUNTARY HEALTH ASSOCIA-

6

TION.—The

7

tion’ means a national nonprofit organization with

8

chapters, other affiliated organizations, or networks

9

in States throughout the United States with experi-

10

ence serving the population of individuals with a

11

rare disease or condition and have demonstrated ex-

12

perience in rare disease or condition research, care,

13

and patient services.

term ‘national voluntary health associa-

14

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

15

disease or condition, means affecting fewer than

16

200,000 individuals in the United States.

17

‘‘(k) AUTHORIZATION

OF

APPROPRIATIONS.—To

18 carry out this section, there are authorized to be appro19 priated $5,000,000 for each of fiscal years 2018 through
20 2023.’’.
21

(c) AHRQ HEALTH CARE PROVIDER AWARENESS

22 ACTIVITIES.—

lotter on DSK3G9T082PROD with BILLS

23

(1) IN

GENERAL.—The

Director of the Agency

24

for Healthcare Research and Quality shall expand

25

and intensify the activities of the Agency to increase

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00008

Fmt 6652

Sfmt 6201

E:\BILLS\H5115.IH

H5115

 9
1

the awareness and knowledge of health care pro-

2

viders about rare diseases and conditions.

3

(2) DEFINITION.—In this subsection, the term

4

‘‘rare diseases and conditions’’ refers to any disease

5

or condition affecting fewer than 200,000 individuals

6

in the United States.

7

(d) NATIONAL ACADEMIES REPORT.—

8

(1) IN

GENERAL.—The

Secretary of Health and

9

Human Services shall seek to enter into an arrange-

10

ment with the National Academies (or another ap-

11

propriate entity if the National Academies decline)

12

to update and republish, by not later than 3 years

13

after the date of enactment of this Act, the 2010 re-

14

port of the National Academies entitled ‘‘Rare Dis-

15

eases and Orphan Products: Accelerating Research

16

and Development’’.

17

(2) AUTHORIZATION

OF APPROPRIATIONS.—To

18

carry out this subsection, there is authorized to be

19

appropriated $1,000,000.

lotter on DSK3G9T082PROD with BILLS

Æ

•HR 5115 IH
VerDate Sep 11 2014

03:49 Mar 02, 2018

Jkt 079200

PO 00000

Frm 00009

Fmt 6652

Sfmt 6301

E:\BILLS\H5115.IH

H5115