I 
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 Com-
mittee 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, bio-
logical 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 Representa-
tives 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’’. 
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SEC. 2. FINDINGS. 
Congress finds the following: 
(1) As of 2009, the standards of the Food and 
Drug Administration for approval of drugs, biologi-
cal 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 de-
vices. 
(3) The emphasis on statistical analysis of clin-
ical 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 pro-
viding 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 rep-
resent 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. 
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(6) The use of combinations of available inves-
tigational 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 individual-
ized or personalized therapies. 
SEC. 3. COMPASSIONATE INVESTIGATIONAL ACCESS AP-
PROVAL 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 fol-
lowing: 
‘‘(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 mar-
keting begins. 
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‘‘(2) ACCESS.—Notwithstanding any other pro-
vision 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 Sec-
retary shall permit such investigational drug, biologi-
cal product, or device, to be made available for ex-
panded access under a treatment investigational new 
drug application or treatment investigational device 
exemption if the Secretary determines that the re-
quirements of this section are met with respect to 
Compassionate Investigational Access. 
‘‘(3) COMPASSIONATE
INVESTIGATIONAL
AC-
CESS.—Notwithstanding any other provision of law, 
an investigational drug, biological product, or device 
that receives approval for Compassionate Investiga-
tional Access under this subsection shall be subject 
to the provisions of section 505(i) or 520(g), as ap-
plicable, and regulations promulgated by the Sec-
retary pursuant to this Act. The Secretary and the 
sponsor may inform national, State, and local med-
ical associations and societies, voluntary health asso-
ciations, and other appropriate persons about the 
availability of an investigational drug or investiga-
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tional device under Compassionate Investigational 
Access as approved under this subsection. The infor-
mation 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 Investiga-
tional Access approval of the product shall sub-
mit to the Secretary a notice of claimed exemp-
tion under section 505(i) or 520(g), as applica-
ble, (referred to in this subsection as an ‘appli-
cation for Compassionate Investigational Ac-
cess’), which shall contain— 
‘‘(i) data and information from com-
pleted Phase I clinical investigations and 
any other nonclinical or clinical investiga-
tions; 
‘‘(ii) preliminary evidence that the 
product may be effective in humans 
against a serious or life-threatening condi-
tion or disease, which evidence may be 
based on uncontrolled data such as case 
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histories, information about the pharma-
cological 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 pa-
tient population; 
‘‘(iii) evidence that the product is safe 
at the dose and duration proposed, consid-
ering whether the potential risk to a pa-
tient of the condition or disease outweighs 
the potential risk to a patient of the pro-
posed dose and duration of treatment with 
the product, consistent with the level of in-
formation 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 Inves-
tigational Access approval shall be based upon 
multiple considerations that shall include clin-
ical evaluation and unmet patient needs. 
‘‘(5) DETERMINATION BY SECRETARY.— 
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data that the sponsor must provide so that the 
application may be so approved. 
‘‘(6) APPEAL.—If the Secretary does not pro-
vide 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 fol-
lowing the hearing either concurring in the non-
approval or so approving the application. The Sec-
retary 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 de-
vice, 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 sub-
population) 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 
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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
COMPAS-
SIONATE ACCESS.—In order for a patient to access 
a product available through Compassionate Inves-
tigational 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 reason-
able candidate, which shall include consider-
ation of a patient’s ineligibility for participation 
in clinical trials, the lack of source of supply 
and geographic factors. 
‘‘(9) PRODUCT LABELING.—To receive Compas-
sionate Investigational Access approval under this 
subsection, the sponsor of the product shall provide 
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labeling approved by the Secretary for the drug, bio-
logical product, or device that— 
‘‘(A) states that the product is intended 
for use by a patient whose physician has docu-
mented 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 reason-
able candidate; and 
‘‘(ii) unsuccessfully sought treatment, 
or obtained treatment that was not effec-
tive with an investigational drug, biological 
product, or device for which such indi-
vidual 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 de-
scribed under part 50 of title 21, Code of 
Federal Regulations (or any successor reg-
ulations); and 
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‘‘(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 INVES-
TIGATIONAL
ACCESS.—A sponsor or investigator 
may charge for a Compassionate Investigational Ac-
cess 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 com-
plete. 
‘‘(12) IMMUNITY.— 
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‘‘(A) IN GENERAL.—A manufacturer, dis-
tributor, administrator, sponsor, or physician 
who manufactures, supplies, distributes or pre-
scribes a product approved under an application 
for Compassionate Investigational Access shall 
be immune from suit or liability caused by, aris-
ing out of, or relating to the design, develop-
ment, clinical testing and investigation, manu-
facture, labeling, distribution, sale, purchase, 
donation, dispensing, prescribing, administra-
tion, efficacy, or use of a drug, biological prod-
uct, or device subject to an approved Compas-
sionate Investigational Access application. 
‘‘(B) CLAIMS.—No claim or cause of ac-
tion against a manufacturer, distributor, ad-
ministrator, sponsor, or physician who manu-
factures, 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 prop-
erty, personal injury, or death caused by, aris-
ing out of, or relating to the design, develop-
ment, clinical testing and investigation, manu-
facture, labeling, distribution, sale, purchase, 
donation, dispensing, prescribing, administra-
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tion, efficacy, or use of a drug, biological prod-
uct, or device subject to an approved Compas-
sionate Investigational Access application. Any 
such claim or cause of action that is filed in 
Federal or State court shall be immediately dis-
missed. 
‘‘(13) FINAL APPROVAL.—For purposes of this 
Act, the term ‘final approval’ means— 
‘‘(A) with respect to a new drug or new bi-
ological product, approval of such drug or prod-
uct 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, clear-
ance of such device under section 510(k) or ap-
proval 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 fol-
lowing: 
‘‘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 
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regulations to provide for the treatment of an inves-
tigational drug, biological product, or device that re-
ceives 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 investiga-
tional 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 rep-
resentative of a small patient subpopulation) 
suffering from a serious or life-threatening con-
dition or disease; and 
‘‘(B) a statement that the sponsor is ac-
tively pursuing marketing approval with due 
diligence. 
‘‘(3) DETERMINATION BY SECRETARY.— 
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‘‘(A) IN
GENERAL.—Not later than 120 
days after the receipt of an application for Ac-
celerated Approval, the Secretary shall either— 
‘‘(i) provide Accelerated Approval of 
the application; or 
‘‘(ii) refer the application to the Accel-
erated Approval Advisory Committee. 
‘‘(B) RECOMMENDATION.—Not later than 
90 days after receipt of an application for Ac-
celerated Approval, the Accelerated Approval 
Advisory Committee shall issue a recommenda-
tion to the Secretary on whether the Secretary 
should provide Accelerated Approval of the ap-
plication. 
‘‘(C) LIMITATION.—The Accelerated Ap-
proval 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 thera-
pies in determination of whether an investiga-
tional drug provides an improvement over treat-
ments that are already available. 
‘‘(D) FINAL DECISION.—Not later than 30 
days after receipt of the recommendation from 
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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 rea-
sons why the application was not so approved 
and the specific data that the sponsor must 
provide so that the application may be so ap-
proved. 
‘‘(4) APPEAL.—If the Secretary does not pro-
vide Accelerated Approval of an application, the 
sponsor of the application shall have the right to ap-
peal 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 re-
sponsibility described in this paragraph to any other 
person. 
‘‘(A) with respect to a new drug or new bi-
ological product, approval of such drug or prod-
uct under section 505(b)(1) or 505(b)(2) or 
section 351 of the Public Health Service Act, as 
the case may be; and 
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‘‘(B) with respect to a new device, clear-
ance of such device under section 510(k) or ap-
proval of such device under section 515(c)(1). 
‘‘(b) 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 
establish the Accelerated Approval Advisory Com-
mittee (referred to in this subsection as the ‘Com-
mittee’). 
‘‘(2) DELEGATION.—The Secretary may dele-
gate 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 
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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. 
‘‘(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 
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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 
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. 
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‘‘(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— 
‘‘(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; 
‘‘(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. 
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‘‘(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. 
‘‘(C) SUMMARIES.—At all meetings of the 
Committee, the Secretary shall provide a sum-
mary to the Committee of all applications sub-
mitted under this subsection and section 561(d) 
that the Committee did not consider that were 
approved by the Secretary since the last meet-
ing 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 biologi-
cal 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’’ 
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for purposes of the amendments made by this Act, consid-
ering 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 Com-
passionate 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 in-
formation required under such section.’’. 
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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 inves-
tigational 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 de-
vices 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 recog-
nizing that the use of available investigational prod-
ucts for treatment is the responsibility of the physi-
cian and the patient, and also by recognizing the 
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goal of providing additional treatment options for 
patients and their physicians to consider. 
‘‘(b) IMPLEMENTATION OF EXPANDED ACCESS PRO-
GRAMS.— 
‘‘(1) TRAINING
OF
PERSONNEL.—Not later 
than 90 days after the date of enactment of this sec-
tion, the Secretary shall implement training pro-
grams at the Food and Drug Administration with 
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. 
‘‘(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 develop-
ment 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; 
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‘‘(B) request the Institute of Medicine to 
undertake a study to identify validated surro-
gate 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 para-
graph (1)(B) shall include endpoints and biomarkers 
that address the unmet medical needs of subpopula-
tions of patients and that facilitate the development 
of individualized treatment approaches for patients 
with serious or life-threatening conditions or dis-
eases.’’. 
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SEC. 5. DEMONSTRATION PROJECT FOR COVERAGE OF 
CERTAIN DRUGS, BIOLOGICAL PRODUCTS, 
AND DEVICES UNDER THE MEDICARE PRO-
GRAM. 
(a) IN GENERAL.—The Secretary of Health and 
Human Services (in this section referred to as the ‘‘Sec-
retary’’) 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 de-
vice 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 Secu-
rity Act (42 U.S.C. 1395i) and the Federal Supple-
mentary Medical Insurance Trust Fund under sec-
tion 1841 of such Act (42 U.S.C. 1395t), in such 
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proportion as the Secretary determines to be appro-
priate, 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 Sec-
retary 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 pur-
pose 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 ap-
proved for Compassionate Investigational Access and the 
total cost of payments made under the Medicare program 
resulting from the demonstration project. The report shall 
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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 ADMIN-
ISTRATION. 
(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 IN-
FORMATION. 
‘‘(a) IN GENERAL.— 
‘‘(1) NONSTATISTICAL
MEASURES.—The Sec-
retary shall give consideration to clinical judgment 
and risks to the patient from the disease or condi-
tion involved in the evaluation of the safety and ef-
fectiveness of drugs, biological products, and devices 
that treat serious or life-threatening diseases or con-
ditions. This policy shall apply— 
‘‘(A) in evaluating clinical study designs 
and endpoints; and 
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‘‘(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 infor-
mation 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. 
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‘‘(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 em-
ployees who are intended to actively participate in the dis-
cussion shall attend a meeting. Minutes of a meeting shall 
be promptly prepared and exchanged by both parties im-
mediately following the meeting and shall accurately sum-
marize 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’’. 
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SEC. 7. MEMBERSHIP OF ONCOLOGY DRUGS ADVISORY 
COMMITTEE AND THE CELLULAR, TISSUE, 
AND GENE THERAPY ADVISORY COMMITTEE. 
Notwithstanding any other provision of law, member-
ship of the Oncology Drugs Advisory Committee, the Cel-
lular, Tissue, and Gene Therapy Advisory Committee of 
the Food and Drug Administration, and any other com-
mittee created by such Administration to evaluate or ad-
vise 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 mem-
bers of the committee. 
Æ 
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