[Federal Register: July 23, 2004 (Volume 69, Number 141)]
[Notices]               
[Page 44039-44040]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr23jy04-79]                         

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2001D-0582]

 
Guidance for Industry on Available Therapy; 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 ``Available Therapy.'' 
The document is intended to provide guidance to industry on the meaning 
of the term ``available therapy'' as used by the Center for Drug 
Evaluation and Research (CDER) and the Center for Biologics Evaluation 
and Research (CBER).

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://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments.
 See the SUPPLEMENTARY INFORMATION section for electronic 

access to the guidance document.

FOR FURTHER INFORMATION CONTACT:
For information regarding human drug products: Janet Jones, Center for 
Drug Evaluation and Research (HFD-040), Food and Drug Administration, 
5600 Fishers Lane, Rockville, MD 20857, 301-594-5445.
For information regarding biological products: Robert Yetter, Center 
for Biologics Evaluation and Research (HFM-10), Food and Drug 
Administration, 1401 Rockville Pike, Rockville, MD 20852-1148, 301-827-
0373.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Available Therapy.'' The term ``available therapy'' and 
related terms, such as ``existing treatments'' and ``existing 
therapy,'' appear in a number of regulations and policy statements 
issued by CDER and CBER, but these terms have never been formally 
defined by the agency. Some confusion has arisen about, for example, 
whether ``available therapy'' refers only to products approved by FDA 
for the use in question, or whether the term could also refer to 
products used off-label or to treatments not regulated by FDA, such as 
surgery. The guidance document is intended to inform the public of the 
agency's interpretation of the term ``available therapy.''
    In the Federal Register of February 7, 2002 (67 FR 5831), FDA 
announced the availability of a draft guidance entitled ``Available 
Therapy.'' The document provided interested persons an opportunity to 
submit comments by April 8, 2002. On October 17, 2002, the United 
States District Court for the District of Columbia invalidated the 
``Regulations Requiring Manufacturers to Assess the Safety and 
Effectiveness of New Drugs and Biological Products in Pediatric 
Patients'' (the pediatric rule) and enjoined FDA from enforcing the 
rule. (See Association of Am. Physicians and Surgeons, Inc. v. United 
States Food and Drug Admin., 2002 U.S. Dist. LEXIS 19689 (Oct. 17, 
2002).) As a result, FDA has deleted all references to the pediatric 
rule in the guidance.
    In addition, FDA has revised the definition of ``available 
therapy.'' The revised definition seeks to resolve issues raised in 
comments requesting clarification of the proposed definition and 
confusion about situations where the only available therapy has been 
approved under the accelerated approval regulations (21 CFR 314.500 and 
601.40). The term ``available therapy'' has been revised to explain

[[Page 44040]]

that the existence of a therapy already approved under the accelerated 
approval regulations will not necessarily preclude additional therapies 
for the same specific indication from being approved under the 
accelerated approval regulations or designated for the Fast Track drug 
development programs.
    The revisions to the definition of ``available therapy'' affect 
FDA's Fast Track drug development programs. As a result, FDA has 
similarly revised its guidance for industry on Fast Track Drug 
Development Programs--Designation, Development and Application Review 
to discuss situations where the only available therapy is approved 
under the accelerated approval regulations.
    This Level 1 guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). It represents the 
agency's current thinking on this topic. 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 comments on the guidance. Two 
copies of any mailed comments are to be submitted, 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 
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 
either http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/ohrms/dockets/default.htm, http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cder/guidance/index.htm, or http://www.fda.gov/cber/

guidelines.htm.

    Dated: July 16, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-16761 Filed 7-22-04; 8:45 am]

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