I 

116TH CONGRESS 

1ST SESSION H. R. 4712 

To amend the Federal Food, Drug, and Cosmetic Act with respect to limitations on exclusive approval or licensure of orphan drugs, and for other 

purposes. 

IN THE HOUSE OF REPRESENTATIVES 

OCTOBER 17, 2019 

Ms. DEAN (for herself, Mr. VEASEY, Mr. CARTER of Georgia, and Mr. MCKINLEY) 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 with 

respect to limitations on exclusive approval or licensure 

of orphan drugs, 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 ‘‘Fairness in Orphan 

5 Drug Exclusivity Act’’. 

6 SEC. 2. LIMITATIONS ON EXCLUSIVE APPROVAL OR LICEN7 SURE OF ORPHAN DRUGS. 

8 (a) IN GENERAL.—Section 527 of the Federal Food, 

9 Drug, and Cosmetic Act (21 U.S.C. 360cc) is amended— 


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1 (1) in subsection (a), by striking ‘‘Except as 

2 provided in subsection (b)’’ and inserting ‘‘Except as 

3 provided in subsection (b) or (f)’’; and 

4 (2) by adding at the end the following: 

5 ‘‘(f) LIMITATIONS ON EXCLUSIVE APPROVAL, CER6 TIFICATION, OR LICENSE.— 

7 ‘‘(1) IN GENERAL.—For a drug designated 

8 under section 526 for a rare disease or condition 

9 pursuant to the criteria set forth in subsection 

10 (a)(2)(B) of such section, the Secretary shall not 

11 grant, recognize, or apply exclusive approval or licen12 sure under subsection (a), and, if such exclusive ap13 proval or licensure has been granted, recognized, or 

14 applied, shall revoke such exclusive approval or licen15 sure, unless the sponsor of the application for such 

16 drug demonstrates— 

17 ‘‘(A)(i) with respect to an application ap18 proved or a license issued after the date of en19 actment of this subsection, upon such approval 

20 or issuance, that there is no reasonable expecta21 tion at the time of such approval or issuance 

22 that the cost of developing and making avail23 able in the United States such drug for such 

24 disease or condition will be recovered from sales 

25 in the United States of such drug, taking into 


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1 account all sales made or reasonably expected 

2 to be made without a time limitation; or 

3 ‘‘(ii) with respect to an application ap4 proved or a license issued on or prior to the 

5 date of enactment of this subsection, not later 

6 than 60 days after such date of enactment, that 

7 there was no reasonable expectation at the time 

8 of such approval or issuance that the cost of de9 veloping and making available in the United 

10 States such drug for such disease or condition 

11 would be recovered from sales in the United 

12 States of such drug, taking into account all 

13 sales made or reasonably expected to be made 

14 without a time limitation; and 

15 ‘‘(B) annually for the duration of the 7- 

16 year period described in subsection (a) with re17 spect to the drug, that there continues to be no 

18 reasonable expectation that the cost of devel19 oping and making available in the United 

20 States such drug for such disease or condition 

21 will be recovered from sales in the United 

22 States of such drug, taking into account all 

23 sales made or reasonably expected to be made 

24 without a time limitation. 


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1 ‘‘(2) CONSIDERATIONS.—For purposes of sub2 paragraphs (A) and (B) of paragraph (1), the Sec3 retary and the sponsor of the application for the 

4 drug designated for a rare disease or condition de5 scribed in such paragraph shall consider sales from 

6 all drugs for such disease or condition that— 

7 ‘‘(A) are developed or marketed by the 

8 same sponsor or manufacturer of the drug (or 

9 a licensor, predecessor in interest, or other re10 lated entity to the sponsor or manufacturer); 

11 and 

12 ‘‘(B) contain the same active moiety as the 

13 drug, regardless of whether or not the drugs 

14 also contain different or additional active 

15 moieties. 

16 ‘‘(3) CRITERIA.—No drug designated under 

17 section 526 for a rare disease or condition pursuant 

18 to the criteria set forth in subsection (a)(2)(B) of 

19 such section shall be eligible for exclusive approval 

20 or licensure under this section unless it met such 

21 criteria under such subsection on the date on which 

22 the drug was approved or licensed.’’. 

23 (b) RULE OF CONSTRUCTION.—The amendments 

24 made in subsection (a) shall apply to any drug that has 

25 been or is hereafter designated under section 526 of the 


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1 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb) 

2 for a rare disease or condition pursuant to the criteria 

3 under subsection (a)(2)(B) of such section regardless of— 

4 (1) the date on which such drug is designated 

5 or becomes the subject of a designation request 

6 under such section; 

7 (2) the date on which such drug is approved 

8 under section 505 of such Act (21 U.S.C. 355) or 

9 licensed under section 351 of the Public Health 

10 Service Act (42 U.S.C. 262) or becomes the subject 

11 of an application for such approval or licensure; and 

12 (3) the date on which such drug is granted ex13 clusive approval or licensure under section 527 of 

14 the Federal Food, Drug, and Cosmetic Act (21 

15 U.S.C. 360cc) or becomes the subject of a request 

16 for such exclusive approval or licensure. 

Æ 


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