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 Administra-
tion to seek advice from external experts regarding rare 
diseases, the burden of rare diseases, and the unmet 
medical needs of individuals with rare diseases. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Expanding and Pro-
moting Expertise in Review of Rare Treatments Act of 
2012’’ or ‘‘EXPERRT Act of 2012’’. 
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SEC. 2. FINDINGS. 
Congress finds as follows: 
(1) Biomedical research is yielding discoveries 
that are leading to the development of new therapies 
that hold great promise for treating disease. 
(2) Scientists are increasingly unlocking the po-
tential for targeting treatments according to genetic 
defect. 
(3) Many of the new therapies that are under 
development in laboratories across the Nation are 
targeted to rare diseases, small subsets of diseases 
of significant incidence, or even small subsets of rare 
diseases. 
(4) Progress in the development of targeted 
therapies, while of great promise for those with dis-
ease or disability, poses challenges for the Food and 
Drug Administration, as the agency is asked to de-
velop or obtain expertise in many diseases and dis-
ease subtypes. 
(5) The Food and Drug Administration could 
benefit from consultation with external experts who 
have a deep understanding of the diseases or disease 
subtypes that are targeted by new therapies. 
(6) External experts could provide valuable ad-
vice about rare diseases or disease subtypes, disease 
severity, and unmet medical needs. 
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SEC. 3. CONSULTATION WITH EXTERNAL EXPERTS. 
Subchapter E of chapter V of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb et seq.) is 
amended by adding at the end the following: 
‘‘SEC. 568. CONSULTATION WITH EXTERNAL EXPERTS ON 
RARE DISEASES, TARGETED THERAPIES, AND 
GENETIC TARGETING OF TREATMENTS. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
program for consultation with external experts on the top-
ics described in subsection (d), for the purpose of inform-
ing and strengthening the Food and Drug Agency’s review 
of drugs and biologic products for rare diseases and drugs 
and biologic products that are genetically targeted. 
‘‘(b) PROGRAM OPERATION.—Under the program es-
tablished under subsection (a), each review division within 
the Center for Drug Evaluation and Research and the 
Center for Biologics Evaluation and Research shall, when 
appropriate, seek the opinion of external experts on any 
topic described in subsection (d) by initiating contact with 
such experts. The external experts may also request the 
opportunity to meet with a review division regarding any 
topic described in subsection (d). 
‘‘(c) EXTERNAL EXPERTS.—The external experts 
under subsection (a) may include— 
‘‘(1) representatives of patient, consumer, re-
search, and health professional organizations; 
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‘‘(2) experts on rare diseases, rare subtypes of 
rare and other diseases, and genetic targeting of 
treatments, including experts from academia; and 
‘‘(3) experts in innovative clinical trial designs 
for small target populations. 
‘‘(d) TOPICS FOR CONSULTATION.—Topics for con-
sultation may include— 
‘‘(1) rare diseases; 
‘‘(2) the severity of rare diseases; 
‘‘(3) the unmet medical need associated with 
rare diseases; 
‘‘(4) the willingness and ability of individuals 
with a rare disease to participate in clinical trials; 
‘‘(5) an assessment of the benefits and risks, 
including side effects, of current and investigational 
therapies; 
‘‘(6) the design of clinical trials for rare disease 
populations and subpopulations; and 
‘‘(7) demographics and the clinical description 
of patient populations. 
‘‘(e) TIMING
OF CONSULTATION.—The Secretary 
may determine the timing of each consultation with the 
external experts, which may occur prior to, or following, 
the filing of an investigational new drug application under 
section 505(i), a new drug application under section 
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505(b), or a biologics license application under section 351 
of the Public Health Service Act. 
‘‘(f) CLASSIFICATION AS SPECIAL GOVERNMENT EM-
PLOYEES.—The external experts who are consulted under 
this section shall be considered special government em-
ployees, as defined under section 202 of title 18, United 
States Code.’’. 
Æ 
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