[Federal Register Volume 63, Number 182 (Monday, September 21, 1998)]
[Notices]
[Pages 50251-50252]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 98-25102]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. 98D-0656]
Draft Guidance for Industry on Submission of Abbreviated Reports
and Synopses in Support of Marketing Applications; 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availability of a draft guidance for industry entitled ``Submission of
Abbreviated Reports and Synopses in Support of Marketing
Applications.'' This draft guidance, which implements section 118 of
the Food and Drug Administration Modernization Act of 1997
(Modernization Act), is intended to assist applicants who wish to
submit abbreviated reports and synopses in lieu of full reports for
certain clinical studies, both in marketing applications for new
products and in supplements to approved applications. The draft
guidance describes which studies may be submitted as abbreviated
reports or synopses and describes a format for such submissions. In
addition to seeking general comments on the draft guidance, FDA is
soliciting comment on three specific issues related to certain types of
study submissions and their formats.
DATES: Written comments may be submitted on the draft guidance by
November 20, 1998. General comments on the agency guidance documents
are welcome at any time.
ADDRESSES: Copies of this draft guidance for industry are available on
the Internet at ``http://www.fda.gov/cder/guidance/index.htm'' or
``http://www.fda.gov/cber/guidelines.htm''. Submit written requests for
single copies of the draft guidance to the Drug Information Branch
(HFD-210), Center for Drug Evaluation and Research, Food and Drug
Administration, 5600 Fishers Lane, Rockville, MD 20857 or the
Manufacturers Assistance and Communication Staff (HFM-42), Center for
Biologics Evaluation and Research, Food and Drug Administration, 1401
Rockville Pike, Rockville, MD 20852-1448. Send one self-addressed
adhesive label to assist that office in processing your requests.
Submit written comments on the draft guidance to the Dockets Management
Branch (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm.
1061, Rockville, MD 20852. Comments are to be identified with the
docket number found in brackets in the heading of this document.
FOR FURTHER INFORMATION CONTACT: Debbie J. Henderson, Center for Drug
Evaluation and Research (HFD-6), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20857, 301-594-6779.
SUPPLEMENTARY INFORMATION: FDA is announcing the availability of a
draft guidance for industry entitled ``Submission of Abbreviated
Reports and Synopses in Support of Marketing Applications.'' Section
505(b)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355(b)(1)) provides that full reports of the investigations used to
demonstrate a product's safety and effectiveness be submitted in a new
drug application (NDA). Similarly, for biologics license applications
(BLA's) FDA often requires that a manufacturer submit full reports to
demonstrate that the biological product is safe, pure, and potent.
Section 118 of the Modernization Act, ``Data requirements for drugs
and biologics,'' directs FDA to issue guidance on when abbreviated
study reports may be submitted in NDA's and BLA's in lieu of full
reports. This draft guidance is intended to fulfill the requirements of
section 118 of the Modernization Act by providing guidance on the types
of studies that may be submitted in abbreviated reports or synopses.
This draft guidance also provides recommendations on the formats that
should be used.
[[Page 50252]]
The NDA regulations at 21 CFR 314.50, which define what must be
submitted in an application, do not explicitly define a ``full
report,'' but require, among other things, submission of a
``description and analysis of each controlled clinical study pertinent
to a proposed use of the drug'' and of ``any other data or information
relevant to an evaluation of the safety and effectiveness of the drug
product.''
In 1988, FDA issued ``Guidelines for the Format and Content of the
Clinical and Statistical Sections of New Drug Applications''
(hereinafter referred to as the Clin/Stat Guideline), which described
the contents of a full report of a study. This guidance called for full
study reports for studies that contributed effectiveness data as well
as safety information. For other studies, sponsors were advised to
submit abbreviated reports of the effectiveness results.
In 1996, the International Conference on Harmonisation of the
Technical Requirements for Registration of Pharmaceuticals (ICH)
``Guidelines for the Structure and Content of Clinical Study Reports''
(ICH E3) provided an updated description of the contents of a full
study report and specific provisions for submitting less-than-full
study reports.
Applicants have not used the provisions to submit less-thanfull
study reports contained in both the Clin/Stat Guideline and ICH E3 as
often as they could have because of difficulties experienced in
deciding when a full study report is required by the reviewing body.
For example, clinical drug and biologic product development programs
often include numerous clinical studies and resulting data that are not
intended to contribute to the evaluation of the effectiveness of a
product for a particular use and are not needed to support information
included in labeling. Accordingly, such studies may be submitted as
abbreviated reports or synopses, and this guidance is intended to
facilitate their submission.
In developing the guidance, FDA identified the following three
issues on which it is specifically seeking public comment:
(1) In describing the format of an abbreviated study report, the
draft guidance references selected sections of the study report format
in ICH E3 and states that the abbreviated report should include these
sections. An alternative approach considered by the agency was to
recommend that all of the sections in the ICH E3 clinical study report
format be included, but that some of them contain detailed information
while others contain only minimal information. Which of these
approaches is preferable, or is there another approach that the agency
should consider?
(2) The draft guidance indicates that, in general, applicants
should submit full reports of negative studies (studies adequately
designed to evaluate efficacy that failed to demonstrate efficacy), but
provides for the submission of abbreviated reports of such studies in
some cases with agreement from the relevant review division. Should
abbreviated reports of negative studies be recommended, and, if so,
should more detailed information be provided on these trials than is
contemplated by the proposed abbreviated report format?
(3) The draft guidance states that in the case of products that are
the subject of very limited drug development programs (those with fewer
than six studies from any phase of development designed to determine
effectiveness including dose comparison trials), full reports of all
studies ordinarily should be provided. The rationale for this provision
is that, in such programs, even studies less central to the proposed
application (e.g., related indication, different dosage form) often
form a substantive proportion of the total clinical data base. The
agency is seeking comment on whether the proposed definition of ``very
limited drug development programs'' is appropriate. Should full reports
of all studies be provided for drug development programs with fewer or
more than six studies designed to determine effectiveness, or can
commenters propose an alternative definition of ``very limited drug
development programs?''
This draft guidance represents the agency's current thinking on
submission of full study reports, abbreviated reports, and synopses of
information related to effectiveness for new drugs and biological
products. 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 statute, regulations, or both.
Interested persons may submit written comments on the draft
guidance to the Dockets Management Branch (address above). Two copies
of any comments are to be submitted, except that individuals may submit
one copy. The draft guidance and received comments are available for
public examination in the Dockets Management Branch between 9 a.m. and
4 p.m., Monday through Friday.
Dated: August 11, 1998.
William K. Hubbard,
Associate Commissioner for Policy Coordination.
[FR Doc. 98-25102 Filed 9-15-98; 4:19 pm]
BILLING CODE 4160-01-F