II 

112TH CONGRESS 

2D SESSION S. 2113 

To empower the Food and Drug Administration to ensure a clear and effective 

pathway that will encourage innovative products to benefit patients and 

improve public health. 

IN THE SENATE OF THE UNITED STATES 

FEBRUARY 15, 2012 

Mrs. HAGAN introduced the following bill; which was read twice and referred 

to the Committee on Health, Education, Labor, and Pensions 

A BILL 

To empower the Food and Drug Administration to ensure 

a clear and effective pathway that will encourage innovative products to benefit patients and improve public 

health. 

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; TABLE OF CONTENTS; REF4 ERENCES IN ACT. 

5 (a) SHORT TITLE.—This Act may be cited as the 

6 ‘‘Transforming the Regulatory Environment to Accelerate 

7 Access to Treatments’’ or ‘‘TREAT Act’’. 


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1 (b) TABLE OF CONTENTS.—The table of contents of 

2 this Act is as follows: 

Sec. 1. Short title; table of contents; references in Act. 

TITLE I—ELEVATING FDA AND EMPOWERING OPERATIONAL 

EXCELLENCE 

Sec. 101. Mission statement. 

Sec. 102. Management Review Board. 

TITLE II—ADVANCING REGULATORY SCIENCE AND INNOVATION 

Sec. 201. Chief innovation officer. 

Sec. 202. Enhancing access to external scientific and medical expertise. 

TITLE III—ENABLING MODERNIZED PATIENT-CENTRIC CLINICAL 

DEVELOPMENT 

Sec. 301. Enhancement of accelerated patient access to new medical treatments. 

Sec. 302. Electronic health records. 

Sec. 303. Disclosure to drug sponsors of reasons for non-approval of a new 

drug application. 

3 (c) REFERENCES IN ACT.—Except as otherwise spec4 ified, amendments made by this Act to a section or other 

5 provision of law are amendments to such section or other 

6 provision of the Federal Food, Drug, and Cosmetic Act 

7 (21 U.S.C. 301 et seq.). 

8 TITLE I—ELEVATING FDA AND 

9 EMPOWERING OPERATIONAL 

10 EXCELLENCE 

11 SEC. 101. MISSION STATEMENT. 

12 Section 1003(b) (21 U.S.C. 393(b)) is amended— 

13 (1) by redesignating paragraphs (3) and (4) as 

14 paragraphs (4) and (5), respectively; 

15 (2) by inserting after paragraph (2), the fol16 lowing: 


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1 ‘‘(3) advance medical innovation, and strive to 

2 make novel products available to those who need 

3 them, by incorporating modern scientific tools, 

4 standards, methodologies, and approaches to ensure 

5 the timely and effective review, and the expeditious 

6 clearance, licensure, or approval, as appropriate, of 

7 innovative drugs, devices, and other regulated prod8 ucts;’’; and 

9 (3) in paragraph (5), as so redesignated, by 

10 striking ‘‘(1) through (3)’’ and inserting ‘‘(1) 

11 through (4)’’. 

12 SEC. 102. MANAGEMENT REVIEW BOARD. 

13 Chapter VII (21 U.S.C. 371 et seq.) is amended by 

14 inserting after section 713 the following: 

15 ‘‘SEC. 714. MANAGEMENT REVIEW BOARD. 

16 ‘‘(a) IN GENERAL.—Not later than 60 days after the 

17 date of enactment of the TREAT Act, the Secretary shall 

18 establish an advisory council within the Food and Drug 

19 Administration to be known as the Management Review 

20 Board (referred to in this section as the ‘Board’). 

21 ‘‘(b) DUTIES.— 

22 ‘‘(1) IN GENERAL.—The Board shall provide 

23 advice to the Secretary regarding the management 

24 and organization of the Food and Drug Administra25 tion. 


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1 ‘‘(2) REPORTS.—The Board shall— 

2 ‘‘(A) periodically review the organization 

3 and responsibilities of individual offices, cen4 ters, and divisions within the Food and Drug 

5 Administration (referred to in this section as 

6 the ‘Administration’) in order to determine the 

7 optimal allocation of responsibilities and to im8 prove the efficiency and effectiveness of each of9 fice, center, and division in achieving individual 

10 and overall missions of the Administration; 

11 ‘‘(B) issue proposed and final reports on 

12 whether and to what extent changes should be 

13 made to the management and organization of 

14 the Administration to further the Administra15 tion’s mission as set forth in section 1003(b); 

16 and 

17 ‘‘(C) for any proposal for organizational 

18 changes to which the Board gives significant 

19 consideration as a recommendation, consider— 

20 ‘‘(i) the budgetary and operational 

21 consequences of the proposed change; and 

22 ‘‘(ii) an estimation of the level of re23 sources that would be needed to implement 

24 the proposed change. 


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1 ‘‘(3) CONSULTATION.—In carrying out para2 graph (2), the Board shall consult with— 

3 ‘‘(A) the heads of centers and divisions 

4 within the Administration who are not members 

5 of the Board; 

6 ‘‘(B) other scientific leaders who are offi7 cers or employees of the Administration and are 

8 not members of the Board; and 

9 ‘‘(C) organizations representing regulated 

10 industries, venture capital, patients, and disease 

11 research, and that are not otherwise rep12 resented on the Board. 

13 ‘‘(4) TOPICS FOR REVIEW.— 

14 ‘‘(A) REQUEST OF SECRETARY.—The Sec15 retary may, at any time, submit requests about 

16 management or organizational issues to the 

17 Board for assessment. 

18 ‘‘(B) PUBLIC INPUT.—The Board shall 

19 seek input from the public on management and 

20 organizational issues that should be assessed by 

21 the Board, at such times as determined appro22 priate by the Board. 

23 ‘‘(5) POWERS.—The Board may secure directly 

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1 essary or appropriate for the Board to review issues 

2 under consideration. 

3 ‘‘(6) CONFLICT OF INTEREST EXEMPTION.— 

4 Notwithstanding any other provision of law, the 

5 Board shall not be subject to section 712. 

6 ‘‘(c) COMPOSITION OF BOARD.— 

7 ‘‘(1) IN GENERAL.—The Board shall consist 

8 of— 

9 ‘‘(A) the Secretary, who shall be a perma10 nent nonvoting member on an ex officio basis; 

11 and 

12 ‘‘(B) 21 additional members, all of whom 

13 shall be voting members, in accordance with 

14 paragraph (2). 

15 ‘‘(2) VOTING MEMBERS.—The membership of 

16 the Board shall consist of the following: 

17 ‘‘(A) OFFICERS AND EMPLOYEES OF THE

18 FOOD AND DRUG ADMINISTRATION.—The Sec19 retary shall designate not less than 9 individ20 uals who are directors of centers within the Ad21 ministration, directors of divisions within such 

22 Administration, or other similarly senior offi23 cials within such Administration. 

24 ‘‘(B) OTHER MEMBERS.—The Secretary 

25 shall designate other individuals from among 


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1 individuals who are not officers or employees of 

2 the United States. Such members shall in3 clude— 

4 ‘‘(i) individuals representing the inter5 ests of public or private academic medical 

6 centers, physicians, and patient advocacy 

7 and disease research organizations; 

8 ‘‘(ii) individuals representing the in9 terests of industries regulated by the Ad10 ministration, which shall include at least 1 

11 representative from each of the pharma12 ceutical, biotechnology, medical device, and 

13 food industries; and 

14 ‘‘(iii) individuals with broad expertise 

15 regarding how the Administration func16 tions and with experience in successfully 

17 managing or consulting for large scientific 

18 research or other organizations (other than 

19 public or private entities described under 

20 clause (i)). 

21 ‘‘(3) TERM; VACANCIES.— 

22 ‘‘(A) TERMS.—The members appointed 

23 under paragraph (2)(B) shall be appointed for 

24 a term of 3 years, which may be renewed once. 


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1 ‘‘(B) VACANCIES.—A vacancy on the 

2 Board— 

3 ‘‘(i) shall not affect the powers of the 

4 Board; and 

5 ‘‘(ii) shall be filled in the same man6 ner as the original appointment was made. 

7 ‘‘(d) CHAIR.—The Chair of the Board shall be se8 lected by the Secretary from among the members of the 

9 Board appointed under subsection (c)(1). The term of of10 fice of the Chair shall be 3 years. 

11 ‘‘(e) MEETINGS.— 

12 ‘‘(1) IN GENERAL.—The Board shall meet at 

13 the call of the Chair or upon the request of the Sec14 retary, but not fewer than 6 times with respect to 

15 issuing any particular report under subsection 

16 (b)(2). The location of the meetings of the Board is 

17 subject to the approval of the Secretary. 

18 ‘‘(2) PARTICULAR MEETINGS TO RECEIVE PUB19 LIC INPUT.—Of the meetings held under paragraph 

20 (1) with respect to proposals for management or or21 ganizational changes being considered under sub22 section (b)(2)— 

23 ‘‘(A) 1 or more shall be directed towards 

24 receiving input from the pharmaceutical, med25 ical device, and biotechnology industries, clinical 


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1 researchers, and the physician and medical re2 search communities to address regulatory and 

3 scientific needs and opportunities related to 

4 such proposals; 

5 ‘‘(B) 1 or more shall be directed towards 

6 receiving input from patient advocacy, disease 

7 research organizations, and consumer groups to 

8 address patient and consumer needs and oppor9 tunities related to such proposals; and 

10 ‘‘(C) 1 or more shall be directed towards 

11 receiving input from food, cosmetic, and dietary 

12 supplement industries to address regulatory and 

13 scientific needs and opportunities related to 

14 such proposals. 

15 ‘‘(3) AVAILABILITY OF INFORMATION.—For 

16 each meeting held under this subsection, the Sec17 retary shall post on the Internet Web site of the Ad18 ministration a summary of the proceedings. 

19 ‘‘(f) COMPENSATION.—Without regard to the provi20 sions of title 5, United States Code, governing appoint21 ments in the competitive service, and without regard to 

22 provisions of chapter 51 and subchapter III of chapter 53 

23 of such title relating to classification and General Schedule 

24 pay rates, the Secretary may— 

25 ‘‘(1) establish the Board; and 


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1 ‘‘(2) appoint and fix the compensation of the 

2 members of the Board, except that officers and em3 ployees of the United States shall not receive addi4 tional compensation for service as members of such 

5 groups. 

6 ‘‘(g) REPORTS.— 

7 ‘‘(1) PUBLIC COMMENT.— 

8 ‘‘(A) PROPOSED REPORTS.—Each pro9 posed report issued under subsection (b)(2) 

10 shall be posted on the Internet Web site of the 

11 Administration and made available for public 

12 comment for not less than 60 days prior to 

13 being made final and being submitted under 

14 paragraph (2). 

15 ‘‘(B) FINAL REPORTS.—Not later than 90 

16 days after receiving comments from the public 

17 on a proposed report under subparagraph (A), 

18 the Board shall post a final report on such 

19 Internet Web site incorporating an overview of 

20 comments accepted or rejected. 

21 ‘‘(2) CONGRESSIONAL AND SECRETARY RE22 VIEW.—Each final report issued under subsection 

23 (b)(2) shall be submitted to the— 


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1 ‘‘(A) the Committee on Health, Education, 

2 Labor, and Pensions and the Committee on Ap3 propriations of the Senate; 

4 ‘‘(B) the Committee on Energy and Com5 merce and the Committee on Appropriations of 

6 the House of Representatives; and 

7 ‘‘(C) the Secretary. 

8 ‘‘(3) TIMING AND FREQUENCY OF REPORTS.— 

9 Not later than January 31, 2015, the Board shall 

10 issue the first report under subsection (b)(2) and 

11 shall issue subsequent reports not less than once 

12 every 5 years thereafter. 

13 ‘‘(h) PROCESS FOR REVIEW OF RECOMMENDED OR14 GANIZATIONAL OR MANAGEMENT CHANGES.—With re15 spect to recommendations for organizational or manage16 ment changes made in a report issued under subsection 

17 (b)(2), the Secretary shall, except as provided in sub18 section (i)(2), implement the recommendations in accord19 ance with the following process: 

20 ‘‘(1) Not later than 100 days after the report 

21 is submitted to the Secretary under subsection 

22 (g)(2), the Secretary shall initiate the applicable 

23 processes under subsection (i). 

24 ‘‘(2) The recommendations shall be fully imple25 mented not later than the expiration of the 3-year 


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1 period beginning on the date on which such process 

2 is initiated. 

3 ‘‘(i) ACTION BY THE SECRETARY.— 

4 ‘‘(1) IN GENERAL.—Not less than 60 days prior 

5 to implementing any major organizational or man6 agement change recommended under subsection 

7 (b)(2), the Secretary shall provide notice to the con8 gressional committees specified in subsection (g)(2) 

9 of the Secretary’s agreement with the recommenda10 tion and the timeline for implementation. 

11 ‘‘(2) OBJECTION.—Subsection (h) shall not 

12 apply to a recommendation for an organizational or 

13 management change made in a report issued under 

14 subsection (b)(2) if, not later than 90 days after the 

15 report is submitted to the Secretary under sub16 section (g)(2), the Secretary submits to the commit17 tees specified in such subsection a notice indicating 

18 that the Secretary objects to the recommended 

19 change, and setting forth the reasons for such objec20 tion. For purposes of this paragraph, an objection 

21 by the Secretary may be made to the entirety of the 

22 recommended organizational changes contained in a 

23 report issued under subsection (b)(2), or to 1 or 

24 more aspects of any proposed change or changes. 


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1 ‘‘(3) IMPLEMENTATION.—Any aspect of a pro2 posed change not objected to by the Secretary in a 

3 notice under paragraph (2) shall be implemented in 

4 accordance with subsection (h), except as the Sec5 retary may be directed otherwise by law.’’. 

6 TITLE II—ADVANCING REGU7 LATORY SCIENCE AND INNO8 VATION 

9 SEC. 201. CHIEF INNOVATION OFFICER. 

10 Chapter X is amended— 

11 (1) by redesignating the second section 1011 

12 (21 U.S.C. 399c) (as added by section 209(a) of 

13 Public Law 111–353) as section 1011A; and 

14 (2) by adding at the end the following: 

15 ‘‘SEC. 1013. OFFICE OF THE CHIEF INNOVATION OFFICER. 

16 ‘‘(a) ESTABLISHMENT; APPOINTMENT.—The Sec17 retary shall establish within the Office of the Commis18 sioner an office to be known as the Office of the Chief 

19 Innovation Officer. The Secretary shall appoint a Chief 

20 Innovation Officer to lead such Office. 

21 ‘‘(b) DUTIES.—The Chief Innovation Officer shall— 

22 ‘‘(1) identify promising new scientific and regu23 latory approaches to ensure the rapid development, 

24 testing, and review of new drugs and devices, which 

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1 markers, the adoption of novel models or methodolo2 gies to enhance clinical trial design, clinical data 

3 evaluation, or predictive toxicology, and the coordi4 nation and optimization of efficient review processes 

5 for drugs, and devices; 

6 ‘‘(2) ensure that such approaches are integrated 

7 into operations at all applicable levels of the Food 

8 and Drug Administration, and harmonized with the 

9 approaches of other applicable agencies; 

10 ‘‘(3)(A) consider the recommendations of inter11 nal and external bodies involved in advancing inno12 vation in regulatory science activities, such as those 

13 described in paragraph (1); and 

14 ‘‘(B) make such recommendations available on 

15 the Internet Web site of the Food and Drug Admin16 istration; 

17 ‘‘(4) develop pilot programs to implement and 

18 incorporate the recommendations considered under 

19 paragraph (3) into the regulatory review and ap20 proval processes of such Administration; and 

21 ‘‘(5) in consultation with the heads of the cen22 ters and offices within such Administration, imple23 ment other pilot programs as the Chief Innovation 

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1 pation by cross-disciplinary teams in such implemen2 tation, as applicable. 

3 ‘‘(c) REPORTS AND IMPLEMENTATION PLANS.— 

4 ‘‘(1) REPORTS.—The Chief Innovation Officer 

5 shall publish a report summarizing the consideration 

6 of applicable recommendations evaluated under sub7 section (b)(3) at least once every 2 years. Such re8 ports shall— 

9 ‘‘(A) provide an explanation as to whether, 

10 how, and why such recommendations will be im11 plemented by the Food and Drug Administra12 tion; 

13 ‘‘(B) provide a description of pilot pro14 grams being implemented and the progress of 

15 such Administration with respect to the integra16 tion of new scientific and regulatory approaches 

17 into its operations in order to accelerate the 

18 rapid development, review, approval, and pa19 tient access to new drugs and devices; 

20 ‘‘(C) be made available for public comment 

21 for not less than 60 days prior to being made 

22 final; 

23 ‘‘(D) following public comment, be final24 ized by the Chief Innovation Officer to include 


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1 an overview of public comments accepted or re2 jected; and 

3 ‘‘(E) once finalized, be made available on 

4 the Internet Web site of such Administration 

5 and submitted to— 

6 ‘‘(i) the Committee on Health, Edu7 cation, Labor and Pensions of the Senate; 

8 and 

9 ‘‘(ii) the Committee on Energy and 

10 Commerce of the House of Representa11 tives. 

12 ‘‘(2) PUBLIC COMMENT REGARDING IMPLEMEN13 TATION OF PILOT PROGRAMS.—The Chief Innova14 tion Officer shall make each plan to implement a 

15 pilot program under subsection (b)(4) available for 

16 public comment for not less than 60 days before the 

17 implementation of the pilot program. 

18 ‘‘(d) MAINTENANCE OF AUTHORITY OF CENTERS.— 

19 Nothing in this section limits the authority or ability of 

20 the individual Centers of the Food and Drug Administra21 tion to carry out any of the actions described in this sec22 tion.’’. 

23 SEC. 202. ENHANCING ACCESS TO EXTERNAL SCIENTIFIC 

24 AND MEDICAL EXPERTISE. 

25 (a) ADVISORY COMMITTEES.— 


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1 (1) CONFLICTS OF INTEREST.—Section 

2 712(c)(2) (21 U.S.C. 379d–1(c)(2)) is amended— 

3 (A) in subparagraph (A), by striking ‘‘fi4 nancial interest that could be affected by the 

5 advice given to the Secretary with respect to 

6 such matter’’ and inserting ‘‘financial interest 

7 in the outcome of such matter that is direct and 

8 predictable’’; 

9 (B) by striking subparagraph (B) and in10 serting the following: 

11 ‘‘(B) WAIVER.— 

12 ‘‘(i) IN GENERAL.—If the Secretary 

13 makes a determination described in clause 

14 (ii), the Secretary may grant a waiver of 

15 the prohibition in subparagraph (A) to per16 mit a member described in such subpara17 graph to— 

18 ‘‘(I) participate as a non-voting 

19 member with respect to a particular 

20 matter considered in a committee 

21 meeting; or 

22 ‘‘(II) participate as a voting 

23 member with respect to a particular 

24 matter considered in a committee 

25 meeting. 


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1 ‘‘(ii) DETERMINATION.—A determina2 tion described under this clause may be 

3 based on 1 or both of the following deter4 minations: 

5 ‘‘(I) The need for the services of 

6 the individual on the committee out7 weighs the potential for a conflict of 

8 interest created by the financial inter9 est involved. 

10 ‘‘(II) The financial interest is not 

11 so substantial as to be deemed likely 

12 to affect the integrity of the services 

13 provided by that individual.’’; and 

14 (C) by striking subparagraph (C). 

15 (2) PATIENT GROUP REPRESENTATIVES.—Sec16 tion 505(n)(3) (21 U.S.C. 355(n)(3)) is amended— 

17 (A) in subparagraph (C), by striking ‘‘; 

18 and’’ and inserting a semicolon; 

19 (B) in subparagraph (D), by striking the 

20 period at the end and inserting ‘‘; and’’; and 

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

22 ‘‘(E) 2 or more members who are medical 

23 or scientific experts selected from a pool of 

24 nominations provided by patient advocacy or 

25 disease research organizations whose interests 


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1 are in the specific disease or diseases proposed 

2 to be treated by the drug under consideration.’’. 

3 (3) REVISED REGULATIONS.— 

4 (A) IN GENERAL.—The Secretary of 

5 Health and Human Services shall revise and 

6 update the regulations of the Food and Drug 

7 Administration relating to the application of the 

8 Federal Advisory Committee Act (5 U.S.C. 

9 App.) to reflect updated understanding of the 

10 scope of such Act, as embodied in regulations of 

11 the General Services Administration (as in ef12 fect on the date of enactment of this Act) and 

13 case law. 

14 (B) CONTENT.—The revised and updated 

15 regulations under subparagraph (A) shall ex16 plicitly encourage officials of the Food and 

17 Drug Administration to utilize, to the maximum 

18 extent possible, the flexibility and exceptions 

19 provided by the Federal Advisory Committee 

20 Act to interact with stakeholder groups outside 

21 the confines of the advisory committees of such 

22 Act, including patient advocacy organizations, 

23 disease specialty societies, and others. 


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1 (b) CHIEF MEDICAL POLICY OFFICERS.—Chapter X 

2 (21 U.S.C. 391 et seq.), as amended by section 201, is 

3 further amended by adding at the end the following: 

4 ‘‘SEC. 1014. CHIEF MEDICAL POLICY OFFICERS. 

5 ‘‘(a) ESTABLISHMENT.—The Secretary shall estab6 lish an Office of the Chief Medical Policy Officer within 

7 each of the following Offices of the Food and Drug Admin8 istration: 

9 ‘‘(1) The Office of the Director of the Center 

10 for Drug Evaluation and Research. 

11 ‘‘(2) The Office of the Director of the Center 

12 for Biologics Evaluation and Research. 

13 ‘‘(3) The Office of the Director of the Center 

14 for Devices and Radiological Health. 

15 ‘‘(b) SELECTION.—Each Chief Medical Policy Officer 

16 shall be selected from the Senior Executive Service by the 

17 Secretary. 

18 ‘‘(c) DUTIES.—Each Chief Medical Policy Officer 

19 shall— 

20 ‘‘(1) in coordination with the Chief Innovation 

21 Officer, center Directors, and other Chief Medical 

22 Policy Officers, develop proactive and consistent ap23 proaches for the centers within the Food and Drug 

24 Administration and the divisions within such Admin25 istration that review applications for drug or device 


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1 approval to address emerging medical and scientific 

2 policy issues bearing on new product review proc3 esses, including by— 

4 ‘‘(A) advising on and regularly reviewing 

5 the implementation of such approaches by such 

6 centers and divisions; and 

7 ‘‘(B) implementing peer learning programs 

8 to ensure the effective and consistent review 

9 and approval of new drugs and devices, includ10 ing the incorporation of new scientific and regu11 latory approaches recommended by the Chief 

12 Innovation Officer under section 1013(b); 

13 ‘‘(2) in coordination with the center Directors, 

14 sponsors, and relevant patient advocacy and disease 

15 research organizations, promote earlier and im16 proved utilization of advisory committees throughout 

17 the drug and device development and review proc18 esses, including at the investigational testing phase, 

19 and recommend as appropriate the utilization of au20 thorities by the Secretary under section 1007 in 

21 cases where the ability to obtain sufficient external 

22 experts for such advisory committees is limited; 

23 ‘‘(3) in coordination with the Office of Special 

24 Medical Programs and appropriate Center medical 

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1 ternal experts outside of the advisory committee 

2 process, including utilization of authorities in section 

3 1004; 

4 ‘‘(4) periodically solicit input from industry, 

5 academia, and patient advocacy and disease research 

6 organizations on emerging scientific and medical pol7 icy issues bearing on new product review processes, 

8 including clinical trial methodologies; and 

9 ‘‘(5) coordinate with the Chief Innovation Offi10 cer in the implementation of pilot programs under 

11 section 1013(b). 

12 ‘‘(d) EXTERNAL EXPERTS.—When serving as officers 

13 or employees of the United States, the experts described 

14 under subsection (c)(3) shall be considered special govern15 ment employees as defined in section 202(a) of title 18, 

16 United States Code.’’. 

17 TITLE III—ENABLING MODERN18 IZED PATIENT-CENTRIC CLIN19 ICAL DEVELOPMENT 

20 SEC. 301. ENHANCEMENT OF ACCELERATED PATIENT AC21 CESS TO NEW MEDICAL TREATMENTS. 

22 (a) FINDINGS; SENSE OF CONGRESS.— 

23 (1) FINDINGS.—Congress makes the following 

24 findings: 


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1 (A) The Food and Drug Administration 

2 (referred to in this section as the ‘‘FDA’’) 

3 serves a critical role in helping to assure that 

4 new medicines are safe and effective. Regu5 latory innovation is 1 element of the Nation’s 

6 strategy to address serious and life-threatening 

7 diseases or conditions by promoting investment 

8 in and development of innovative treatments for 

9 unmet medical needs. 

10 (B) During the 2 decades following the es11 tablishment of the accelerated approval mecha12 nism, advances in medical sciences, including 

13 genomics, molecular biology, and bioinformatics, 

14 have provided an unprecedented understanding 

15 of the underlying biological mechanism and 

16 pathogenesis of disease. A new generation of 

17 modern, targeted medicines is under develop18 ment to treat serious and life-threatening dis19 eases, some applying drug development strate20 gies based on biomarkers or pharmacogenomics, 

21 predictive toxicology, clinical trial enrichment 

22 techniques, and novel clinical trial designs, such 

23 as adaptive clinical trials. 

24 (C) As a result of these remarkable sci25 entific and medical advances, the FDA should 


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1 be encouraged to implement more broadly effec2 tive processes for the expedited development 

3 and review of innovative new medicines in4 tended to address unmet medical needs for seri5 ous or life-threatening diseases or conditions, 

6 including those for rare diseases or conditions, 

7 using a broad range of surrogate or clinical 

8 endpoints and modern scientific tools earlier in 

9 the drug development cycle when appropriate. 

10 This may result in fewer, smaller, or shorter 

11 clinical trials for the intended patient popu12 lation or targeted subpopulation without com13 promising or altering the high standards of the 

14 FDA for the approval of drugs. 

15 (D) Patients benefit from expedited access 

16 to safe and effective innovative therapies to 

17 treat unmet medical needs for serious or life18 threatening diseases or conditions. 

19 (E) For these reasons, the statutory au20 thority in effect on the day before the date of 

21 enactment of this Act governing expedited ap22 proval of drugs for serious or life-threatening 

23 diseases or conditions should be amended in 

24 order to enhance the authority of the FDA to 

25 consider appropriate scientific data, methods, 


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1 and tools, and to expedite development and ac2 cess to novel treatments for patients with a 

3 broad range of serious or life-threatening dis4 eases or conditions. 

5 (2) SENSE OF CONGRESS.—It is the sense of 

6 Congress that the Food and Drug Administration 

7 should apply the accelerated approval and fast track 

8 provisions set forth in section 506 of the Federal 

9 Food, Drug, and Cosmetic Act (21 U.S.C. 356), as 

10 amended by this section, to the greatest extent pos11 sible to help expedite the development and avail12 ability to patients of treatments for serious or life13 threatening diseases or conditions while maintaining 

14 appropriate safety and effectiveness standards for 

15 such treatments. 

16 (b) EXPEDITED APPROVAL OF DRUGS FOR SERIOUS

17 OR LIFE-THREATENING DISEASES OR CONDITIONS.—Sec18 tion 506 of the Federal Food, Drug, and Cosmetic Act 

19 (21 U.S.C. 356) is amended to read as follows: 

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

21 OR LIFE-THREATENING DISEASES OR CONDI22 TIONS. 

23 ‘‘(a) DESIGNATION OF DRUG AS FAST TRACK PROD24 UCT.— 


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

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

3 development and expedite the review of such drug if 

4 it is intended, whether alone or in combination with 

5 one or more other drugs, for the treatment of a seri6 ous or life-threatening disease or condition, and it 

7 demonstrates the potential to address unmet medical 

8 needs for such a disease or condition. (In this sec9 tion, such a drug is referred to as a ‘fast track prod10 uct’.) 

11 ‘‘(2) REQUEST FOR DESIGNATION.—The spon12 sor of a new drug may request the Secretary to des13 ignate the drug as a fast track product. A request 

14 for the designation may be made concurrently with, 

15 or at any time after, submission of an application 

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

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

18 Act. 

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

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

21 the Secretary shall determine whether the drug that 

22 is the subject of the request meets the criteria de23 scribed in paragraph (1). If the Secretary finds that 

24 the drug meets the criteria, the Secretary shall des25 ignate the drug as a fast track product and shall 


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1 take such actions as are appropriate to expedite the 

2 development and review of the application for ap3 proval of such product. 

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

5 SERIOUS OR LIFE-THREATENING DISEASE OR CONDI6 TION, INCLUDING A FAST TRACK PRODUCT.— 

7 ‘‘(1) IN GENERAL.— 

8 ‘‘(A) ACCELERATED APPROVAL.—The Sec9 retary may approve an application for approval 

10 of a product for a serious or life-threatening 

11 disease or condition, including a fast track 

12 product, under section 505(c) or section 351(a) 

13 of the Public Health Service Act upon a deter14 mination that the product has an effect on a 

15 surrogate endpoint that is reasonably likely to 

16 predict clinical benefit, or on a clinical end17 point, including an endpoint that can be meas18 ured earlier than irreversible morbidity or mor19 tality, that is reasonably likely to predict an ef20 fect on irreversible morbidity or mortality or 

21 other clinical benefit, taking into account the 

22 severity or rarity of the condition and the avail23 ability of alternative treatments. The approval 

24 described in the preceding sentence is referred 

25 to in this section as ‘accelerated approval’. 


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1 ‘‘(B) EVIDENCE.—The evidence to support 

2 that an endpoint is reasonably likely to predict 

3 clinical benefit under subparagraph (A) may in4 clude epidemiological, pathophysiological, thera5 peutic or other evidence developed using bio6 markers, for example, or other scientific meth7 ods or tools. 

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

9 under this subsection may be subject to 1 or both 

10 of the following requirements: 

11 ‘‘(A) That the sponsor conduct appropriate 

12 post-approval studies to verify and describe the 

13 predicted effect on irreversible morbidity or 

14 mortality or other clinical benefit. 

15 ‘‘(B) That the sponsor submit copies of all 

16 promotional materials related to the product 

17 during the preapproval review period and, fol18 lowing approval and for such period thereafter 

19 as the Secretary determines to be appropriate, 

20 at least 30 days prior to dissemination of the 

21 materials. 

22 ‘‘(3) EXPEDITED WITHDRAWAL OF AP23 PROVAL.—The Secretary may withdraw approval of 

24 a product approved under accelerated approval using 

25 expedited procedures (as prescribed by the Secretary 


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1 in regulations which shall include an opportunity for 

2 an informal hearing) if— 

3 ‘‘(A) the sponsor fails to conduct any re4 quired post-approval study of the drug with due 

5 diligence; 

6 ‘‘(B) a study required to verify and de7 scribe the predicted effect on irreversible mor8 bidity or mortality or other clinical benefit of 

9 the product fails to verify and describe such ef10 fect or benefit; 

11 ‘‘(C) other evidence demonstrates that the 

12 product is not safe or effective under the condi13 tions of use; or 

14 ‘‘(D) the sponsor disseminates false or 

15 misleading promotional materials with respect 

16 to the product. 

17 ‘‘(c) REVIEW OF INCOMPLETE APPLICATIONS FOR

18 APPROVAL OF A FAST TRACK PRODUCT.— 

19 ‘‘(1) IN GENERAL.—If the Secretary deter20 mines, after preliminary evaluation of clinical data 

21 submitted by the sponsor, that a fast track product 

22 may be effective, the Secretary shall evaluate for fil23 ing, and may commence review of portions of, an ap24 plication for the approval of the product before the 

25 sponsor submits a complete application. The SecVerDate Mar 15 2010 23:58 Feb 15, 2012 Jkt 019200 PO 00000 Frm 00029 Fmt 6652 Sfmt 6201 E:\BILLS\S2113.IS S2113

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1 retary shall commence such review only if the appli2 cant— 

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

4 information necessary to make the application 

5 complete; and 

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

7 under section 736. 

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

9 of human drug applications that has been agreed to 

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

11 identified in letters of the Secretary (relating to the 

12 use of fees collected under section 736 to expedite 

13 the drug development process and the review of 

14 human drug applications) shall not apply to an ap15 plication submitted under paragraph (1) until the 

16 date on which the application is complete. 

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

18 ‘‘(1) develop and disseminate to physicians, pa19 tient organizations, pharmaceutical and bio20 technology companies, and other appropriate persons 

21 a description of the provisions of this section appli22 cable to accelerated approval and fast track prod23 ucts; and 

24 ‘‘(2) establish a program to encourage the de25 velopment of surrogate and clinical endpoints, inVerDate Mar 15 2010 23:58 Feb 15, 2012 Jkt 019200 PO 00000 Frm 00030 Fmt 6652 Sfmt 6201 E:\BILLS\S2113.IS S2113

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1 cluding biomarkers, and other scientific methods and 

2 tools that can assist the Secretary in determining 

3 whether the evidence submitted in an application is 

4 reasonably likely to predict clinical benefit for seri5 ous or life-threatening conditions for which signifi6 cant unmet medical needs exist.’’. 

7 (c) GUIDANCE; AMENDED REGULATIONS.— 

8 (1) DRAFT GUIDANCE.—Not later than 1 year 

9 after the date of enactment of this Act, the Sec10 retary of Health and Human Services (referred to in 

11 this section as the ‘‘Secretary’’) shall issue draft 

12 guidance to implement the amendments made by 

13 this section. In developing such guidance, the Sec14 retary shall specifically consider issues arising under 

15 the accelerated approval and fast track processes 

16 under section 506 of the Federal Food, Drug, and 

17 Cosmetic Act, as amended by subsection (b), for 

18 drugs designated for a rare disease or condition 

19 under section 526 of such Act (21 U.S.C. 360bb). 

20 (2) FINAL GUIDANCE.—Not later than 1 year 

21 after the issuance of draft guidance under para22 graph (1), and after an opportunity for public com23 ment, the Secretary shall issue final guidance. 

24 (3) CONFORMING CHANGES.—The Secretary 

25 shall issue, as necessary, conforming amendments to 


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1 the applicable regulations under title 21, Code of 

2 Federal Regulations, governing accelerated approval. 

3 (4) NO EFFECT OF INACTION ON REQUESTS.— 

4 If the Secretary fails to issue final guidance or 

5 amended regulations as required by this subsection, 

6 such failure shall not preclude the review of, or ac7 tion on, a request for designation or an application 

8 for approval submitted pursuant to section 506 of 

9 the Federal Food, Drug, and Cosmetic Act, as 

10 amended by subsection (b). 

11 (d) INDEPENDENT REVIEW.—The Secretary may, in 

12 conjunction with other planned reviews, contract with an 

13 independent entity with expertise in assessing the quality 

14 and efficiency of biopharmaceutical development and regu15 latory review programs to evaluate the Food and Drug Ad16 ministration’s application of the processes described in 

17 section 506 of the Federal Food, Drug, and Cosmetic Act, 

18 as amended by subsection (b), and the impact of such 

19 processes on the development and timely availability of in20 novative treatments for patients suffering from serious or 

21 life-threatening conditions. Any such evaluation shall in22 clude consultation with regulated industries, patient advo23 cacy and disease research foundations, and relevant aca24 demic medical centers. 


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1 (e) CONSTRUCTION.—The amendments made by this 

2 section to section 506(b) of the Federal Food, Drug, and 

3 Cosmetic Act are intended to encourage the Secretary to 

4 utilize innovative approaches to the assessment of prod5 ucts under accelerated approval while maintaining appro6 priate safety and effectiveness standards for such prod7 ucts. 

8 SEC. 302. ELECTRONIC HEALTH RECORDS. 

9 Subchapter A of chapter VII (21 U.S.C. 371 et seq.), 

10 as amended by section 103, is further amended by adding 

11 at the end the following: 

12 ‘‘SEC. 715. CLINICAL INFORMATICS COORDINATOR. 

13 ‘‘(a) IN GENERAL.—The Secretary shall appoint, 

14 within the Office of the Commissioner, a Clinical 

15 Informatics Coordinator. 

16 ‘‘(b) DUTIES.—The Clinical Informatics Coordinator 

17 shall— 

18 ‘‘(1) develop a process to validate the use of 

19 health information technology in clinical research 

20 and encourage the use of new health information 

21 technologies in clinical research protocols; and 

22 ‘‘(2) establish pilot programs to explore and 

23 evaluate the methods of incorporating emerging 

24 health information technology to make the clinical 

25 research process more efficient. 


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1 ‘‘(c) GUIDANCE.—Not later than 1 year after the con2 clusion of the pilot programs described in subsection 

3 (b)(2), the Secretary shall issue guidance for the conduct 

4 of clinical trials incorporating health information tech5 nology. The guidance shall explain how the Food and 

6 Drug Administration will evaluate such information when 

7 reviewing new drug and device applications.’’. 

8 SEC. 303. DISCLOSURE TO DRUG SPONSORS OF REASONS 

9 FOR NON-APPROVAL OF A NEW DRUG APPLI10 CATION. 

11 Section 505 (21 U.S.C. 355) is amended by adding 

12 at the end the following: 

13 ‘‘(w) NOTICE OF REASONS FOR DENIAL OF A NEW

14 DRUG APPLICATION.—If the Secretary denies approval of 

15 a new drug application under this section or of an applica16 tion with respect to a biological product under section 351 

17 of the Public Health Service Act, the Secretary shall pro18 vide to the sponsor of such drug or biological product— 

19 ‘‘(1) a written explanation of the reasons for 

20 denying such application, including an explanation of 

21 the specific reasons the Secretary determines that— 

22 ‘‘(A) the data submitted in the application 

23 are inadequate to support approval of the drug 

24 or biological product; and 


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1 ‘‘(B) labeling, risk evaluation and mitiga2 tion strategies under section 505–1, or post3 approval studies or trials are inadequate to sup4 port a determination that the benefits of ap5 proval outweigh the risks; and 

6 ‘‘(2) to the extent practicable, an explanation of 

7 what data will be required and what endpoints will 

8 need to be met in order to obtain approval.’’. 

Æ 


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