[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Draft Guidance for Industry on Submission of Abbreviated Reports 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availability of a draft guidance for industry entitled ``Submission of 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 This draft guidance, which implements section 118 of 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 The draft 
guidance describes which studies may be submitted as abbreviated 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addition to seeking general comments on the draft guidance, FDA is 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DATES: Written comments may be submitted on the draft guidance by 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ADDRESSES: Copies of this draft guidance for industry are available on 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single copies of the draft guidance to the Drug Information Branch 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Submit written comments on the draft guidance to the Dockets Management 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