II 

112TH CONGRESS 

2D SESSION S. 2281 

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 SENATE OF THE UNITED STATES 

MARCH 29, 2012 

Mr. WHITEHOUSE introduced the following bill; which was read twice and 

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

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 Rare Treatments Act of 2012’’ or the 

6 ‘‘ExPERT Act’’. 


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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, requires the Food and Drug Ad17 ministration to develop or obtain expertise in many 

18 diseases and disease subtypes. 

19 (5) In previous circumstances when the Food 

20 and Drug Administration has consulted with rare 

21 drug experts, the agency has been able to move new 

22 therapies to market with greater efficiency. 

23 (6) The Food and Drug Administration benefits 

24 from this type of consultation with external experts 

25 who have a deep understanding of the diseases or 

26 disease subtypes that are targeted by new therapies. 


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1 (7) Access to external experts provides valuable 

2 advice about rare diseases or disease subtypes, dis3 ease severity, and unmet medical needs. 

4 SEC. 3. CONSULTATION WITH EXTERNAL EXPERTS. 

5 Subchapter E of chapter V of the Federal Food, 

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

7 amended by adding at the end the following: 

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

9 RARE DISEASES, TARGETED THERAPIES, AND 

10 GENETIC TARGETING OF TREATMENTS. 

11 ‘‘(a) IN GENERAL.— 

12 ‘‘(1) OPPORTUNITIES FOR CONSULTATION.— 

13 The Secretary shall ensure that opportunities exist, 

14 at a time the Secretary determines appropriate, for 

15 consultation with external experts on the topics de16 scribed in subsection (c), for the purpose of pro17 moting the efficiency of and informing the review by 

18 the Food and Drug Administration of drugs and bio19 logic products for rare diseases and drugs and bio20 logic products that are genetically targeted. 

21 ‘‘(2) CONSULTATION.—The Center for Drug 

22 Evaluation and Research and the Center for Bio23 logics Evaluation and Research shall, when appro24 priate, seek the opinion of external experts on any 

25 topic, including the topics described in subsection 


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1 (c), by initiating contact with such experts. External 

2 experts may also request the opportunity to meet 

3 with a review division regarding any topic described 

4 in subsection (c). 

5 ‘‘(b) EXTERNAL EXPERTS.—The external experts 

6 under subsection (a) may include— 

7 ‘‘(1) representatives of patient, consumer, re8 search, and health professional organizations with 

9 expertise relevant to the review of rare disease prod10 ucts; 

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

12 rare and other diseases, and genetic targeting of 

13 treatments, including experts from academia; and 

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

15 for small target populations. 

16 ‘‘(c) TOPICS FOR CONSULTATION.—Topics for con17 sultation may include— 

18 ‘‘(1) rare diseases; 

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

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

21 rare diseases; 

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

23 with a rare disease to participate in clinical trials; 


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

2 including side effects, of current and investigational 

3 therapies; 

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

5 populations and subpopulations, including regulatory 

6 and scientific policies affecting the design of such 

7 trials; and 

8 ‘‘(7) demographics and the clinical description 

9 of patient populations. 

10 ‘‘(d) CLASSIFICATION AS SPECIAL GOVERNMENT EM11 PLOYEES.—The external experts who are consulted under 

12 this section may be considered special government employ13 ees, as defined under section 202 of title 18, United States 

14 Code. 

15 ‘‘(e) PROPRIETARY INFORMATION.—Nothing in this 

16 section shall be construed to create a right for any exter17 nal expert, as described in subsection (b), to obtain access 

18 to proprietary information of a sponsor without the per19 mission of such sponsor. 

20 ‘‘(f) NO RIGHT OR OBLIGATION.—Nothing in this 

21 section shall be construed to create a legal right for a con22 sultation on any matter or require the Secretary to meet 

23 with any particular expert.’’. 

Æ 


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