II 

117TH CONGRESS 

1ST SESSION S. 250 

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 SENATE OF THE UNITED STATES 

FEBRUARY 4, 2021 

Mr. CASSIDY (for himself, Ms. BALDWIN, and Mrs. SHAHEEN) introduced the 

following bill; which was read twice and referred to the Committee on 

Health, Education, Labor, and Pensions 

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) 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 within 12 years of first marketing 

3 the drug; or 

4 ‘‘(B) with respect to an application ap5 proved or a license issued on or prior to the 

6 date of enactment of this subsection, not later 

7 than 60 days after such date of enactment, that 

8 there was no reasonable expectation at the time 

9 of such approval or issuance that the cost of de10 veloping and making available in the United 

11 States such drug for such disease or condition 

12 would be recovered from sales in the United 

13 States of such drug, taking into account all 

14 sales made or reasonably expected to be made 

15 within 12 years of first marketing the drug. 

16 ‘‘(2) CONSIDERATIONS.—For purposes of sub17 paragraphs (A) and (B) of paragraph (1), the Sec18 retary and the sponsor of the application for the 

19 drug designated for a rare disease or condition de20 scribed in such paragraph shall consider sales from 

21 all drugs that— 

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

23 same sponsor or manufacturer of the drug (or 

24 a licensor, predecessor in interest, or other reVerDate Sep 11 2014 21:12 Feb 20, 2021 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\S250.IS S250

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1 lated entity to the sponsor or manufacturer); 

2 and 

3 ‘‘(B) are covered by the same designation 

4 under section 526. 

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

6 section 526 for a rare disease or condition pursuant 

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

8 such section shall be eligible for exclusive approval 

9 or licensure under this section unless it met such 

10 criteria under such subsection on the date on which 

11 the drug was approved or licensed.’’. 

12 (b) RULE OF CONSTRUCTION.—The amendments 

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

14 been or is hereafter designated under section 526 of the 

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

16 for a rare disease or condition pursuant to the criteria 

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

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

19 or becomes the subject of a designation request 

20 under such section; 

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

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

23 licensed under section 351 of the Public Health 

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

25 of an application for such approval or licensure; and 


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1 (3) the date on which such drug is granted ex2 clusive approval or licensure under section 527 of 

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

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

5 for such exclusive approval or licensure. 

Æ 


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