Compassionate Access Act of 2010 - Amends the Federal Food, Drug, and Cosmetic Act to require the Secretary of Health and Human Services (HHS) to permit an investigational drug, biological product, or device to be made available for expanded access under a treatment investigational new drug application or treatment investigational device exemption if specified Compassionate Investigational Access requirements are met.
Gives immunity to the manufacturer, distributor, administrator, sponsor, or physician from suit or liability relating to products approved under this Act.
Establishes a procedure for accelerated approval of an investigational drug, biological product, or device that is reasonably likely to predict clinical benefit to a patient suffering from a serious or life-threatening condition.
Requires the Secretary to establish: (1) the Accelerated Approval Advisory Committee; (2) a new program to expand access to investigational treatments for individuals with serious or life threatening conditions and diseases; and (3) a demonstration project under the Medicare program to pay for drugs, biological, products, and devices approved under this Act.
Requires the Secretary to consider the clinical judgment and risks to the patient from the disease or condition in evaluating the safety and effectiveness of drugs, biological products, and devices that treat serious or life-threatening diseases or conditions, including the evaluation of nonstatistical information.
Requires any committee evaluating investigational drugs, devices, or biological product applications to have at least two patient representatives as voting members.
[Congressional Bills 111th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4732 Introduced in House (IH)]
111th CONGRESS
2d Session
H. R. 4732
To amend the Federal Food, Drug, and Cosmetic Act to create a new
conditional approval system for drugs, biological products, and devices
that is responsive to the needs of seriously ill patients, and for
other purposes.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
March 2, 2010
Ms. Watson introduced the following bill; which was referred to the
Committee on Energy and Commerce, and in addition to the Committee on
Ways and Means, for a period to be subsequently determined by the
Speaker, in each case for consideration of such provisions as fall
within the jurisdiction of the committee concerned
_______________________________________________________________________
A BILL
To amend the Federal Food, Drug, and Cosmetic Act to create a new
conditional approval system for drugs, biological products, and devices
that is responsive to the needs of seriously ill patients, and for
other purposes.
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 ``Compassionate Access Act of 2010''.
SEC. 2. FINDINGS.
Congress finds the following:
(1) As of 2009, the standards of the Food and Drug
Administration for approval of drugs, biological products, and
devices may deny the benefits of medical progress to seriously
ill patients who face morbidity or death from their disease.
(2) Seriously ill patients have a right to take actions to
preserve their life by accessing available investigational
drugs, biological products, and devices.
(3) The emphasis on statistical analysis of clinical
information needs to be balanced by a reliance on clinical
evaluation and patient-reported outcomes and considered with an
understanding of the risks to patients from their disease, with
the goal of providing additional treatment options for patients
and their physicians to consider.
(4) Food and Drug Administration advisory committees should
have greater representation of medical clinicians and patient
advocates who represent the interests of seriously ill patients
in early access to promising investigational therapies.
(5) The use of available investigational products for
treatment is the responsibility of the physician and the
seriously ill patient.
(6) The use of combinations of available investigational
and approved products for treatment is the responsibility of
the physician and the seriously ill patient.
(7) The Food and Drug Administration should have the
expertise and flexibility to address the growing needs of
seriously ill patients for individualized or personalized
therapies.
SEC. 3. COMPASSIONATE INVESTIGATIONAL ACCESS APPROVAL SYSTEM FOR DRUGS,
BIOLOGICAL PRODUCTS, AND DEVICES.
(a) Compassionate Investigational Access.--Section 561 of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) is amended--
(1) by redesignating subsections (d) and (e) as subsections
(e) and (f), respectively; and
(2) by inserting after subsection (c) the following:
``(d) Compassionate Investigational Access.--
``(1) Purpose.--The purpose of this subsection is to
facilitate the availability of promising new drugs to seriously
ill patients as early in the drug development process as
possible, before general marketing begins.
``(2) Access.--Notwithstanding any other provision of law,
upon submission by a sponsor of an application intended to
provide widespread access to an investigational drug,
biological product, or device for eligible patients (referred
to in this subsection as `Compassionate Investigational
Access'), the Secretary shall permit such investigational drug,
biological product, or device, to be made available for
expanded access under a treatment investigational new drug
application or treatment investigational device exemption if
the Secretary determines that the requirements of this section
are met with respect to Compassionate Investigational Access.
``(3) Compassionate investigational access.--
Notwithstanding any other provision of law, an investigational
drug, biological product, or device that receives approval for
Compassionate Investigational Access under this subsection
shall be subject to the provisions of section 505(i) or 520(g),
as applicable, and regulations promulgated by the Secretary
pursuant to this Act. The Secretary and the sponsor may inform
national, State, and local medical associations and societies,
voluntary health associations, and other appropriate persons
about the availability of an investigational drug or
investigational device under Compassionate Investigational
Access as approved under this subsection. The information
submitted by the Secretary, in accordance with the preceding
sentence, shall be the same type of information that is
required by section 402(i)(3) of the Public Health Service Act.
``(4) Submission of application.--
``(A) Application content.--A sponsor of an
investigational drug, biological product, or device
applying for Compassionate Investigational Access
approval of the product shall submit to the Secretary a
notice of claimed exemption under section 505(i) or
520(g), as applicable, (referred to in this subsection
as an `application for Compassionate Investigational
Access'), which shall contain--
``(i) data and information from completed
Phase I clinical investigations and any other
nonclinical or clinical investigations;
``(ii) preliminary evidence that the
product may be effective in humans against a
serious or life-threatening condition or
disease, which evidence may be based on
uncontrolled data such as case histories,
information about the pharmacological mechanism
of action, data from animal and computer
models, comparison with historical data, or
other preliminary information, and may be based
on a small number of patients or a subset of
the patient population;
``(iii) evidence that the product is safe
at the dose and duration proposed, considering
whether the potential risk to a patient of the
condition or disease outweighs the potential
risk to a patient of the proposed dose and
duration of treatment with the product,
consistent with the level of information needed
to initiate a Phase II clinical trial; and
``(iv) a statement that the sponsor is
actively pursuing marketing approval with due
diligence.
``(B) Limitation.--Compassionate Investigational
Access approval shall be based upon multiple
considerations that shall include clinical evaluation
and unmet patient needs.
``(5) Determination by secretary.--
``(A) In general.--Not later than 30 days after the
receipt of an application for Compassionate
Investigational Access approval, the Secretary shall
either--
``(i) provide Compassionate Investigational
Access approval of the application; or
``(ii) refer the application to the
Accelerated Approval Advisory Committee.
``(B) Recommendation.--Not later than 90 days after
receipt of an application for Compassionate
Investigational Access approval, the Accelerated
Approval Advisory Committee shall issue a
recommendation to the Secretary on whether the
Secretary shall provide Compassionate Investigational
Access approval of the application.
``(C) Final decision.--Not later than 30 days after
receipt of the recommendation from the Accelerated
Approval Advisory Committee, the Secretary shall either
provide Compassionate Investigational Access approval
of the application or shall issue an order setting
forth a detailed explanation of the reasons why the
application was not so approved and the specific data
that the sponsor must provide so that the application
may be so approved.
``(6) Appeal.--If the Secretary does not provide
Compassionate Investigational Access approval of an
application, the sponsor of the application shall have the
right to appeal the decision to the Secretary. The Secretary
shall provide the sponsor with a hearing not later than 30 days
following the nonapproval under this subsection of the
application and shall issue an order not later than 30 days
following the hearing either concurring in the nonapproval or
so approving the application. The Secretary shall not delegate
the responsibility described in this paragraph to any other
person.
``(7) Criteria.--In making a determination under paragraph
(5), the Secretary shall consider whether the totality of the
information available to the Secretary regarding the safety and
effectiveness of an investigational drug, biological product,
or device, as compared to the risk of morbidity or death from a
condition or disease, indicates that a patient (who may be
representative of a small patient subpopulation) may obtain
more benefit than risk if treated with the drug, biological
product, or device. If the potential risk to a patient of the
condition or disease outweighs the potential risk of the
product, and the product may possibly provide benefit to the
patient, the Secretary shall provide Compassionate
Investigational Access approval of the application.
``(8) Patient eligibility for compassionate access.--In
order for a patient to access a product available through
Compassionate Investigational Access, the physician must
document in writing that the patient--
``(A) is seriously ill;
``(B) has examined all treatment options approved
by the Secretary for the condition or disease for which
the patient is a reasonable candidate; and
``(C) has unsuccessfully sought treatment or
obtained treatment that was not effective, with an
investigational drug, biological product, or device for
which such individual is a reasonable candidate, which
shall include consideration of a patient's
ineligibility for participation in clinical trials, the
lack of source of supply and geographic factors.
``(9) Product labeling.--To receive Compassionate
Investigational Access approval under this subsection, the
sponsor of the product shall provide labeling approved by the
Secretary for the drug, biological product, or device that--
``(A) states that the product is intended for use
by a patient whose physician has documented in writing
that the patient has--
``(i) examined all treatment options
approved by Secretary for the condition or
disease for which the patient is a reasonable
candidate; and
``(ii) unsuccessfully sought treatment, or
obtained treatment that was not effective with
an investigational drug, biological product, or
device for which such individual is a
reasonable candidate, which shall include a
patient's ineligibility for participation in
clinical trials, the lack of source of supply
and geographic factors; and
``(B) states that every patient to whom the product
is administered shall, as a mandatory condition of
receiving the product, provide--
``(i) written informed consent, as
described under part 50 of title 21, Code of
Federal Regulations (or any successor
regulations); and
``(ii) consent for the manufacturer of the
product to obtain data and information about
the patient and the patient's use of the
product that may be used to support an
application for Accelerated Approval or final
approval.
``(10) Charging for compassionate investigational access.--
A sponsor or investigator may charge for a Compassionate
Investigational Access drug without notifying the Secretary or
seeking or obtaining prior approval of the amount charged,
provided the sponsor of the drug is actively pursuing marketing
approval with due diligence.
``(11) Commencement of review.--If the Secretary
determines, after preliminary evaluation of the data and
information submitted by the sponsor, that the product may be
effective, the Secretary shall evaluate for filing, and may
commence review of portions of, an application under this
subsection before the sponsor submits a complete application.
The Secretary shall commence such review only if the applicant
provides a schedule for submission of information necessary to
make the application complete.
``(12) Immunity.--
``(A) In general.--A manufacturer, distributor,
administrator, sponsor, or physician who manufactures,
supplies, distributes or prescribes a product approved
under an application for Compassionate Investigational
Access shall be immune from suit or liability caused
by, arising out of, or relating to the design,
development, clinical testing and investigation,
manufacture, labeling, distribution, sale, purchase,
donation, dispensing, prescribing, administration,
efficacy, or use of a drug, biological product, or
device subject to an approved Compassionate
Investigational Access application.
``(B) Claims.--No claim or cause of action against
a manufacturer, distributor, administrator, sponsor, or
physician who manufactures, supplies, distributes or
prescribes a product subject to an approved
Compassionate Investigational Access application shall
exist in any Federal or State court for claims of
property, personal injury, or death caused by, arising
out of, or relating to the design, development,
clinical testing and investigation, manufacture,
labeling, distribution, sale, purchase, donation,
dispensing, prescribing, administration, efficacy, or
use of a drug, biological product, or device subject to
an approved Compassionate Investigational Access
application. Any such claim or cause of action that is
filed in Federal or State court shall be immediately
dismissed.
``(13) Final approval.--For purposes of this Act, the term
`final approval' means--
``(A) with respect to a new drug or new biological
product, approval of such drug or product under section
505(b)(1) or 505(b)(2) or section 351 of the Public
Health Service Act, as the case may be; and
``(B) with respect to a new device, clearance of
such device under section 510(k) or approval of such
device under section 515(c)(1).''.
(b) Accelerated Approval.--Chapter V of the Federal, Food, Drug,
and Cosmetic Act (21 U.S.C. 351 et seq.) is amended by inserting after
section 561 the following:
``SEC. 561A. ACCELERATED APPROVAL.
``(a) In General.--
``(1) In general.--As soon as practicable after the date of
enactment of the Compassionate Access Act of 2010, the
Secretary shall promulgate regulations to provide for the
treatment of an investigational drug, biological product, or
device that receives Accelerated Approval under this section.
This section shall be carried out in accordance with such
regulations (and any successor regulations).
``(2) Application.--A sponsor of an investigational drug,
biological product, or device applying for Accelerated Approval
shall submit to the Secretary an application as described under
section 505(b)(1) or 505(b)(2), or section 510(k) or 515(c)(1)
of this Act, or section 351(a) of the Public Health Service
Act, as applicable, which shall contain--
``(A) data and information that the drug,
biological product, or device has an effect on a
clinical endpoint or on a surrogate endpoint or
biomarker that is reasonably likely to predict clinical
benefit to a patient (who may be representative of a
small patient subpopulation) suffering from a serious
or life-threatening condition or disease; and
``(B) a statement that the sponsor is actively
pursuing marketing approval with due diligence.
``(3) Determination by secretary.--
``(A) In general.--Not later than 120 days after
the receipt of an application for Accelerated Approval,
the Secretary shall either--
``(i) provide Accelerated Approval of the
application; or
``(ii) refer the application to the
Accelerated Approval Advisory Committee.
``(B) Recommendation.--Not later than 90 days after
receipt of an application for Accelerated Approval, the
Accelerated Approval Advisory Committee shall issue a
recommendation to the Secretary on whether the
Secretary should provide Accelerated Approval of the
application.
``(C) Limitation.--The Accelerated Approval
Advisory Committee shall not consider off-label uses of
drugs, biological products, and devices as existing or
available therapies, to the extent that the Accelerated
Approval Advisory Committee weighs existing or
available therapies in determination of whether an
investigational drug provides an improvement over
treatments that are already available.
``(D) Final decision.--Not later than 30 days after
receipt of the recommendation from the Accelerated
Approval Advisory Committee, the Secretary shall either
provide Accelerated Approval of the application or
issue an order setting forth a detailed explanation of
the reasons why the application was not so approved and
the specific data that the sponsor must provide so that
the application may be so approved.
``(4) Appeal.--If the Secretary does not provide
Accelerated Approval of an application, the sponsor of the
application shall have the right to appeal the decision to the
Secretary. The Secretary shall provide the sponsor with a
hearing not later than 30 days following the nonapproval under
this subsection of the application and shall issue an order not
later than 30 days following the hearing either concurring in
such nonapproval or so approving the application. The Secretary
shall not delegate the responsibility described in this
paragraph to any other person.
``(A) with respect to a new drug or new biological
product, approval of such drug or product under section
505(b)(1) or 505(b)(2) or section 351 of the Public
Health Service Act, as the case may be; and
``(B) with respect to a new device, clearance of
such device under section 510(k) or approval of such
device under section 515(c)(1).
``(b) Accelerated Approval Advisory Committee.--
``(1) In general.--In order to facilitate the development
and expedite the review of drugs, biological products, and
devices intended to treat serious or life threatening
conditions, the Secretary shall establish the Accelerated
Approval Advisory Committee (referred to in this subsection as
the `Committee').
``(2) Delegation.--The Secretary may delegate
decisionmaking authority for the Accelerated Approval Advisory
Committee to the Office of the Commissioner of Food and Drugs.
Such authority shall not be further delegated.
``(3) Composition.--
``(A) In general.--The Committee shall be composed
of 11 voting members, including 1 chairperson and 5
permanent members each of whom shall serve a term of 3
years and may be reappointed for a second 3-year term,
and 5 nonpermanent members who shall be appointed to
the Committee for a specific meeting, or part of a
meeting, in order to provide adequate expertise in the
subject being reviewed. The Committee shall include as
voting members no less than 2 representatives of
patient interests, of which 1 shall be a permanent
member of the Committee. The Committee shall include as
nonvoting members a representative of interests of the
drug, biological product, and device industry.
``(B) Appointments.--The Secretary shall appoint to
the Committee persons who are qualified by training and
experience to evaluate the safety and effectiveness of
the types of products to be referred to the Committee
and who, to the extent feasible, possess skill in the
use of, or experience in the development, manufacture,
or utilization of, such products. The Secretary shall
make appointments to the Committee so that the
Committee shall consist of members with adequately
diversified expertise and practical experience in such
fields as clinical medicine, biological and physical
sciences, and other related professions. Scientific,
industry, and consumer organizations and members of the
public shall be afforded an opportunity to nominate
individuals for appointment to the Committee. No
individual who is in the regular full-time employ of
the United States and engaged in the administration of
this chapter may be a member of the Committee.
``(4) Compensation.--Committee members, while attending
meetings or conferences of the Committee or otherwise engaged
in its business, shall be entitled to receive compensation at
rates to be fixed by the Secretary, but not at rates exceeding
the daily equivalent of the rate in effect for grade GS-18 of
the General Schedule, for each day so engaged, including
traveltime, and while so serving away from their homes or
regular places of business each member may be allowed travel
expenses (including per diem in lieu of subsistence) as
authorized by section 5703 of title 5, for persons in the
Government service employed intermittently.
``(5) Assistance.--The Secretary shall furnish the
Committee with adequate clerical and other necessary
assistance.
``(6) Annual training.--The Secretary shall employ
nongovernmental experts to provide annual training to the
Committee on the statutory and regulatory standards for product
approval.
``(7) Timeline.--The Committee shall be scheduled to meet
at such times as may be appropriate for the Secretary to meet
applicable statutory deadlines.
``(8) Meetings.--
``(A) Opportunities for interested persons.--Any
person whose product is specifically the subject of
review by the Committee shall have--
``(i) the same access to data and
information submitted to the Committee as the
Secretary;
``(ii) the opportunity to submit, for
review by the Committee, data or information,
which shall be submitted to the Secretary for
prompt transmittal to the Committee;
``(iii) the same opportunity as the
Secretary to participate in meetings of the
Committee; and
``(iv) consent for the manufacturer of the
product to obtain data about adverse events
relating to the patient's use of the product.
``(B) Adequate time; free and open participation.--
Any meetings of the Committee shall provide adequate
time for initial presentations and for response to any
differing views by persons whose products are
specifically the subject of the Committee review.
``(C) Summaries.--At all meetings of the Committee,
the Secretary shall provide a summary to the Committee
of all applications submitted under this subsection and
section 561(d) that the Committee did not consider that
were approved by the Secretary since the last meeting
of the Committee.
``(c) Final Approval.--For purposes of this Act, the term `final
approval' means--
``(1) with respect to a new drug or new biological product,
approval of such drug or product under section 505(b)(1) or
505(b)(2) or section 351 of the Public Health Service Act, as
the case may be; and
``(2) with respect to a new device, clearance of such
device under section 510(k) or approval of such device under
section 515(c)(1).''.
(c) Regulations.--The Secretary of Health and Human Services shall
promulgate regulations that define the terms ``seriously ill'' and
``serious or life-threatening'' for purposes of the amendments made by
this Act, considering either--
(1) the medical prognosis for an individual's life
expectancy from a disease or condition; or
(2) the prospect of irreversible disability from a disease
or condition.
(d) Conforming Amendment.--Subsection (a)(2) of section 351 of the
Public Health Service Act is amended adding at the end the following
subparagraphs:
``(E) Compassionate investigational access.--A
person that submits an application for a license under
this paragraph and seeks approval of the license for
the purpose of Compassionate Investigational Access
under section 561 of the Federal Food, Drug ,and
Cosmetic Act shall submit to the Secretary as part of
the application any information required under such
section.
``(F) Accelerated approval.--A person that submits
an application for a license under this paragraph and
seeks accelerated approval of the license under section
561A of the Federal Food, Drug, and Cosmetic Act shall
submit to the Secretary as part of the application any
information required under such section.''.
SEC. 4. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS AND DEVICES.
Chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
351 et seq.) is amended by inserting after section 561A, as added by
section 3, the following:
``SEC. 561B. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS AND DEVICES.
``(a) Expanded Access Program.--The Secretary shall establish a new
program to expand access to investigational treatments for individuals
with serious or life threatening conditions and diseases. In carrying
out this expanded access program, the Secretary shall publish and
broadly disseminate written guidance that--
``(1) describes such expanded access programs for
investigational drugs, biological products, and devices
intended to treat serious or life-threatening conditions or
diseases;
``(2) encourages and facilitates submission of applications
and approvals under sections 561(d) and 561A; and
``(3) facilitates the provision of investigational drugs,
biological products, and devices to seriously ill individuals
without unreasonable delay by recognizing that the use of
available investigational products for treatment is the
responsibility of the physician and the patient, and also by
recognizing the goal of providing additional treatment options
for patients and their physicians to consider.
``(b) Implementation of Expanded Access Programs.--
``(1) Training of personnel.--Not later than 90 days after
the date of enactment of this section, the Secretary shall
implement training programs at the Food and Drug Administration
with respect to the expanded access programs established under
this section.
``(2) Policies, regulations, and guidance.--The Secretary
shall establish policies, regulations, and guidance designed to
most directly benefit seriously ill patients.
``(c) Development of Surrogate Endpoints and Biomarkers.--
``(1) In general.--The Secretary shall--
``(A) establish a program or expand upon an
existing program to encourage the development of
surrogate endpoints and biomarkers that are reasonably
likely to predict clinical benefit for serious or life-
threatening conditions for which there exist
significant unmet patient needs;
``(B) request the Institute of Medicine to
undertake a study to identify validated surrogate
endpoints and biomarkers, and recommend research to
validate surrogate endpoints and biomarkers, that may
support approvals for products intended for the
treatment of serious or life-threatening conditions or
diseases; and
``(C) make widely available to the public a list of
drugs, biological products, and devices that are being
investigated for serious or life-threatening conditions
or diseases and that have not yet received approval
under section section 561(d) or 561A for marketing.
``(2) Study content.--The study under paragraph (1)(B)
shall include endpoints and biomarkers that address the unmet
medical needs of subpopulations of patients and that facilitate
the development of individualized treatment approaches for
patients with serious or life-threatening conditions or
diseases.''.
SEC. 5. DEMONSTRATION PROJECT FOR COVERAGE OF CERTAIN DRUGS, BIOLOGICAL
PRODUCTS, AND DEVICES UNDER THE MEDICARE PROGRAM.
(a) In General.--The Secretary of Health and Human Services (in
this section referred to as the ``Secretary'') shall establish a
demonstration project under the Medicare program under title XVIII of
the Social Security Act (42 U.S.C. 1395 et seq.) under which payment is
made for drugs, biological products, and devices approved for
Compassionate Investigational Access under section section 561(d) of
the Federal Food, Drug, and Cosmetic Act in the case where the drug,
biological product, or device is not otherwise covered under the
Medicare program or by any other organization or entity (including a
public assistance program or the sponsor of the application for such
drug, biological product, or device).
(b) Duration.--The demonstration project under this section shall
be conducted for a 5-year period.
(c) Funding.--
(1) In general.--The Secretary shall provide for the
transfer from the Federal Hospital Insurance Trust Fund under
section 1817 of the Social Security Act (42 U.S.C. 1395i) and
the Federal Supplementary Medical Insurance Trust Fund under
section 1841 of such Act (42 U.S.C. 1395t), in such proportion
as the Secretary determines to be appropriate, of such sums as
are necessary for the costs of carrying out the demonstration
project under this section.
(2) Budget neutrality.--In conducting the demonstration
project under this section, the Secretary shall ensure that the
aggregate payments made by the Secretary do not exceed the
amount which the Secretary estimates would have been paid if
the demonstration project under this section was not
implemented.
(d) Waiver Authority.--The Secretary may waive such requirements of
title XVIII of the Social Security Act (42 U.S.C. 1395 et seq.) as may
be necessary for the purpose of carrying out the demonstration project
under this section.
(e) Report.--Not later than 90 days after the last day of the 5-
year period of the demonstration project under this section, the
Secretary shall submit to Congress a report describing the rates of
utilization by Medicare beneficiaries of drugs, biological products,
and devices approved for Compassionate Investigational Access and the
total cost of payments made under the Medicare program resulting from
the demonstration project. The report shall describe recommendations
for legislation or administrative action as the Secretary deems
appropriate.
(f) Termination.--The Secretary shall terminate payments under this
section on the day after the last day of the 5-year period of the
demonstration project under this section.
SEC. 6. MODERNIZATION OF THE FOOD AND DRUG ADMINISTRATION.
(a) In General.--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. POLICIES RELATED TO STUDY EVALUATION INFORMATION.
``(a) In General.--
``(1) Nonstatistical measures.--The Secretary shall give
consideration to clinical judgment and risks to the patient
from the disease or condition involved in the evaluation of the
safety and effectiveness of drugs, biological products, and
devices that treat serious or life-threatening diseases or
conditions. This policy shall apply--
``(A) in evaluating clinical study designs and
endpoints; and
``(B) in making decisions with respect to product
applications for approval under section section 561(d)
or 561A.
``(2) Types of nonstatistical measures.--The policy
established under paragraph (1), for the purposes described in
such paragraph--
``(A) shall include such nonstatistical information
as--
``(i) clinical evaluation information, such
as case history reports;
``(ii) scientific and clinical studies
designed to measure or define mechanisms of
action or molecular targeting;
``(iii) data from animal and computer
models; and
``(iv) comparison with historical data; and
``(B) shall incorporate the use of--
``(i) evaluations of the adverse effect of
delaying the availability of an investigational
drug to even a small subpopulation of seriously
ill patients; and
``(ii) scientific, observational, or
clinical studies designed and conducted to
collect well-documented information.
``(b) Meetings.--A meeting to address any pending scientific,
medical, regulatory, or other issue relating to the development,
investigation, review, or other aspect of a drug, biological product,
or device shall ordinarily be held not later than 15 days of the
receipt of a written request for the meeting by the sponsor of the
product, which may be extended to 30 days for good cause. Such meetings
shall ordinarily be conducted in person, but may be conducted by
telephone or other form of communication if both parties agree. In
order to reduce the burden of meetings, only those Food and Drug
Administration employees who are intended to actively participate in
the discussion shall attend a meeting. Minutes of a meeting shall be
promptly prepared and exchanged by both parties immediately following
the meeting and shall accurately summarize what occurred at the
meeting.
``(c) Rule of Construction.--The provisions of this chapter and
section 351 of the Public Health Service Act shall be construed to
incorporate the policy established in this section.''.
(b) Conforming Amendment.--Subsection (j) of section 351 of the
Public Health Service Act is amended by--
(1) striking ``and'' before ``505-1''; and
(2) inserting ``, and 568'' after ``505-1''.
SEC. 7. MEMBERSHIP OF ONCOLOGY DRUGS ADVISORY COMMITTEE AND THE
CELLULAR, TISSUE, AND GENE THERAPY ADVISORY COMMITTEE.
Notwithstanding any other provision of law, membership of the
Oncology Drugs Advisory Committee, the Cellular, Tissue, and Gene
Therapy Advisory Committee of the Food and Drug Administration, and any
other committee created by such Administration to evaluate or advise
with respect to applications submitted under section section 561(d) or
561A of the Federal Food, Drug, and Cosmetic Act (as added by this
Act), shall consist of no less than 2 patient representatives who are
voting members of the committee.
<all>
Introduced in House
Introduced in House
Referred to House Energy and Commerce
Referred to the Committee on Energy and Commerce, and in addition to the Committee on Ways and Means, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.
Referred to House Ways and Means
Referred to the Subcommittee on Health.
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