[Federal Register Volume 69, Number 17 (Tuesday, January 27, 2004)]
[Notices]
[Pages 3923-3925]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 04-1591]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2004D-0014]


Draft Guidance for Industry on Information Program on Clinical 
Trials for Serious or Life-Threatening Diseases and Conditions; 
Availability

AGENCY:  Food and Drug Administration, HHS.

ACTION:  Notice.

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SUMMARY:  The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Information 
Program on Clinical Trials for Serious or Life-Threatening Diseases and 
Conditions.'' FDA is revising its March 2002 guidance

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for industry of the same title to include guidance for sponsors who 
will be submitting information required by the Best Pharmaceuticals for 
Children Act (BPCA). The BPCA amended the Public Health Service Act 
(PHS Act) to require that additional information be included in the 
Clinical Trials Data Bank established as required by the Food and Drug 
Administration Modernization Act of 1997 (Modernization Act). This 
draft guidance explains how to provide that information.

DATES:  Submit written or electronic comments on the draft guidance by 
March 29, 2004. General comments on agency guidance documents are 
welcome at any time.

ADDRESSES:  Submit written requests for single copies of the draft 
guidance to the Division of Drug Information (HFD-240), Center for Drug 
Evaluation and Research, Food and Drug Administration, 5600 Fishers 
Lane, Rockville, MD 20857 or to the Office of Communication, Training, 
and Manufacturers Assistance (HFM-40), Center for Biologics Evaluation 
and Research (CBER), Food and Drug Administration, 1401 Rockville Pike, 
Rockville, MD 20852-1448. The draft guidance may also be obtained by 
mail by calling the CBER Voice Information System at 1-800-835-4709 or 
301-827-1800. Send one self-addressed adhesive label to assist that 
office in processing your requests. Submit written comments on the 
draft guidance to the Division of Dockets Management (HFA-305), Food 
and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 
20852. Submit electronic comments to http://www.fda.gov/dockets/ecomments. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Theresa Toigo, Office of Special 
Health Issues (HF-12), Food and Drug Administration, 5600 Fishers Lane, 
Rockville, MD 20857, 301-827-4460.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Information Program on Clinical Trials for Serious or Life-
Threatening Diseases and Conditions'' to assist sponsors who will be 
submitting information to the Clinical Trials Data Bank established by 
section 113 of the Modernization Act (42 U.S.C. 282). This draft 
guidance revises the guidance of the same title issued in March 2002 
(67 FR 12022, March 18, 2002) to include assistance on submitting 
information required by the BPCA (Public Law 107-109). This draft 
guidance updates the March 2002 guidance.
    The BPCA amends section 402(j)(3)(A) of the PHS Act (42 U.S.C. 
282(j)(3)(A)) to require that additional information be included in the 
Clinical Trials Data Bank established as required under section 113 of 
the Modernization Act. Additional information to be submitted includes 
a description of whether, and through what procedure, the manufacturer 
or sponsor of an investigation of a new drug will respond to requests 
for a protocol exception, with appropriate safeguards, for single-
patient and expanded access use of the investigational drug, 
particularly in children.
    Section 113 of the Modernization Act, enacted November 21, 1997, 
directs the Secretary of Health and Human Services (the Secretary), 
acting through the Director of the National Institutes of Health (NIH), 
to establish, maintain, and operate a data bank of information on 
clinical trials for drugs to treat serious or life-threatening diseases 
and conditions. The Clinical Trials Data Bank is intended to be a 
central resource, providing current information on clinical trials to 
individuals with serious or life-threatening diseases or conditions, to 
other members of the public, and to health care providers and 
researchers.
    Specifically, section 113 of the Modernization Act requires that 
the Clinical Trials Data Bank contain the following information: (1) 
Information about Federally and privately funded clinical trials for 
experimental treatments (drug and biological products) for patients 
with serious or life-threatening diseases or conditions, (2) a 
description of the purpose of each experimental drug, (3) patient 
eligibility criteria, (4) a description of the location of clinical 
trial sites, and (5) a point of contact for patients wanting to enroll 
in the trial. Section 113 of the Modernization Act also requires that 
information provided through the Clinical Trials Data Bank be in a form 
that can be readily understood by the public (42 U.S.C. 282(j)(3)(A)). 
The BPCA, signed by the President on January 4, 2002, requires that the 
Clinical Trials Data Bank contain additional information including a 
description of whether, and through what procedure, the manufacturer or 
sponsor of an IND will respond to requests for protocol exception, with 
appropriate safeguards, for single-patient and expanded access use of 
the investigational drug, particularly in children.
    The NIH, through its National Library of Medicine (NLM) and with 
input from FDA and others, developed the Clinical Trials Data Bank. The 
first version of the Clinical Trials Data Bank was made available to 
the public on February 29, 2000, on the Internet at http://clinicaltrials.gov. At that time, the data bank included primarily NIH-
sponsored trials.
    Shortly thereafter, FDA made available two draft guidances. The 
first draft guidance provided recommendations for industry on the 
submission of protocol information to the Clinical Trials Data Bank. It 
included information about the types of clinical trials for which 
submissions are required under section 113 of the Modernization Act as 
well as information about the content of those submissions. The second 
draft guidance addressed procedural issues, including how to submit 
required and voluntary protocol information to the Clinical Trials Data 
Bank. It also discussed issues related to submitting certification to 
the Secretary that disclosure of information for a particular protocol 
would substantially interfere with the timely enrollment of subjects in 
the clinical investigation. The second draft guidance also proposed a 
timeframe for submitting the information. The March 2002 guidance 
combined the two draft guidances into a single guidance (available at 
http://www.fda.gov/cder/guidance/4856fnl.htm or http://www.fda.gov/cber/gdlns/clintrial.htm).
    This draft guidance updates the March 2002 guidance to include 
information on how to comply with new statutory requirements contained 
in the BPCA, for submitting details about single-patient use and 
expanded access use contained in the BPCA. This draft guidance also 
includes several minor updates to the information in it and to the 
format. Additional updates on procedural issues not related to the BPCA 
will be discussed in future revisions to the guidance.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the agency's current thinking on the 
information program on clinical trials for serious or life-threatening 
diseases and conditions. It does not create or confer any rights for or 
on any person and does not operate to bind FDA or the public. An 
alternative approach may be used if such approach satisfies the 
requirements of the applicable statutes and regulations.

II. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic

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comments on the draft guidance. Submit two copies of mailed comments, 
except that individuals may submit one copy. Comments are to be 
identified with the docket number found in brackets in the heading of 
this document. The draft guidance and received comments are available 
for public examination in the Division of Dockets Management between 9 
a.m. and 4 p.m., Monday through Friday.

III. Electronic Access

    Persons with access to the Internet may obtain the document at 
http://www.fda.gov/cder/guidance/index.htm, http://www.fda.gov/cber/guidelines.htm or http://www.fda.gov/ohrms/dockets/default.htm.

    Dated: January 20, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-1591 Filed 1-26-04; 8:45 am]
BILLING CODE 4160-01-S