[Federal Register: April 14, 2005 (Volume 70, Number 71)]
[Notices]
[Page 19764-19765]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr14ap05-50]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 2005D-0122]
Draft Guidance for Industry on Exploratory Investigational New
Drugs 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availability of a draft guidance for industry entitled ``Exploratory
IND Studies.'' This draft guidance clarifies what preclinical and
clinical issues (including chemistry, manufacturing, and controls
issues) should be considered when planning exploratory studies in
humans, including studies of closely related drugs or biologics, under
an investigational new drug (IND) application. This draft guidance
emphasizes the concept that limited investigations in humans can be
initiated with more limited preclinical support because such studies
present fewer potential risks than do traditional phase 1 studies that
look for dose-limiting toxicities.
DATES: Submit written or electronic comments on the draft guidance by
July 13, 2005. General comments on agency guidance documents are
welcome at any time.
ADDRESSES: Submit written requests for single copies of the 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
draft guidance to the Division of Dockets Management (HFA-305), Food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access to the draft guidance document.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: 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FDA is announcing the availability of a draft guidance for industry
entitled ``Exploratory IND Studies.'' In its March 2004 Critical Path
Report, the agency explained that to reduce the time and resources
expended during early drug development on candidates that are unlikely
to succeed, 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.
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. Nevertheless, sponsors have not
always taken advantage of that flexibility and limited, early phase 1
studies, such as those described in this document, are often supported
by a more extensive preclinical database than is needed. In many cases,
a more extensive workup is done because sponsors intend to move
immediately into a more traditional phase 1 trial if the screening
results are favorable. Because exploratory studies will typically
involve administering either subtherapeutic doses of a product, or
doses expected to produce a pharmacological, but not a toxic effect,
the potential risk to human subjects is less than for a traditional
phase 1 study that, for example, seeks to establish a maximally
tolerated dose.
This guidance applies to exploratory studies (i.e., early phase 1
clinical studies), involving investigational new drug and biological
products, that assess feasibility for further development of a drug or
biological product. 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 intent.
Typically, these exploratory studies are conducted prior to the
traditional
[[Page 19765]]
dose evaluation, safety, and tolerance studies that ordinarily initiate
a clinical drug development program. Thus, FDA believes that,
typically, the duration of dosing would be limited (e.g., 7 days). The
agency is, however, interested in soliciting comment from the public on
the appropriate duration of dosing for such exploratory studies.
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. Thus, this guidance emphasizes the concept that limited
investigations in humans can be initiated with more limited preclinical
support because such studies present fewer potential risks than do
traditional phase 1 studies that look for dose-limiting toxicities. The
studies discussed here ordinarily do not have therapeutic intent. They
are designed to evaluate whether a particular candidate should be
entered into a drug development program.
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 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 draft guidance contains information collection provisions that
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 in this guidance has been approved under OMB
control number 0910-0014 and expires on January 31, 2006.
III. Comments
Interested persons may submit to the Division of Dockets Management
(see ADDRESSES) written or electronic comments on the draft guidance.
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. 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.
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: April 8, 2005.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 05-7485 Filed 4-13-05; 8:45 am]
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