I 

112TH CONGRESS 

2D SESSION H. R. 4156 

To amend the Federal Food, Drug, and Cosmetic Act to strengthen the 

ability of the Food and Drug Administration to seek advice from external 

experts regarding rare diseases, the burden of rare diseases, and the 

unmet medical needs of individuals with rare diseases. 

IN THE HOUSE OF REPRESENTATIVES 

MARCH 7, 2012 

Mr. MARKEY (for himself, Mr. MARINO, and Mr. STEARNS) 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 to 

strengthen the ability of the Food and Drug Administration to seek advice from external experts regarding rare 

diseases, the burden of rare diseases, and the unmet 

medical needs of individuals with rare diseases. 

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 ‘‘Expanding and Pro5 moting Expertise in Review of Rare Treatments Act of 

6 2012’’ or ‘‘EXPERRT Act of 2012’’. 


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1 SEC. 2. FINDINGS. 

2 Congress finds as follows: 

3 (1) Biomedical research is yielding discoveries 

4 that are leading to the development of new therapies 

5 that hold great promise for treating disease. 

6 (2) Scientists are increasingly unlocking the po7 tential for targeting treatments according to genetic 

8 defect. 

9 (3) Many of the new therapies that are under 

10 development in laboratories across the Nation are 

11 targeted to rare diseases, small subsets of diseases 

12 of significant incidence, or even small subsets of rare 

13 diseases. 

14 (4) Progress in the development of targeted 

15 therapies, while of great promise for those with dis16 ease or disability, poses challenges for the Food and 

17 Drug Administration, as the agency is asked to de18 velop or obtain expertise in many diseases and dis19 ease subtypes. 

20 (5) The Food and Drug Administration could 

21 benefit from consultation with external experts who 

22 have a deep understanding of the diseases or disease 

23 subtypes that are targeted by new therapies. 

24 (6) External experts could provide valuable ad25 vice about rare diseases or disease subtypes, disease 

26 severity, and unmet medical needs. 


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1 SEC. 3. CONSULTATION WITH EXTERNAL EXPERTS. 

2 Subchapter E of chapter V of the Federal Food, 

3 Drug, and Cosmetic Act (21 U.S.C. 360bbb et seq.) is 

4 amended by adding at the end the following: 

5 ‘‘SEC. 568. CONSULTATION WITH EXTERNAL EXPERTS ON 

6 RARE DISEASES, TARGETED THERAPIES, AND 

7 GENETIC TARGETING OF TREATMENTS. 

8 ‘‘(a) IN GENERAL.—The Secretary shall establish a 

9 program for consultation with external experts on the top10 ics described in subsection (d), for the purpose of inform11 ing and strengthening the Food and Drug Agency’s review 

12 of drugs and biologic products for rare diseases and drugs 

13 and biologic products that are genetically targeted. 

14 ‘‘(b) PROGRAM OPERATION.—Under the program es15 tablished under subsection (a), each review division within 

16 the Center for Drug Evaluation and Research and the 

17 Center for Biologics Evaluation and Research shall, when 

18 appropriate, seek the opinion of external experts on any 

19 topic described in subsection (d) by initiating contact with 

20 such experts. The external experts may also request the 

21 opportunity to meet with a review division regarding any 

22 topic described in subsection (d). 

23 ‘‘(c) EXTERNAL EXPERTS.—The external experts 

24 under subsection (a) may include— 

25 ‘‘(1) representatives of patient, consumer, re26 search, and health professional organizations; 


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1 ‘‘(2) experts on rare diseases, rare subtypes of 

2 rare and other diseases, and genetic targeting of 

3 treatments, including experts from academia; and 

4 ‘‘(3) experts in innovative clinical trial designs 

5 for small target populations. 

6 ‘‘(d) TOPICS FOR CONSULTATION.—Topics for con7 sultation may include— 

8 ‘‘(1) rare diseases; 

9 ‘‘(2) the severity of rare diseases; 

10 ‘‘(3) the unmet medical need associated with 

11 rare diseases; 

12 ‘‘(4) the willingness and ability of individuals 

13 with a rare disease to participate in clinical trials; 

14 ‘‘(5) an assessment of the benefits and risks, 

15 including side effects, of current and investigational 

16 therapies; 

17 ‘‘(6) the design of clinical trials for rare disease 

18 populations and subpopulations; and 

19 ‘‘(7) demographics and the clinical description 

20 of patient populations. 

21 ‘‘(e) TIMING OF CONSULTATION.—The Secretary 

22 may determine the timing of each consultation with the 

23 external experts, which may occur prior to, or following, 

24 the filing of an investigational new drug application under 

25 section 505(i), a new drug application under section 


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1 505(b), or a biologics license application under section 351 

2 of the Public Health Service Act. 

3 ‘‘(f) CLASSIFICATION AS SPECIAL GOVERNMENT EM4 PLOYEES.—The external experts who are consulted under 

5 this section shall be considered special government em6 ployees, as defined under section 202 of title 18, United 

7 States Code.’’. 

Æ 


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