Guidance for Industry
Standards for the Prompt Review of
Efficacy Supplements, Including
Priority Efficacy Supplements 
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
Center for Biologics Evaluation and Research (CBER)
May 1998
Procedural Guidance 4 Guidance for Industry
Standards for the Prompt Review of
Efficacy Supplements, Including
Priority Efficacy Supplements 
Comments and suggestions regarding this document should be submitted within 90 days of
publication in the Federal Register of the notice announcing the availability of the guidance.  All
comments should be identified with the docket number provided at the beginning of the notice.
Submit comments to the Dockets Management Branch (HFA-305), Food and Drug
Administration, 12420 Parklawn Dr., rm. 1-23, Rockville, MD 20857.  
After the comment period closes, comments should be provided in writing to the Center for Drug
Evaluation and Research (CDER), Food and Drug Administration, 5600 Fishers Lane, Rockville,
MD 20857; or Center for Biologics Evaluation and Research (CBER), 1401 Rockville Pike,
Rockville, MD 20852-1448.
Additional copies are available from:
The Drug Information Branch (HFD-210),Center for Drug Evaluation and Research (CDER),
5600 Fishers Lane, Rockville, MD  20857 (Tel) 301-827-4573
http://www.fda.gov/cder/guidance/index.htm
or
Office of Communication, Training, and Manufacturers Assistance (HFM-40),
Center for Biologics Evaluation and Research (CBER)
1401 Rockville Pike, Rockville, MD  20852-1448,
http://www.fda.gov/cber/guidelines.htm; (Fax) 888-CBERFAX or 301-827-3844
(Voice Information) 800-835-4709 or 301-827-1800 GUIDANCE FOR INDUSTRY1
Standards for the Prompt Review of
Efficacy Supplements, Including Priority Efficacy Supplements
I. INTRODUCTION
Section 403(a) of the Food and Drug Administration Modernization Act of 1997 (the
Modernization Act) requires that FDA publish in the Federal Register standards for the prompt
review of supplemental applications submitted for approved articles . . . .  The legislative history
indicates that this provision was directed at certain types of efficacy supplements (i.e.,
supplemental applications proposing to add a new use of an approved drug to the product
labeling).2   Section 403(b)(3) of the Modernization Act requires that FDA provide guidance to
define supplemental applications that are eligible for priority review.  This guidance fulfills both
Modernization Act requirements.
II. STANDARDS FOR THE PROMPT REVIEW OF EFFICACY SUPPLEMENTS
Section 101 of the Modernization Act reauthorized for an additional five years, with certain
technical changes, the user fee program described in the Prescription Drug User Fee Act of 1992. 
Section 101 directed that the user fees authorized by the amendments in that subtitle be
dedicated toward expediting the drug development process and the review of human drug
applications as set forth in the [performance] goals identified . . . in the letters from the Secretary
of Health and Human Services to the chairman of the Committee on Commerce of the House of
Representatives and the chairman of the Committee on Labor and Human Resources of the
Senate, as set forth in the Congressional Record.  The referenced performance goals include
standards for the review of all efficacy supplements (including those proposing to add a new use
of an approved drug to the product labeling) as follows:
1This guidance has been prepared by the Review Management Working Group comprising individuals in the
Centers for Drug Evaluation and Research (CDER) and Biologics Evaluation and Research (CBER) at the Food and
Drug Administration. This guidance document represents the Agencys current thinking on the standards for the prompt
review of efficacy supplements.  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.
2 See U.S. Congress, Senate Committee on Labor and Human Resources, Food and Drug Administration
Modernization Act of 1997, S. Rept. 105-43 on S. 830, pp.41-42, 105th Cong., 1st sess., 1 July 1997; and House
Committee on Commerce, Prescription Drug User Fee Reauthorization and Drug Regulation Act of 1997, H. Rept.
105-310 on H.R. 1411, pp. 63-64, 105th Cong., 1st sess., 7 October 1997. Fiscal Year 1998:
Standard Efficacy Supplements - 90% of reviews to be completed within 12 months
Priority Efficacy Supplements - 90% of reviews to be completed within 6 months
Fiscal Year 1999
Standard Efficacy Supplements - 30% of reviews to be completed within 10 months
              90% of reviews to be completed within 12 months
Priority Efficacy Supplements - 90% of reviews to be completed within 6 months
Fiscal Year 2000
Standard Efficacy Supplements - 50% of reviews to be completed within 10 months
     90% of reviews to be completed within 12 months
Priority Efficacy Supplements - 90% of reviews to be completed within 6 months
Fiscal Year 2001 
Standard Efficacy Supplements - 70% of reviews to be completed within 10 months
     90% of reviews to be completed within 12 months
  Priority Efficacy Supplements - 90% of reviews to be completed within 6 months
Fiscal Year 2002
Standard Efficacy Supplements - 90% of reviews to be completed within 10 months
Priority Efficacy Supplements - 90% of reviews to be completed within 6 months
The Agency intends to use these performance goals to fulfill the requirement of the Modernization
Act that it establish standards for the prompt review of efficacy supplements.
III. DEFINITION OF SUPPLEMENTS ELIGIBLE FOR PRIORITY REVIEW
Both the Center for Drug Evaluation and Research (CDER) and the Center for Biologics
Evaluation and Research (CBER) have previously established the criteria used to designate
original and supplemental applications as appropriate for priority review, and these criteria were
used in the implementation of the Prescription Drug User Fee Act of 1992.  CDERs policy
specifies, in relevant part, that an application or supplement for a drug product will receive a
priority review if the product, if approved would be a significant improvement, compared to
marketed products, including non-drug products/therapies in the treatment, diagnosis, or
prevention of a disease.3
 
3 CDER Manual of Policies and Procedures, 6020.3 - Priority Review Policy.
2 CBERs standard operating procedures specify that a biological product original or supplemental
application will receive priority review if the product, if approved, would be a significant
improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious
or life-threatening disease.4
These criteria and definitions are being used currently by the Agency to determine whether an
efficacy supplement is eligible for priority review.
4 CBER Standard Operating Procedures and Policy Manual, 8405.
3