I 

112TH CONGRESS 

1ST SESSION H. R. 3737 

To amend the Federal Food, Drug, and Cosmetic Act with respect to fast 

track approval of certain orphan drugs. 

IN THE HOUSE OF REPRESENTATIVES 

DECEMBER 20, 2011 

Mr. STEARNS (for himself and Mr. TOWNS) 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 fast track approval of certain orphan 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 ‘‘Unlocking Lifesaving 

5 Treatments for Rare-Diseases Act’’ or ‘‘ULTRA’’. 


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1 SEC. 2. IMPROVING THE ACCELERATED APPROVAL PATH2 WAY FOR FAST TRACK PRODUCTS TO SERVE 

3 THE UNMET NEEDS OF INDIVIDUALS WITH 

4 ULTRA RARE DISEASES. 

5 Section 506 of the Federal Food, Drug, and Cosmetic 

6 Act (21 U.S.C. 356) is amended by adding at the end the 

7 following: 

8 ‘‘(e) SCIENTIFIC STANDARDS FOR APPROVAL OF

9 CERTAIN ORPHAN DRUGS AS FAST TRACK PRODUCTS.— 

10 ‘‘(1) IN GENERAL.—The Secretary may approve 

11 an application for a drug designated under section 

12 526 for a rare disease or condition as a fast track 

13 product using a surrogate endpoint as described 

14 under paragraph (2) if— 

15 ‘‘(A) the Secretary makes an initial deter16 mination that the drug is eligible for approval— 

17 ‘‘(i) as a drug designated for a rare 

18 disease or condition under section 526; and 

19 ‘‘(ii) as a fast track product under 

20 this section; and 

21 ‘‘(B) the drug is a treatment for a disease 

22 or condition that affects a small number of pa23 tients in the United States, as determined by 

24 the Secretary in designating the drug for a rare 

25 disease or condition under section 526. 


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1 ‘‘(2) SURROGATE ENDPOINT DEFINITION FOR

2 CERTAIN FAST TRACK PRODUCTS.— 

3 ‘‘(A) IN GENERAL.—If a drug meets the 

4 criteria established in paragraph (1), the Sec5 retary— 

6 ‘‘(i) may use a surrogate endpoint for 

7 the approval of the drug as a fast track 

8 product based on the existence of reason9 able scientific data that support and qual10 ify the relevance of the surrogate endpoint 

11 to the disease state and treatment; and 

12 ‘‘(ii) shall not require clinical treat13 ment data or other historical clinical data 

14 on the surrogate endpoint as a prerequisite 

15 to assessment of the surrogate endpoint 

16 under this subsection if such data are not 

17 available. 

18 ‘‘(B) USE OF CLINICAL DATA.— 

19 ‘‘(i) Subject to subparagraph (A)(ii), 

20 in a surrogate endpoint assessment under 

21 this subsection, the Secretary may take 

22 into consideration any reliable clinical data 

23 that are readily available and published. 

24 ‘‘(ii) For a surrogate endpoint which 

25 the Secretary decides to use in accordance 


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1 with subparagraph (A), nothing in this 

2 subsection shall preclude the Secretary 

3 from requiring clinical data that makes use 

4 of the surrogate endpoint as a condition of 

5 approval for the fast track product. 

6 ‘‘(C) GUIDANCE AND CONSIDERATIONS.— 

7 Not later than 1 year after the date of enact8 ment of the Unlocking Lifesaving Treatments 

9 for Rare-Diseases Act, the Secretary shall issue 

10 guidance providing details and options for 

11 qualifying surrogate endpoints without clinical 

12 data pursuant to this subsection. In qualifying 

13 a surrogate endpoint under this subsection, the 

14 Secretary shall take into account and balance 

15 the following considerations: 

16 ‘‘(i) The unmet need served by the 

17 drug and the adverse effects of the rare 

18 disease or condition on quality of life and 

19 length of life. 

20 ‘‘(ii) The very low likelihood that clin21 ical data would exist or that clinical stud22 ies would be completed to support a surro23 gate endpoint due to the small size of the 

24 patient population in the United States 

25 and other significant barriers inherent in 


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1 performing such clinical studies due to the 

2 prevalence of the disease or related factors. 

3 ‘‘(iii) The full scope of available basic 

4 scientific data and information describing 

5 the pathophysiology of the disease, mecha6 nism of action of the drug, biology of the 

7 relevant disease pathway, information re8 garding the quality of the biomarker assay, 

9 model treatment data, or other supportive 

10 scientific information that the Secretary 

11 deems reasonably predictive of a clinical 

12 benefit in the absence of clinical data.’’. 

Æ 


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