I 

112TH CONGRESS 

2D SESSION H. R. 5334 

To amend chapter V of the Federal Food, Drug, and Cosmetic Act to 

expedite the development and review of breakthrough therapies. 

IN THE HOUSE OF REPRESENTATIVES 

MAY 7, 2012 

Mr. BILBRAY (for himself and Ms. DEGETTE) introduced the following bill; 

which was referred to the Committee on Energy and Commerce 

A BILL 

To amend chapter V of the Federal Food, Drug, and Cosmetic Act to expedite the development and review of 

breakthrough therapies. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. BREAKTHROUGH THERAPIES. 

4 (a) IN GENERAL.—Section 506 (21 U.S.C. 356) is 

5 amended— 

6 (1) by redesignating subsection (d) as sub7 section (f); 

8 (2) by redesignating subsections (a) through (c) 

9 as subsections (b) through (d), respectively; 


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1 (3) by inserting before subsection (b), as so re2 designated, the following: 

3 ‘‘(a) DESIGNATION OF A DRUG AS A BREAKTHROUGH

4 THERAPY.— 

5 ‘‘(1) IN GENERAL.—The Secretary shall, at the 

6 request of the sponsor of a drug, expedite the devel7 opment and review of such drug if the drug is in8 tended, alone or in combination with 1 or more other 

9 drugs, to treat a serious or life-threatening disease 

10 or condition and preliminary clinical evidence indi11 cates that the drug may demonstrate substantial im12 provement over existing therapies on 1 or more clini13 cally significant endpoints, such as substantial treat14 ment effects observed early in clinical development. 

15 (In this section, such a drug is referred to as a 

16 ‘breakthrough therapy’.) 

17 ‘‘(2) REQUEST FOR DESIGNATION.—The spon18 sor of a drug may request the Secretary to designate 

19 the drug as a breakthrough therapy. A request for 

20 the designation may be made concurrently with, or 

21 at any time after, the submission of an application 

22 for the investigation of the drug under section 505(i) 

23 or section 351(a)(3) of the Public Health Service 

24 Act. 

25 ‘‘(3) DESIGNATION.— 


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1 ‘‘(A) IN GENERAL.—Not later than 60 cal2 endar days after the receipt of a request under 

3 paragraph (2), the Secretary shall determine 

4 whether the drug that is the subject of the re5 quest meets the criteria described in paragraph 

6 (1). If the Secretary finds that the drug meets 

7 the criteria, the Secretary shall designate the 

8 drug as a breakthrough therapy and shall take 

9 such actions as are appropriate to expedite the 

10 development and review of the application for 

11 approval of such drug. 

12 ‘‘(B) ACTIONS.—The actions to expedite 

13 the development and review of an application 

14 under subparagraph (A) may include, as appro15 priate— 

16 ‘‘(i) holding meetings with the sponsor 

17 and the review team throughout the devel18 opment of the drug; 

19 ‘‘(ii) providing timely advice to, and 

20 interactive communication with, the spon21 sor regarding the development of the drug 

22 to ensure that the development program to 

23 gather the non-clinical and clinical data 

24 necessary for approval is as efficient as 

25 practicable; 


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1 ‘‘(iii) involving senior managers and 

2 experienced review staff, as appropriate, in 

3 a collaborative, cross-disciplinary review; 

4 ‘‘(iv) assigning a cross-disciplinary 

5 project lead for the Food and Drug Ad6 ministration review team to facilitate an 

7 efficient review of the development pro8 gram and to serve as a scientific liaison be9 tween the review team and the sponsor; 

10 and 

11 ‘‘(v) taking steps to ensure that the 

12 design of the clinical trials is as efficient as 

13 practicable, when scientifically appropriate, 

14 such as by minimizing the number of pa15 tients exposed to a potentially less effica16 cious treatment.’’; 

17 (4) in subsection (f)(1), as so redesignated, by 

18 striking ‘‘applicable to accelerated approval’’ and in19 serting ‘‘applicable to breakthrough therapies, accel20 erated approval, and’’; and 

21 (5) by adding at the end the following: 

22 ‘‘(g) REPORT.—Beginning in fiscal year 2013, the 

23 Secretary shall annually prepare and submit to the Com24 mittee on Health, Education, Labor, and Pensions of the 

25 Senate and the Committee on Energy and Commerce of 


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1 the House of Representatives, and make publicly available, 

2 with respect to this section for the previous fiscal year— 

3 ‘‘(1) the number of drugs for which a sponsor 

4 requested designation as a breakthrough therapy; 

5 and 

6 ‘‘(2) the number of products designated as a 

7 breakthrough therapy.’’. 

8 (b) GUIDANCE; AMENDED REGULATIONS.— 

9 (1) IN GENERAL.— 

10 (A) GUIDANCE.—Not later than 18 

11 months after the date of enactment of this Act, 

12 the Secretary of Health and Human Services 

13 (referred to in this section as the ‘‘Secretary’’) 

14 shall issue draft guidance on implementing the 

15 requirements with respect to breakthrough 

16 therapies, as set forth in section 506(a) of the 

17 Federal Food, Drug, and Cosmetic Act (21 

18 U.S.C. 356(a)), as amended by this section. 

19 The Secretary shall issue final guidance not 

20 later than 1 year after the close of the comment 

21 period for the draft guidance. 

22 (B) AMENDED REGULATIONS.— 

23 (i) IN GENERAL.—If the Secretary de24 termines that it is necessary to amend the 

25 regulations under title 21, Code of Federal 


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1 Regulations in order to implement the 

2 amendments made by this section to sec3 tion 506(a) of the Federal Food, Drug, 

4 and Cosmetic Act, the Secretary shall 

5 amend such regulations not later than 2 

6 years after the date of enactment of this 

7 Act. 

8 (ii) PROCEDURE.—In amending regu9 lations under clause (i), the Secretary 

10 shall— 

11 (I) issue a notice of proposed 

12 rulemaking that includes the proposed 

13 regulation; 

14 (II) provide a period of not less 

15 than 60 days for comments on the 

16 proposed regulation; and 

17 (III) publish the final regulation 

18 not less than 30 days before the effec19 tive date of the regulation. 

20 (iii) RESTRICTIONS.—Notwithstanding 

21 any other provision of law, the Secretary 

22 shall promulgate regulations implementing 

23 the amendments made by this section only 

24 as described in clause (ii). 


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1 (2) REQUIREMENTS.—Guidance issued under 

2 this section shall— 

3 (A) specify the process and criteria by 

4 which the Secretary makes a designation under 

5 section 506(a)(3) of the Federal Food, Drug, 

6 and Cosmetic Act; and 

7 (B) specify the actions the Secretary shall 

8 take to expedite the development and review of 

9 a breakthrough therapy pursuant to such des10 ignation under such section 506(a)(3), includ11 ing updating good review management practices 

12 to reflect breakthrough therapies. 

13 (c) INDEPENDENT REVIEW.—Not later than 3 years 

14 after the date of enactment of this Act, the Comptroller 

15 General of the United States, in consultation with appro16 priate experts, shall assess the manner by which the Food 

17 and Drug Administration has applied the processes de18 scribed in section 506(a) of the Federal Food, Drug, and 

19 Cosmetic Act, as amended by this section, and the impact 

20 of such processes on the development and timely avail21 ability of innovative treatments for patients affected by se22 rious or life-threatening conditions. Such assessment shall 

23 be made publicly available upon completion. 

24 (d) CONFORMING AMENDMENTS.—Section 506B(e) 

25 (21 U.S.C. 356b) is amended by striking ‘‘section 


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1 506(b)(2)(A)’’ each place such term appears and inserting 

2 ‘‘section 506(c)(2)(A)’’. 

Æ 


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