Expanding and Promoting Expertise in Review of Rare Treatments Act of 2012 or EXPERRT Act of 2012 - Amends the Federal Food, Drug, and Cosmetic Act to direct the Secretary of Health and Human Services (HHS) to establish a program for consultation with external experts to inform and strengthen the Food and Drug Administration's (FDA's) review of drugs and biologic products for rare diseases and drugs and biologic products that are genetically targeted.
Requires, under such program, each review division within 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. Allows external experts to request the opportunity to meet with a review division regarding any such topic. Authorizes the Secretary to determine the timing of each consultation, which may occur prior to, or following, the filing of an investigational new drug application, a new drug application, or a biologies license application.
Requires the experts consulted to be considered special government employees.
[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>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
Llama 3.2 · runs locally in your browser
Ask anything about this bill. The AI reads the full text to answer.
Enter to send · Shift+Enter for new line