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[Congressional Bills 112th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4156 Introduced in House (IH)]

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.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Expanding and Promoting Expertise in 
Review of Rare Treatments Act of 2012'' or ``EXPERRT Act of 2012''.

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 potential 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 disease or disability, 
        poses challenges for the Food and Drug Administration, as the 
        agency is asked to develop or obtain expertise in many diseases 
        and disease 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 advice about 
        rare diseases or disease subtypes, disease severity, and unmet 
        medical needs.

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 topics described in 
subsection (d), for the purpose of informing 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 established 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, research, and 
        health professional organizations;
            ``(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 consultation 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 
505(b), or a biologics license application under section 351 of the 
Public Health Service Act.
    ``(f) Classification as Special Government Employees.--The external 
experts who are consulted under this section shall be considered 
special government employees, as defined under section 202 of title 18, 
United States Code.''.
                                 <all>
```