‘‘(f) EXPEDITED WITHDRAWAL OF APPROVAL.—The 
Secretary may withdraw Tier I or Tier II approval using 
expedited procedures (as prescribed by the Secretary in 
regulations which shall include an opportunity for a hear-
ing) if— 
‘‘(1) the sponsor fails to conduct post-approval 
studies with due diligence, considering all of the cir-
cumstances involved; 
‘‘(2) a post-approval study fails to verify clinical 
benefit of the product for even a small patient sub-
population; 
‘‘(3) other evidence demonstrates that the prod-
uct is not safe or effective under the conditions of 
use for even a small patient subpopulation; or 
‘‘(4) the sponsor disseminates false or mis-
leading promotional materials with respect to the 
product and fails to correct the material promptly 
after written notice from the Secretary. 
‘‘(g) ACCELERATED
APPROVAL
ADVISORY
COM-
MITTEE.— 
‘‘(1) IN GENERAL.—In order to facilitate the 
development and expedite the review of drugs, bio-
logical products, and devices intended to treat seri-
ous or life threatening conditions, the Secretary shall 
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establish the Accelerated Approval Advisory Com-
mittee. 
‘‘(2) DELEGATION.—The Secretary may dele-
gate authority for the Accelerated Approval Advisory 
Committee to the Commissioner of Food and Drugs. 
The Accelerated Approval Advisory Committee shall 
be staffed and administered in the Office of the 
Commissioner. 
‘‘(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 ex-
pertise in the subject being reviewed. The Com-
mittee 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 in-
dustry. 
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‘‘(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, manu-
facture, or utilization of, such products. The 
Secretary shall make appointments to the Com-
mittee so that the Committee shall consist of 
members with adequately diversified expertise 
and practical experience in such fields as clin-
ical medicine, biological and physical sciences, 
and other related professions. Scientific, indus-
try, 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 en-
gaged in the administration of this chapter may 
be a member of the Committee. 
‘‘(4) 
COMPENSATION.—Committee 
members, 
while attending meetings or conferences of the Com-
mittee or otherwise engaged in its business, shall be 
entitled to receive compensation at rates to be fixed 
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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 en-
gaged, including traveltime, and while so serving 
away from their homes or regular places of business 
each member may be allowed travel expenses (in-
cluding per diem in lieu of subsistence) as author-
ized 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 nec-
essary assistance. 
‘‘(6) ANNUAL TRAINING.—The Secretary shall 
employ nongovernmental experts to provide annual 
training to the Committee on the statutory and reg-
ulatory standards for product approval. 
‘‘(7) TIMELINE.—The Committee shall be 
scheduled to meet at such times as may be appro-
priate for the Secretary to meet applicable statutory 
deadlines. 
‘‘(8) MEETINGS.— 
‘‘(A) OPPORTUNITIES
FOR
INTERESTED 
PERSONS.—Any person whose product is spe-
cifically the subject of review by the Committee 
shall have— 
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‘‘(i) the same access to data and in-
formation submitted to the Committee as 
the Secretary; 
‘‘(ii) the opportunity to submit, for re-
view by the Committee, data or informa-
tion, which shall be submitted to the Sec-
retary for prompt transmittal to the Com-
mittee; and 
‘‘(iii) the same opportunity as the 
Secretary to participate in meetings of the 
Committee. 
‘‘(B) ADEQUATE TIME; FREE AND OPEN 
PARTICIPATION.—Any meetings of the Com-
mittee 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, and shall 
encourage free and open participation by all in-
terested persons. 
‘‘(C) SUMMARIES.—At all meetings of the 
Committee, the Secretary shall provide a sum-
mary to the Committee of all Tier I and Tier 
II applications that the Committee did not con-
sider that were approved by the Secretary since 
the last meeting of the Committee. 
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‘‘(h) 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 
for Tier I or Tier II approval 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 applica-
tion complete. 
‘‘(i) INAPPLICABILITY
OF PROVISIONS.—The fol-
lowing provisions shall not apply to Tier I or Tier II appli-
cations and approvals: 
‘‘(1) Chapter VII, subchapter C, parts 2 and 3 
relating to fees for drugs, biological products, and 
devices. 
‘‘(2) The provisions of the Drug Price Competi-
tion and Patent Term Restoration Act of 1984 that 
authorize approval of abbreviated new drug applica-
tions and applications submitted under section 
505(b)(2). Market exclusivity and patent term res-
toration of Tier I and Tier II approved drugs, bio-
logical products, and devices shall be determined 
solely at the time of Tier III approval without re-
gard to prior Tier I or Tier II approval. Prior to 
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Tier III approval, the Secretary shall not approve 
any application submitted under section 505(b)(2) 
or section 505(j) that references a drug approved 
under subsections (b) or (c) of this section.’’. 
SEC. 4. ETHICS IN HUMAN TESTING. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 351 et seq.) is amended by adding at the 
end of section 505(i) the following: 
‘‘(5) Notwithstanding any other provision of 
law, the Secretary shall prohibit placebo-only or no- 
treatment-only concurrent controls in any clinical in-
vestigation conducted under this chapter or, in the 
use of the last-observation-carried-forward conven-
tion, in any clinical investigation conducted under 
this chapter or section 351 of the Public Health 
Service Act with respect to any life-threatening con-
dition or disease where reasonably effective approved 
alternative therapies exist for the specific indica-
tion.’’. 
SEC. 5. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS 
AND DEVICES. 
(a) IN GENERAL.—Chapter V of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is amend-
ed by adding at the end of section 561 the following: 
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‘‘(f) EXPANDED ACCESS PROGRAM.—The Food and 
Drug Administration shall establish a new program to ex-
pand access to investigational treatments for individuals 
with serious or life threatening conditions and diseases. 
In carrying out this expanded access program, the Sec-
retary shall publish and broadly disseminate written guid-
ance that— 
‘‘(1) describes such expanded access programs 
for investigational drugs, biological products, and de-
vices intended to treat serious or life-threatening 
conditions or diseases; 
‘‘(2) encourages and facilitates submission of 
Tier I and Tier II applications and approvals; and 
‘‘(3) facilitates the provision of investigational 
drugs 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. 
‘‘(g) IMPLEMENTATION OF EXPANDED ACCESS PRO-
GRAMS.— 
‘‘(1) TRAINING
OF
PERSONNEL.—Not later 
than 90 days after the date of enactment of this 
subsection, the Secretary shall implement training 
programs at the Food and Drug Administration with 
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respect to the expanded access programs established 
under this section. 
‘‘(2) POLICIES, 
REGULATIONS, 
AND
GUID-
ANCE.—The Secretary shall establish policies, regu-
lations, and guidance designed to most directly ben-
efit seriously ill patients. 
‘‘(h) DEVELOPMENT
OF SURROGATE ENDPOINTS 
AND BIOMARKERS.—The Secretary shall— 
‘‘(1) establish a program to encourage the de-
velopment 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 medical needs; 
‘‘(2) request the Institute of Medicine to under-
take 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 
‘‘(3) make widely available to the public a list 
of drugs, biological products, and devices that are 
being investigated for serious or life-threatening con-
ditions or diseases and that have not yet received 
Tier I or Tier II approval for marketing.’’. 
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(b) CONFORMING AMENDMENT.—Section 561(c) of 
the Federal Food, Drug, and Cosmetic Act is amended 
by striking the heading and inserting ‘‘EXPANDED ACCESS 
TO INVESTIGATIONAL DRUGS AND DEVICES FOR SERI-
OUSLY ILL PATIENTS’’. 
SEC. 6. MODERNIZATION OF THE FOOD AND DRUG ADMIN-
ISTRATION. 
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. 565. POLICIES RELATED TO STUDY EVALUATION IN-
FORMATION. 
‘‘(a) IN GENERAL.— 
‘‘(1) NONSTATISTICAL
MEASURES.—The Sec-
retary shall give equal weight to clinical judgment 
and statistical analysis in the evaluation of the safe-
ty and effectiveness of drugs, biological products, 
and devices, and shall not disapprove a product ap-
plication solely on the basis of a statistical analysis 
or the rigid use of the 95 percent confidence level 
convention. This policy shall apply— 
‘‘(A) in evaluating clinical study designs 
and endpoints; and 
‘‘(B) in making decisions with respect to 
product applications. 
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‘‘(2) TYPES OF NONSTATISTICAL MEASURES.— 
The policy established under paragraph (1), for the 
purposes described in such paragraph— 
‘‘(A) shall include but not be limited to 
such nonstatistical information as— 
‘‘(i) clinical evaluation information, 
such as case history reports; 
‘‘(ii) scientific and clinical studies de-
signed 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 investiga-
tional drug to even a small subpopulation 
of seriously ill patients; and 
‘‘(ii) scientific, observational, or clin-
ical studies designed and conducted to col-
lect 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 
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