[Federal Register Volume 71, Number 10 (Tuesday, January 17, 2006)]
[Notices]
[Pages 2551-2552]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 06-354]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2005D-0122]


Guidance for Industry on Exploratory Investigational New Drug 
Studies; 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 guidance for industry entitled ``Exploratory IND 
Studies.'' This guidance describes the preclinical and clinical issues 
as well as chemistry, manufacturing, and controls information that 
should be considered when planning exploratory studies, including 
studies of closely related drugs or biologics, under an investigational 
new drug (IND) application.

DATES: Submit written or electronic comments on agency guidances at any 
time.

ADDRESSES: Submit written requests for single copies of this 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. Send one self-addressed adhesive label to 
assist that office in processing your requests. Submit written comments 
on the 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 guidance document.

FOR FURTHER INFORMATION CONTACT: David Jacobson-Kram, Center for Drug 
Evaluation and Research (HFD-24), Food and Drug Administration, 5600 
Fishers Lane, Rockville, MD 20857, 301-443-5346.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Exploratory IND Studies.'' In its March

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2004, Critical Path Report,\1\ the agency explained that to reduce the 
time and resources expended during early drug development on candidates 
that are unlikely to succeed,\2\ tools are needed to allow developers 
to distinguish earlier in the process those candidates that hold 
promise from those that do not. This guidance describes some 
exploratory approaches that will protect human subjects while providing 
early information about candidate performance in humans.
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    \1\Food and Drug Administration, ``Innovation or Stagnation, 
Challenge and Opportunity on the critical Path to New Medical 
Products,'' March 2004.
    \2\A new medical compound entering phase 1 testing, often 
representing the culmination of upwards of a decade of preclinical 
screening and evaluation, is estimated to have only an eight percent 
chance of reaching the market, ``Critical Path Report,'' March 2004.
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    Exploratory IND studies have a number of different goals. In some 
cases, an exploratory study can help developers gain an understanding 
of the relationship between a specific mechanism of action and the 
treatment of a disease. In other cases, a study can provide important 
information on pharmacokinetics, including, for example, 
biodistribution of a candidate drug. Whatever the goal of the study, 
exploratory IND studies can help sponsors identify, early in the 
process, promising candidates for continued development.
    Existing regulations allow a great deal of flexibility in terms of 
the amount of data that need to be submitted in an IND application, 
depending on the goals of an investigation, the specific human testing 
being proposed, and the expected risks. But sponsors have not always 
taken advantage of that flexibility, and limited, early phase 1 
studies, such as those described in this guidance, are often supported 
by a more extensive preclinical database than is needed.
    This guidance applies to exploratory studies (i.e., early phase 1 
clinical studies), involving IND and biological products, that assess 
feasibility for further development of a drug or biological product.\3\ 
For the purposes of this guidance the phrase ``exploratory study'' is 
intended to describe clinical trials that occur very early in phase 1, 
involve very limited human exposure, and often have no therapeutic or 
diagnostic intent.
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    \3\This guidance applies to drug and certain well-characterized 
therapeutic biological products (e.g., recombinant therapeutic 
proteins and monoclonal antibodies regulated by the Center for Drug 
Evaluation and Research). The guidance does not apply to human cell 
or tissue products, blood and blood proteins, vaccines, or to 
products regulated as devices.
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    Typically, these exploratory studies are conducted prior to the 
traditional dose evaluation, safety, and tolerance studies that 
ordinarily initiate a clinical drug development program. The amount and 
type of preclinical information necessary to support an exploratory 
study will depend on the planned nature and extent of human exposure 
relative to the toxicity (or lack thereof) at the planned dose. The 
studies discussed in this guidance ordinarily do not have therapeutic 
intent. They are designed to evaluate whether a particular candidate 
should be entered into a drug development program.
    FDA published a notice in the Federal Register of April 14, 2005 
(70 FR 19764), announcing the availability of a draft version of this 
guidance. The agency was interested in soliciting input on the draft 
guidance. The comment period closed on July 13, 2005. A number of 
comments were received on the draft, and the agency considered them 
very carefully during finalization of the guidance. A number of 
clarifying changes were made during finalization of the guidance, but 
substantive changes were not made.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
agency's current thinking on exploratory IND studies. 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. Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The 
collection of information has been approved under OMB control number 
0910-0014.

III. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic comments regarding this document. 
Submit a single copy of electronic comments or two paper copies of any 
mailed comments, except that individuals may submit one paper copy. 
Comments are to be identified with the docket number found in brackets 
in the heading of this document. Received comments may be seen in the 
Division of Dockets Management between 9 a.m. and 4 p.m., Monday 
through Friday.

IV. Electronic Access

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

    Dated: January 3, 2006.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 06-354 Filed 1-12-06; 8:45 am]
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