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.

SECTION 1. SHORT TITLE.

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

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, 
        requires the Food and Drug Administration to develop or obtain 
        expertise in many diseases and disease subtypes.
            (5) In previous circumstances when the Food and Drug 
        Administration has consulted with rare drug experts, the agency 
        has been able to move new therapies to market with greater 
        efficiency.
            (6) The Food and Drug Administration benefits from this 
        type of consultation with external experts who have a deep 
        understanding of the diseases or disease subtypes that are 
        targeted by new therapies.
            (7) Access to external experts provides 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.--
            ``(1) Opportunities for consultation.--The Secretary shall 
        ensure that opportunities exist, at a time the Secretary 
        determines appropriate, for consultation with external experts 
        on the topics described in subsection (c), for the purpose of 
        promoting the efficiency of and informing the review by the 
        Food and Drug Administration of drugs and biologic products for 
        rare diseases and drugs and biologic products that are 
        genetically targeted.
            ``(2) Consultation.--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, including the topics described in subsection (c), 
        by initiating contact with such experts. External experts may 
        also request the opportunity to meet with a review division 
        regarding any topic described in subsection (c).
    ``(b) External Experts.--The external experts under subsection (a) 
may include--
            ``(1) representatives of patient, consumer, research, and 
        health professional organizations with expertise relevant to 
        the review of rare disease products;
            ``(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.
    ``(c) 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, including regulatory and 
        scientific policies affecting the design of such trials; and
            ``(7) demographics and the clinical description of patient 
        populations.
    ``(d) Classification as Special Government Employees.--The external 
experts who are consulted under this section may be considered special 
government employees, as defined under section 202 of title 18, United 
States Code.
    ``(e) Proprietary Information.--Nothing in this section shall be 
construed to create a right for any external expert, as described in 
subsection (b), to obtain access to proprietary information of a 
sponsor without the permission of such sponsor.
    ``(f) No Right or Obligation.--Nothing in this section shall be 
construed to create a legal right for a consultation on any matter or 
require the Secretary to meet with any particular expert.''.

Summary: Expanding and Promoting Expertise in Rare Treatments Act of 2012 or EXPERT Act of 2012 - Amends the Federal Food, Drug, and Cosmetic Act to direct the Secretary of Health and Human Services (HHS) to ensure that opportunities exist for consultation with external experts to promote the efficiency of and inform the review by the Food and Drug Administration (FDA) of drugs and biologic products for rare diseases and drugs and biologic products that are genetically targeted.

Requires the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research to seek the opinion of external experts on topics that 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. Authorizes: (1) external experts to request the opportunity to meet with a review division regarding any such topic, and (2) external experts who are consulted to be considered special government employees.

Declares that nothing in this Act shall be construed to: (1) create a right for any external expert to obtain access to proprietary information of a sponsor without that sponsor's permission or a legal right for a consultation on any matter, or (2) require the Secretary to meet with any particular expert.