I 
109TH CONGRESS 
2D SESSION 
H. R. 6303 
To amend the Federal Food, Drug, and Cosmetic Act to create a new 
three-tiered 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 
SEPTEMBER 29, 2006 
Mr. SHAYS 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 
create a new three-tiered 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 Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Access, Compassion, 
Care, and Ethics for Seriously Ill Patients Act’’ or the 
‘‘ACCESS Act’’. 
SEC. 2. FINDINGS. 
Congress finds the following: 
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(1) The necessity of placebo controlled studies 
has been questioned on both scientific and ethical 
grounds for seriously ill patients. 
(2) The current standards of the Food and 
Drug Administration for approval of drugs, biologi-
cal products, and devices deny the benefits of med-
ical progress to seriously ill patients who face mor-
bidity or death from their disease. 
(3) Promising therapies intended to treat seri-
ous or life threatening conditions or diseases and 
which address unmet medical needs have received 
unjustified delays and denials of approval. 
(4) Seriously ill patients have a right to access 
available investigational drugs, biological products, 
and devices. 
(5) The current Food and Drug Administration 
and National Cancer Institute case-by-case exception 
for compassionate access must be required to permit 
all seriously ill patients access to available experi-
mental therapies as a treatment option. 
(6) The current emphasis on statistical analysis 
of clinical information needs to be balanced by a 
greater reliance on clinical evaluation of this infor-
mation. 
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(7) Food and Drug Administration advisory 
committees should have greater representation of 
medical clinicians who represent the interests of seri-
ously ill patients in early access to promising inves-
tigational therapies. 
(8) The use of available investigational products 
for treatment is the responsibility of the physician 
and the patient. 
(9) The use of combinations of available inves-
tigational and approved products for treatment is 
the responsibility of the physician and the patient. 
(10) The development and approval of drugs, 
biological products, and devices intended to address 
serious or life-threatening conditions or diseases is 
often delayed by the inability of sponsors to obtain 
prompt meetings with the Food and Drug Adminis-
tration and to obtain prompt resolution of scientific 
and regulatory issues related to the investigation 
and review of new technologies. 
SEC. 3. TIERED APPROVAL SYSTEM FOR DRUGS, BIOLOGI-
CAL PRODUCTS, AND DEVICES. 
Section 506 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 356) is amended to read as follows: 
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‘‘SEC. 506. TIERED APPROVAL SYSTEM. 
‘‘(a) IN GENERAL.—Notwithstanding any other pro-
vision of law, the sponsor of an investigational drug, bio-
logical product, or device may submit an application to 
the Secretary for Tier I or Tier II approval in accordance 
with this section. 
‘‘(b) TIER I APPROVAL.— 
‘‘(1) IN GENERAL.— 
‘‘(A) APPLICATION CONTENT.—A sponsor 
of an investigational drug, biological product, or 
device applying for Tier I approval of the prod-
uct shall submit to the Secretary an application 
as described under section 505(b)(1) or 
505(b)(2), section 351(a) of the Public Health 
Service Act, or section 510(k) or 515(c)(1), as 
applicable, 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 against a serious 
or life-threatening condition or disease, 
which evidence may be based on uncon-
trolled data such as case histories, infor-
mation about the pharmacological mecha-
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nism of action, data from animal and com-
puter models, comparison with historical 
data, or other preliminary information, and 
may be based on a small number of pa-
tients; and 
‘‘(iii) an assurance that the sponsor 
will continue clinical investigation to obtain 
Tier III approval. 
‘‘(B) LIMITATION.—Tier I approval shall 
be primarily based upon clinical evaluation, not 
statistical analysis. 
‘‘(2) DETERMINATION BY SECRETARY.— 
‘‘(A) IN
GENERAL.—Not later than 30 
days after the receipt of an application for Tier 
I approval, the Secretary shall either— 
‘‘(i) approve the application; or 
‘‘(ii) refer the application to the Accel-
erated Approval Advisory Committee. 
‘‘(B) RECOMMENDATION.—Within 90 days 
after receipt of an application for approval, the 
Accelerated Approval Advisory Committee shall 
issue a recommendation to the Secretary on 
whether the Secretary should approve the appli-
cation. 
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‘‘(C) FINAL
DECISION.—Within 30 days 
after receipt of the recommendation from the 
Accelerated Approval Advisory Committee, the 
Secretary shall either approve the application or 
shall issue an order setting forth a detailed ex-
planation of the reasons why the application 
was not approved and the specific data that the 
sponsor must provide so that the application 
may be approved. 
‘‘(3) APPEAL.—If the Secretary does not ap-
prove an application for which the Accelerated Ap-
proval Advisory Committee recommended approval, 
the sponsor of the application shall have the right to 
appeal the decision to the Commissioner of Food 
and Drugs. The Commissioner shall provide the 
sponsor with a hearing within 30 days following the 
nonapproval of the application and shall issue an 
order within 30 days following the hearing either 
concurring in the nonapproval or approving the ap-
plication. The Commissioner shall not delegate the 
responsibility described in this paragraph to any 
other person. 
‘‘(4) CRITERIA.—In making a determination 
under paragraph (2), the Secretary shall consider 
whether the totality of the information available to 
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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 
disease outweighs the potential risk of the product, 
and the product may possibly provide benefit to the 
patient, the Secretary shall approve the application. 
‘‘(5) PRODUCT
LABELING.—The labeling ap-
proved by the Secretary for the drug, biological 
product, or device— 
‘‘(A) shall state that the product is in-
tended for use by a patient whose physician has 
documented in writing that the patient has— 
‘‘(i) exhausted 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, biologi-
cal product, or device for which such indi-
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vidual is a reasonable candidate (which 
may include consideration of the lack of a 
source of supply or geographic factors); 
and 
‘‘(B) shall state 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; 
‘‘(ii) a written waiver of the right to 
sue the manufacturer or sponsor of the 
drug, biological product, or device, or the 
physicians who prescribed the product or 
the institution where it was administered, 
for an adverse event caused by the prod-
uct, which shall be binding in every State 
and Federal court; and 
‘‘(iii) 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 Tier II or Tier III ap-
proval. 
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‘‘(6) LIMITATION ON CONDITIONS.—Tier I ap-
proval may be subject to the requirement that the 
sponsor conduct appropriate post-approval studies. 
‘‘(c) TIER II APPROVAL.— 
‘‘(1) IN GENERAL.—A sponsor of an investiga-
tional drug, biological product, or device applying for 
Tier II approval shall submit to the Secretary an ap-
plication as described under section 505(b)(1) or 
505(b)(2), section 351(a) of the Public Health Serv-
ice Act, or section 510(k) or 515(c)(1), as applica-
ble, 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) an assurance that the sponsor will 
continue clinical investigation to obtain Tier III 
approval. 
‘‘(2) DETERMINATION BY SECRETARY.— 
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‘‘(A) IN
GENERAL.—Not later than 30 
days after the receipt of an application for Tier 
II approval, the Secretary shall either— 
‘‘(i) approve the application; or 
‘‘(ii) refer the application to the Accel-
erated Approval Advisory Committee. 
‘‘(B) RECOMMENDATION.—Within 90 days 
after receipt of an application for approval, the 
Accelerated Approval Advisory Committee shall 
issue a recommendation to the Secretary on 
whether the Secretary should approve the appli-
cation. 
‘‘(C) FINAL
DECISION.—Within 30 days 
after receipt of the recommendation from the 
Accelerated Approval Advisory Committee, the 
Secretary shall either approve the application or 
issue an order setting forth a detailed expla-
nation of the reasons why the application was 
not approved and the specific data that the 
sponsor must provide so that the application 
may be approved. 
‘‘(3) APPEAL.—If the Secretary does not ap-
prove an application for which the Accelerated Ap-
proval Advisory Committee recommended approval, 
the sponsor of the application shall have the right to 
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appeal the decision to the Commissioner of Food 
and Drugs. The Commissioner shall provide the 
sponsor with a hearing within 30 days following the 
nonapproval of the application and shall issue an 
order within 30 days following the hearing either 
concurring in the nonapproval or approving the ap-
plication. The Commissioner shall not delegate the 
responsibility described in this paragraph to any 
other person. 
‘‘(4) LIMITATION ON CONDITIONS.— 
‘‘(A) POST-APPROVAL
STUDIES.—Tier II 
approval may be subject to the requirement 
that the sponsor conduct appropriate post-ap-
proval studies to validate the surrogate end-
point or biomarker or otherwise confirm the ef-
fect on the clinical endpoint. 
‘‘(B) RULE OF CONSTRUCTION.—Nothing 
in this subsection shall be construed to permit 
the Secretary to condition Tier II approval on 
compliance with any other standards, including 
any standard necessary to meet Tier III ap-
proval. 
‘‘(d) TIER III APPROVAL.—For purposes of this Act, 
the term ‘Tier III approval’ means— 
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‘‘(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). 
‘‘(e) PROMOTIONAL
MATERIALS.—Approval of a 
product under either Tier I or II may be subject to the 
requirements that— 
‘‘(1) the sponsor submit copies of all advertising 
and promotional materials related to the product 
during the preapproval review period and, following 
approval and for such period thereafter as the Sec-
retary determines to be appropriate, and at least 30 
days prior to the dissemination of the materials; 
‘‘(2) all advertising and promotional materials 
prominently disclose the limited approval for the 
product and data available supporting the safety and 
effectiveness of the product; and 
‘‘(3) the sponsor shall not disseminate adver-
tising or promotional material prior to obtaining 
written notification from the Secretary that the ad-
vertising or promotional material complies with this 
subchapter. 
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