I 

112TH CONGRESS 

2D SESSION H. R. 4132 

To amend section 506 of the Federal Food, Drug, and Cosmetic Act to 

expedited approval of drugs for serious or life-threatening diseases or 

conditions. 

IN THE HOUSE OF REPRESENTATIVES 

MARCH 5, 2012 

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 section 506 of the Federal Food, Drug, and Cosmetic Act to expedited approval of drugs for serious 

or life-threatening diseases or conditions. 

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 ‘‘Faster Access to Spe5 cialized Treatments Act’’ or ‘‘FAST Act’’. 

6 SEC. 2. FINDINGS; SENSE OF CONGRESS. 

7 (a) FINDINGS.—Congress finds the following: 

8 (1) The Food and Drug Administration (FDA) 

9 serves a critical role in helping to assure that new 


smartinez on DSK6TPTVN1PROD with BILLS
2 

•HR 4132 IH

1 medicines are safe and effective. Regulatory innova2 tion is one element of the Nation’s strategy to ad3 dress serious and life-threatening diseases or condi4 tions by promoting investment in and development 

5 of innovative treatments for unmet medical needs. 

6 (2) Over the previous two decades, since the ac7 celerated approval mechanism was established, ad8 vances in medical sciences, including genomics, mo9 lecular biology, and bioinformatics, have provided an 

10 unprecedented understanding of the underlying bio11 logical mechanism and pathogenesis of disease. A 

12 new generation of modern, targeted medicines is cur13 rently under development to treat serious and life14 threatening diseases, some applying drug develop15 ment strategies based on biomarkers or 

16 pharmacogenomics, predictive toxicology, clinical 

17 trial enrichment techniques, and novel clinical trial 

18 designs, such as adaptive clinical trials. 

19 (3) As a result of these remarkable scientific 

20 and medical advances, FDA should be encouraged to 

21 implement more broadly effective processes for the 

22 expedited development and review of innovative new 

23 medicines intended to address unmet medical needs 

24 for serious or life-threatening diseases or conditions, 

25 including those for rare diseases or conditions, using 


smartinez on DSK6TPTVN1PROD with BILLS
3 

•HR 4132 IH

1 a broad range of surrogate or clinical endpoints and 

2 modern scientific tools earlier in the drug develop3 ment cycle when appropriate. This may result in 

4 fewer, smaller, or shorter clinical trials for the in5 tended patient population or targeted subpopulation 

6 without compromising or altering FDA’s existing 

7 high standards for the approval of drugs. 

8 (4) Patients benefit from expedited access to 

9 safe and effective innovative therapies to treat 

10 unmet medical needs for serious or life-threatening 

11 diseases or conditions. 

12 (5) For these reasons, the existing statutory au13 thority governing expedited approval of drugs or se14 rious or life-threatening conditions should be amend15 ed in order to enhance FDA’s authority to consider 

16 appropriate scientific data, methods, and tools, and 

17 to expedite development and access to novel treat18 ments for patients with a broad range oofserious or 

19 life-threatening diseases or conditions. 

20 (b) SENSE OF CONGRESS.—It is the sense of Con21 gress that the Food and Drug Administration should 

22 apply the accelerated approval and the fast track provi23 sions set forth in section 506 of the Federal Food, Drug, 

24 and Cosmetic Act (21 U.S.C. 356), as amended by section 

25 3, to the greatest extent possible to help expedite the deVerDate Mar 15 2010 23:54 Mar 05, 2012 Jkt 019200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H4132.IH H4132

smartinez on DSK6TPTVN1PROD with BILLS
4 

•HR 4132 IH

1 velopment and availability to patients of treatments for 

2 serious or life-threatening diseases or conditions while 

3 maintaining appropriate safety and effectiveness stand4 ards for such treatments. 

5 SEC. 3. EXPEDITED APPROVAL OF DRUGS FOR SERIOUS OR 

6 LIFE-THREATENING DISEASES OR CONDI7 TIONS. 

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

9 Act (21 U.S.C. 356) is amended to read as follows: 

10 ‘‘SEC. 506. EXPEDITED APPROVAL OF DRUGS FOR SERIOUS 

11 OR LIFE-THREATENING DISEASES OR CONDI12 TIONS. 

13 ‘‘(a) DESIGNATION OF DRUG AS A FAST TRACK

14 PRODUCT.— 

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

16 request of the sponsor of a new drug, facilitate the 

17 development and expedite the review of such drug if 

18 it is intended, whether alone or in combination with 

19 one or more other drugs, for the treatment of a seri20 ous or life-threatening disease or condition, and it 

21 demonstrates the potential to address unmet medical 

22 needs for such a disease or condition. (In this sec23 tion, such a drug is referred to as a ‘fast track prod24 uct’.) 


smartinez on DSK6TPTVN1PROD with BILLS
5 

•HR 4132 IH

1 ‘‘(2) REQUEST FOR DESIGNATION.—The spon2 sor of a new drug may request the Secretary to des3 ignate the drug as a fast track product. A request 

4 for the designation may be made concurrently with, 

5 or at any time after, submission of an application 

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

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

8 Act. 

9 ‘‘(3) DESIGNATION.—Within 60 calendar days 

10 after the receipt of a request under paragraph (2), 

11 the Secretary shall determine whether the drug that 

12 is the subject of the request meets the criteria de13 scribed in paragraph (1). If the Secretary finds that 

14 the drug meets the criteria, the Secretary shall des15 ignate the drug as a fast track product and shall 

16 take such actions as are appropriate to expedite the 

17 development and review of the application for ap18 proval of such product. 

19 ‘‘(b) ACCELERATED APPROVAL OF A DRUG FOR A

20 SERIOUS OR LIFE-THREATENING DISEASE OR CONDI21 TION, INCLUDING A FAST TRACK PRODUCT.— 

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

23 an application for approval of a product for a seri24 ous or life-threatening disease or condition, including 

25 a fast track product, under section 505(c) or section 


smartinez on DSK6TPTVN1PROD with BILLS
6 

•HR 4132 IH

1 351(a) of the Public Health Service Act upon mak2 ing a determination (taking into account the severity 

3 or rarity of the disease or condition and the avail4 ability of alternative treatments) that the product 

5 has an effect on— 

6 ‘‘(A) a surrogate endpoint that is reason7 ably likely to predict clinical benefit; or 

8 ‘‘(B) a clinical endpoint, including an end9 point that can be measured earlier than irre10 versible morbidity or mortality, that is reason11 ably likely to predict an effect on irreversible 

12 morbidity or mortality or other clinical benefit. 

13 The evidence to support that an endpoint is reason14 ably likely to predict clinical benefit may include epi15 demiological, pathophysiologic, pharmacologic, thera16 peutic or other evidence developed using, for exam17 ple, biomarkers, or other scientific methods or tools. 

18 ‘‘(2) LIMITATION.—Approval of a product 

19 under this subsection may, as determined by the 

20 Secretary, be subject to the following require21 ments— 

22 ‘‘(A) that the sponsor conduct appropriate 

23 post-approval studies to verify and describe the 

24 predicted effect of the product on irreversible 


smartinez on DSK6TPTVN1PROD with BILLS
7 

•HR 4132 IH

1 morbidity or mortality or other clinical benefit; 

2 and 

3 ‘‘(B) that the sponsor submit copies of all 

4 promotional materials related to the product, at 

5 least 30 days prior to dissemination of the ma6 terials during— 

7 ‘‘(i) the preapproval review period; 

8 and 

9 ‘‘(ii) following approval, for a period 

10 that the Secretary determines to be appro11 priate. 

12 ‘‘(3) EXPEDITED WITHDRAWAL OF AP13 PROVAL.—The Secretary may withdraw approval of 

14 a product approved pursuant to this subsection 

15 using expedited procedures (as prescribed by the 

16 Secretary in regulations, which shall include an op17 portunity for an informal hearing) if— 

18 ‘‘(A) the sponsor fails to conduct any re19 quired post-approval study of the product with 

20 due diligence; 

21 ‘‘(B) a study required to verify and de22 scribe the predicted effect on irreversible mor23 bidity or mortality or other clinical benefit of 

24 the product fails to verify and describe such ef25 fect or benefit; 


smartinez on DSK6TPTVN1PROD with BILLS
8 

•HR 4132 IH

1 ‘‘(C) other evidence demonstrates that the 

2 product is not safe or effective under the condi3 tions of use; or 

4 ‘‘(D) the sponsor disseminates false or 

5 misleading promotional materials with respect 

6 to the product. 

7 ‘‘(c) REVIEW OF INCOMPLETE APPLICATIONS FOR

8 APPROVAL OF A FAST TRACK PRODUCT.— 

9 ‘‘(1) IN GENERAL.—If the Secretary deter10 mines, after preliminary evaluation of clinical data 

11 submitted by the sponsor, that a fast track product 

12 may be effective, the Secretary shall evaluate for fil13 ing, and may commence review of portions of, an ap14 plication for the approval of the product before the 

15 sponsor submits a complete application. The Sec16 retary shall commence such review only if the appli17 cant— 

18 ‘‘(A) provides a schedule for submission of 

19 information necessary to make the application 

20 complete; and 

21 ‘‘(B) pays any fee that may be required 

22 under section 736. 

23 ‘‘(2) EXCEPTION.—Any time period for review 

24 of human drug applications that has been agreed to 

25 by the Secretary and that has been set forth in goals 


smartinez on DSK6TPTVN1PROD with BILLS
9 

•HR 4132 IH

1 identified in letters of the Secretary (relating to the 

2 use of fees collected under section 736 to expedite 

3 the drug development process and the review of 

4 human drug applications) shall not apply to an ap5 plication submitted under paragraph (1) until the 

6 date on which the application is complete. 

7 ‘‘(d) AWARENESS EFFORTS.—The Secretary shall— 

8 ‘‘(1) develop and disseminate to physicians, pa9 tient organizations, pharmaceutical and bio10 technology companies, and other appropriate persons 

11 a description of the provisions of this section appli12 cable to accelerated approval and fast track prod13 ucts; and 

14 ‘‘(2) establish a program to encourage the de15 velopment of surrogate and clinical endpoints, in16 cluding biomarkers, and other scientific methods and 

17 tools that can assist the Secretary in determining 

18 whether the evidence submitted in an application is 

19 reasonably likely to predict clinical benefit for seri20 ous or life-threatening conditions for which there 

21 exist significant unmet medical needs.’’. 

22 SEC. 4. GUIDANCE; AMENDED REGULATIONS. 

23 (a) INITIAL GUIDANCE.—Not later than one year 

24 after the date of enactment of this Act, the Secretary of 

25 Health and Human Services (hereinafter ‘‘the Secretary’’) 


smartinez on DSK6TPTVN1PROD with BILLS
10 

•HR 4132 IH

1 shall issue draft guidance to implement the amendments 

2 made by section 3. 

3 (b) FINAL GUIDANCE.—Not later than one year after 

4 the issuance of draft guidance under subsection (a), after 

5 an opportunity for public comment, the Secretary shall 

6 issue— 

7 (1) final guidance to implement the amend8 ments made by section 3; and 

9 (2) amend the regulations governing accelerated 

10 approval in parts 314 and 601 of title 21, Code of 

11 Federal Regulations, as necessary to conform such 

12 regulations with the amendments made by section 3. 

13 (c) CONSIDERATIONS.—In developing the guidance 

14 under subsections (a) and (b)(1) and the amendments 

15 under subsection (b)(2), the Secretary shall consider— 

16 (1) issues arising under the accelerated ap17 proval and fast track processes under section 506 of 

18 the Federal Food, Drug, and Cosmetic Act (as 

19 amended by section 3) for drugs designated for a 

20 rare disease or condition under section 526 of the 

21 Federal, Food, Drug, and Cosmetic Act; and 

22 (2) how to incorporate novel approaches to the 

23 review of surrogate endpoints based on patho24 physiologic and pharmacologic evidence in such guid25 ance, especially in instances where the low prevaVerDate Mar 15 2010 23:54 Mar 05, 2012 Jkt 019200 PO 00000 Frm 00010 Fmt 6652 Sfmt 6201 E:\BILLS\H4132.IH H4132

smartinez on DSK6TPTVN1PROD with BILLS
11 

•HR 4132 IH

1 lence of a disease renders the existence or collection 

2 of other types of data unlikely or impractical. 

3 (d) NO DELAY IN REVIEW OR APPROVAL.—The 

4 issuance (or non-issuance) of guidance or conforming reg5 ulations implementing the amendments made by section 

6 3 shall not preclude the review of, or action on, a request 

7 for designation or an application for approval submitted 

8 pursuant to section 506 of the Federal Food, Drug, and 

9 Cosmetic Act, as amended by section 3. 

10 SEC. 5. INDEPENDENT REVIEW. 

11 (a) IN GENERAL.—The Secretary shall, in conjunc12 tion with other planned reviews of the new drug review 

13 process, contract with an independent entity with expertise 

14 in assessing the quality and efficiency of biopharma15 ceutical development and regulatory review programs, to 

16 evaluate the Food and Drug Administration’s application 

17 of the processes described in section 506 of the Federal 

18 Food, Drug, and Cosmetic Act, as amended by section 3, 

19 and the impact of such processes on the development and 

20 timely availability of innovative treatments for patients 

21 suffering from serious or life-threatening conditions. 

22 (b) CONSULTATION.—Any evaluation under sub23 section (a) shall include consultation with regulated indus24 tries, patient advocacy and disease research foundations, 

25 and relevant academic medical centers. 


smartinez on DSK6TPTVN1PROD with BILLS
12 

•HR 4132 IH

1 SEC. 6. RULE OF CONSTRUCTION. 

2 The amendments made to section 506(b) of the Fed3 eral Food, Drug and Cosmetic Act by this Act shall be 

4 construed in a manner that encourages the Secretary to 

5 utilize innovative approaches for the assessment of prod6 ucts under accelerated approval while maintaining appro7 priate safety and effectiveness standards for such prod8 ucts. 

Æ 


smartinez on DSK6TPTVN1PROD with BILLS