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 Committee 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. 

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 ‘‘Helping Experts Accel5 erate Rare Treatments Act of 2021’’. 

6 SEC. 2. IMPROVING THE TREATMENT OF RARE DISEASES 

7 AND CONDITIONS. 

8 (a) ANNUAL REPORT ON ORPHAN DRUG PRO9 GRAM.—Subchapter B of chapter V of the Federal Food, 


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1 Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) is 

2 amended by adding at the end the following new section: 

3 ‘‘SEC. 529B. ANNUAL REPORT ON ORPHAN DRUG PROGRAM. 

4 ‘‘(a) IN GENERAL.—Not later than 1 year after the 

5 date of enactment of the Helping Experts Accelerate Rare 

6 Treatments Act of 2021, and not less than annually there7 after, the Secretary shall submit to the Congress a report 

8 summarizing the activities of the Food and Drug Adminis9 tration for designating drugs under section 526 for a rare 

10 disease or condition and approving such drugs under sec11 tion 505 of this Act or licensing such drugs under section 

12 351 of the Public Health Service Act, including— 

13 ‘‘(1) the number of applications for such drugs 

14 under sections 505 of this Act and section 351 of 

15 the Public Health Service Act received by the Food 

16 and Drug Administration, the number of such appli17 cations considered by each Food and Drug Adminis18 tration division, and the number of such applications 

19 pending and approved; 

20 ‘‘(2) the number of applications described in 

21 paragraph (1) disaggregated by— 

22 ‘‘(A) the disease or condition to be ad23 dressed; and 

24 ‘‘(B) the target subpopulations identified 

25 in such applications; 


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1 ‘‘(3) the prevalence in the United States of each 

2 disease or condition addressed by an application de3 scribed in paragraph (1); 

4 ‘‘(4) integration of Food and Drug Administra5 tion staff with expertise in rare diseases and condi6 tions in each review of an application described in 

7 paragraph (1); 

8 ‘‘(5) identification of all external experts with 

9 expertise in rare diseases and conditions who are 

10 consulted in connection with, or who otherwise par11 ticipate in, each review of an application described in 

12 paragraph (1); and 

13 ‘‘(6) the status of each application described in 

14 paragraph (1). 

15 ‘‘(b) PUBLIC AVAILABILITY.—The Secretary shall 

16 make each report under subsection (a) available to the 

17 public, including by posting the report on the website of 

18 the Food and Drug Administration.’’. 

19 (b) STUDY ON EUROPEAN UNION SAFETY AND EFFI20 CACY REVIEWS OF DRUGS FOR RARE DISEASES AND CON21 DITIONS.— 

22 (1) IN GENERAL.—The Comptroller General of 

23 the United States shall conduct a study on Euro24 pean Union safety and efficacy reviews of drugs for 

25 rare diseases and conditions, including— 


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1 (A) any differential prevalence-based mech2 anisms; and 

3 (B) consideration and use of supplemental 

4 data submitted during the review process, in5 cluding data associated with open label exten6 sion studies and expanded access programs. 

7 (2) REPORT.—Not later than 1 year after the 

8 date of enactment of this Act, the Comptroller Gen9 eral shall— 

10 (A) complete the study under paragraph 

11 (1); 

12 (B) submit a report on the results of such 

13 study to the Congress; and 

14 (C) include in such report recommenda15 tions for changes to the processes and authori16 ties of the Food and Drug Administration to fa17 cilitate development of, and access to, treat18 ments for rare diseases and conditions. 

19 (3) PUBLIC AVAILABILITY.—The Comptroller 

20 General of the United States shall make each report 

21 under paragraph (2) available to the public, includ22 ing by posting the report on the website of the Gov23 ernment Accountability Office. 


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1 (c) REVIEW PROCESS.—Subparagraph (A) of section 

2 569(a)(2) of the Federal Food, Drug, and Cosmetic Act 

3 (21 U.S.C. 360bbb–8(a)(2)) is amended to read as follows: 

4 ‘‘(A) IN GENERAL.— 

5 ‘‘(i) LIST.—The Secretary shall de6 velop and maintain a list of external ex7 perts who, because of their special exper8 tise, are qualified to provide advice on rare 

9 disease and condition issues, including top10 ics described in subsection (b). 

11 ‘‘(ii) AVAILABILITY AT MEETINGS.— 

12 The Secretary shall ensure availability, for 

13 consultation purposes, of an external ex14 pert on rare diseases, as described in sub15 paragraph (B), in connection with each 

16 drug product advisory committee meeting 

17 concerning a drug or biological product for 

18 a rare disease or condition. 

19 ‘‘(iii) REVIEW OF APPLICATIONS.— 

20 The Secretary shall ensure that each re21 view of an application submitted under sec22 tion 505 of this Act or section 351 of the 

23 Public Health Service Act for an indication 

24 associated with a rare disease or condition 

25 includes Food and Drug Administration 


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1 staff with expertise on rare diseases and 

2 conditions as an integral part of the review 

3 team.’’. 

4 (d) RISK EVALUATION AND MITIGATION STRATEGY

5 ELEMENTS.—Subsection (a) of section 505–1 of the Fed6 eral Food, Drug, and Cosmetic Act (21 U.S.C. 355–1) is 

7 amended by adding at the end the following new para8 graph: 

9 ‘‘(5) ORPHAN DRUGS.—In making any deter10 mination regarding the inclusion, in the risk evalua11 tion and mitigation strategy for a drug that has 

12 been designated as a drug for a rare disease or con13 dition pursuant to section 526, of an element that 

14 requires patient action, restrictions on use of the 

15 drug, or patient participation, the Secretary shall 

16 consult with patients impacted by the rare disease or 

17 condition in considering the burden of participation 

18 and likely patient compliance.’’. 

Æ 


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