I 
117TH CONGRESS 
1ST SESSION H. R. 1184 
To amend the Federal Food, Drug, and Cosmetic Act to improve the 
treatment of rare diseases and conditions, and for other purposes. 
IN THE HOUSE OF REPRESENTATIVES 
FEBRUARY 18, 2021 
Mr. TONKO (for himself, Mr. MCKINLEY, Mr. FITZPATRICK, and Mr. SAN 
NICOLAS) introduced the following bill; which was referred to the Com-
mittee on Energy and Commerce 
A BILL 
To amend the Federal Food, Drug, and Cosmetic Act to 
improve the treatment of rare diseases and conditions, 
and for other purposes. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Helping Experts Accel-
erate Rare Treatments Act of 2021’’. 
SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES 
AND CONDITIONS. 
(a) ANNUAL REPORT
ON ORPHAN DRUG PRO-
GRAM.—Subchapter B of chapter V of the Federal Food, 
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Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) is 
amended by adding at the end the following new section: 
‘‘SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM. 
‘‘(a) IN GENERAL.—Not later than 1 year after the 
date of enactment of the Helping Experts Accelerate Rare 
Treatments Act of 2021, and not less than annually there-
after, the Secretary shall submit to the Congress a report 
summarizing the activities of the Food and Drug Adminis-
tration for designating drugs under section 526 for a rare 
disease or condition and approving such drugs under sec-
tion 505 of this Act or licensing such drugs under section 
351 of the Public Health Service Act, including— 
‘‘(1) the number of applications for such drugs 
under sections 505 of this Act and section 351 of 
the Public Health Service Act received by the Food 
and Drug Administration, the number of such appli-
cations considered by each Food and Drug Adminis-
tration division, and the number of such applications 
pending and approved; 
‘‘(2) the number of applications described in 
paragraph (1) disaggregated by— 
‘‘(A) the disease or condition to be ad-
dressed; and 
‘‘(B) the target subpopulations identified 
in such applications; 
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‘‘(3) the prevalence in the United States of each 
disease or condition addressed by an application de-
scribed in paragraph (1); 
‘‘(4) integration of Food and Drug Administra-
tion staff with expertise in rare diseases and condi-
tions in each review of an application described in 
paragraph (1); 
‘‘(5) identification of all external experts with 
expertise in rare diseases and conditions who are 
consulted in connection with, or who otherwise par-
ticipate in, each review of an application described in 
paragraph (1); and 
‘‘(6) the status of each application described in 
paragraph (1). 
‘‘(b) PUBLIC AVAILABILITY.—The Secretary shall 
make each report under subsection (a) available to the 
public, including by posting the report on the website of 
the Food and Drug Administration.’’. 
(b) STUDY ON EUROPEAN UNION SAFETY AND EFFI-
CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON-
DITIONS.— 
(1) IN GENERAL.—The Comptroller General of 
the United States shall conduct a study on Euro-
pean Union safety and efficacy reviews of drugs for 
rare diseases and conditions, including— 
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(A) any differential prevalence-based mech-
anisms; and 
(B) consideration and use of supplemental 
data submitted during the review process, in-
cluding data associated with open label exten-
sion studies and expanded access programs. 
(2) REPORT.—Not later than 1 year after the 
date of enactment of this Act, the Comptroller Gen-
eral shall— 
(A) complete the study under paragraph 
(1); 
(B) submit a report on the results of such 
study to the Congress; and 
(C) include in such report recommenda-
tions for changes to the processes and authori-
ties of the Food and Drug Administration to fa-
cilitate development of, and access to, treat-
ments for rare diseases and conditions. 
(3) PUBLIC
AVAILABILITY.—The Comptroller 
General of the United States shall make each report 
under paragraph (2) available to the public, includ-
ing by posting the report on the website of the Gov-
ernment Accountability Office. 
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(c) REVIEW PROCESS.—Subparagraph (A) of section 
569(a)(2) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 360bbb–8(a)(2)) is amended to read as follows: 
‘‘(A) IN GENERAL.— 
‘‘(i) LIST.—The Secretary shall de-
velop and maintain a list of external ex-
perts who, because of their special exper-
tise, are qualified to provide advice on rare 
disease and condition issues, including top-
ics described in subsection (b). 
‘‘(ii) AVAILABILITY
AT
MEETINGS.— 
The Secretary shall ensure availability, for 
consultation purposes, of an external ex-
pert on rare diseases, as described in sub-
paragraph (B), in connection with each 
drug product advisory committee meeting 
concerning a drug or biological product for 
a rare disease or condition. 
‘‘(iii) REVIEW
OF
APPLICATIONS.— 
The Secretary shall ensure that each re-
view of an application submitted under sec-
tion 505 of this Act or section 351 of the 
Public Health Service Act for an indication 
associated with a rare disease or condition 
includes Food and Drug Administration 
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staff with expertise on rare diseases and 
conditions as an integral part of the review 
team.’’. 
(d) RISK EVALUATION AND MITIGATION STRATEGY 
ELEMENTS.—Subsection (a) of section 505–1 of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355–1) is 
amended by adding at the end the following new para-
graph: 
‘‘(5) ORPHAN DRUGS.—In making any deter-
mination regarding the inclusion, in the risk evalua-
tion and mitigation strategy for a drug that has 
been designated as a drug for a rare disease or con-
dition pursuant to section 526, of an element that 
requires patient action, restrictions on use of the 
drug, or patient participation, the Secretary shall 
consult with patients impacted by the rare disease or 
condition in considering the burden of participation 
and likely patient compliance.’’. 
Æ 
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