II 

112TH CONGRESS 

2D SESSION S. 2236 

To provide for the expedited development and evaluation of drugs designated 

as breakthrough drugs. 

IN THE SENATE OF THE UNITED STATES 

MARCH 26, 2012 

Mr. BENNET (for himself, Mr. HATCH, and Mr. BURR) introduced the following bill; which was read twice and referred to the Committee on 

Health, Education, Labor, and Pensions 

A BILL 

To provide for the expedited development and evaluation 

of drugs designated as breakthrough drugs. 

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 ‘‘Advancing Break5 through Therapies for Patients Act of 2012’’. 

6 SEC. 2. BREAKTHROUGH THERAPIES AND FAST TRACK 

7 PRODUCTS. 

8 (a) IN GENERAL.—Section 506 of the Federal Food, 

9 Drug, and Cosmetic Act (21 U.S.C. 356) is amended— 


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1 (1) in the heading, by inserting ‘‘BREAK2 THROUGH THERAPIES AND’’ before ‘‘FAST’’; 

3 (2) by redesignating subsections (a) through (d) 

4 as subsections (b) through (e), respectively; 

5 (3) by inserting before subsection (b), as so re6 designated, the following: 

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

8 THERAPY.— 

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

10 request of the sponsor of a drug, expedite the devel11 opment and review of such drug if the drug is in12 tended, alone or in combination with 1 or more other 

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

14 or condition and preliminary clinical evidence indi15 cates that the drug may demonstrate substantial im16 provement over existing therapies on 1 or more clini17 cally significant endpoints (such as substantial treat18 ment effects observed early in clinical development). 

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

20 ‘breakthrough therapy’.) 

21 ‘‘(2) REQUEST FOR DESIGNATION.—The spon22 sor of a drug may request the Secretary to designate 

23 the drug as a breakthrough therapy. A request for 

24 the designation may be made concurrently with, or 

25 at any time after, the submission of an application 


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

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

3 Act. 

4 ‘‘(3) DESIGNATION.— 

5 ‘‘(A) IN GENERAL.—Not later than 60 cal6 endar days after the receipt of a request under 

7 paragraph (2), the Secretary shall determine 

8 whether the drug that is the subject of the re9 quest meets the criteria described in paragraph 

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

11 the criteria, the Secretary shall designate the 

12 drug as a breakthrough therapy and shall take 

13 such actions as are appropriate to expedite the 

14 development and review of the application for 

15 approval of such drug. 

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

17 the development and review of an application 

18 under subparagraph (A) shall include— 

19 ‘‘(i) holding meetings with the sponsor 

20 and the review team throughout the devel21 opment of the drug; 

22 ‘‘(ii) providing timely advice to the 

23 sponsor regarding the development of the 

24 drug to ensure that the development pro25 gram to gather the non-clinical and clinical 


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1 data necessary for approval is as efficient 

2 as practicable; 

3 ‘‘(iii) involving senior managers and 

4 experienced review staff, as appropriate, in 

5 a collaborative, cross-disciplinary review; 

6 ‘‘(iv) providing timely interactive com7 munication with sponsors; 

8 ‘‘(v) assigning a cross-disciplinary 

9 project lead for the Food and Drug Ad10 ministration review team to facilitate an 

11 efficient review of the development pro12 gram and to serve as a scientific liaison be13 tween the review team and the sponsor; 

14 and 

15 ‘‘(vi) taking steps to ensure that the 

16 design of the clinical trials is as efficient as 

17 practicable, when scientifically appropriate, 

18 such as by minimizing the number of pa19 tients enrolled in the trial and the duration 

20 of the trial and considering alternatives to 

21 the traditional multi-phase, sequential de22 velopment approach, designed to abbre23 viate, consolidate, and condense clinical 

24 trials and studies.’’; 


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1 (4) in subsection (e)(1), as so redesignated, by 

2 inserting ‘‘breakthrough therapies and’’ after ‘‘appli3 cable to’’; and 

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

5 ‘‘(f) GUIDANCE; AMENDED REGULATIONS.— 

6 ‘‘(1) IN GENERAL.— 

7 ‘‘(A) GUIDANCE.—Not later than 18 

8 months after the date of enactment of the Ad9 vancing Breakthrough Therapies for Patients 

10 Act of 2012, the Secretary shall issue draft 

11 guidance on implementing the requirements 

12 with respect to breakthrough therapies, acceler13 ated approval, and fast track products as set 

14 forth in subsections (a) through (c), as amend15 ed by the Advancing Breakthrough Therapies 

16 for Patients Act of 2012. After an opportunity 

17 for public comment and not later than 2 years 

18 after the date of enactment of the Advancing 

19 Breakthrough Therapies for Patients Act of 

20 2012, the Secretary shall issue final guidance. 

21 ‘‘(B) AMENDED REGULATIONS.—Not later 

22 than 2 years after the date of enactment of the 

23 Advancing Breakthrough Therapies for Patients 

24 Act of 2012, the Secretary shall amend the ap25 plicable regulations under title 21, Code of FedVerDate Mar 15 2010 02:58 Mar 27, 2012 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S2236.IS S2236

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1 eral Regulations, as may be necessary to imple2 ment the requirements under subsections (a) 

3 through (c), as amended by the Advancing 

4 Breakthrough Therapies for Patients Act of 

5 2012. 

6 ‘‘(2) REQUIREMENTS.—Guidance and regula7 tions promulgated under this section shall— 

8 ‘‘(A) distinguish between products that 

9 may qualify for— 

10 ‘‘(i) treatment as a breakthrough 

11 therapy; 

12 ‘‘(ii) treatment as a fast track prod13 uct; 

14 ‘‘(iii) accelerated approval; and 

15 ‘‘(iv) a combination of all of the des16 ignations described in clauses (i) through 

17 (iii); and 

18 ‘‘(B) specify the actions the Secretary shall 

19 take to expedite the development and review of 

20 a breakthrough therapy pursuant to such des21 ignation under 506(a)(3), including updating 

22 good review management practices to reflect 

23 breakthrough therapies. 

24 ‘‘(g) INDEPENDENT REVIEW.—Not later than 3 

25 years after the date of enactment of this Act, the SecVerDate Mar 15 2010 02:58 Mar 27, 2012 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S2236.IS S2236

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1 retary shall, in conjunction with other planned reviews, 

2 contract with an independent entity with expertise in as3 sessing the quality, efficiency, and predictability of bio4 pharmaceutical development and regulatory review pro5 grams to evaluate the manner by which the Food and 

6 Drug Administration has applied the processes described 

7 in the section, as amended by the Advancing Break8 through Therapies for Patients Act of 2012, and the im9 pact of such processes on the development and timely 

10 availability of innovative treatments for patients affected 

11 by serious or life-threatening conditions. Such evaluation 

12 shall be completed not later than 4 years after the date 

13 of enactment of the Advancing Breakthrough Therapies 

14 for Patients Act of 2012 and shall be made publicly avail15 able upon completion. 

16 ‘‘(h) REPORT.—Beginning in fiscal year 2013, the 

17 Secretary shall annually prepare and submit to the Com18 mittee on Health, Education, Labor, and Pensions of the 

19 Senate and the Committee on Energy and Commerce of 

20 the House of Representatives, and make publicly available, 

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

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

23 requested designation as a breakthrough therapy; 

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

25 breakthrough therapy; and 


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1 ‘‘(3) for each breakthrough therapy approved in 

2 the fiscal year— 

3 ‘‘(A) the point in the drug development 

4 and review process at which such breakthrough 

5 designation occurred; 

6 ‘‘(B) the total time from designation as a 

7 breakthrough therapy, including the total time 

8 to review and act on an application designated 

9 as a breakthrough therapy, to approval of the 

10 drug; and 

11 ‘‘(C) the number of breakthrough therapies 

12 approved on the first review out of the total 

13 number of such therapies so approved.’’. 

14 (b) CONFORMING AMENDMENTS.—Section 506B(e) 

15 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

16 356b) is amended by striking ‘‘section 506(b)(2)(A)’’ each 

17 place such term appears and inserting ‘‘section 

18 506(c)(2)(A)’’. 

Æ 


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