
[Federal Register Volume 77, Number 242 (Monday, December 17, 2012)]
[Notices]
[Pages 74670-74671]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-30274]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-D-1145]


Draft Guidance for Industry on Enrichment Strategies for Clinical 
Trials to Support Approval of Human Drugs and Biological Products; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Enrichment 
Strategies for Clinical Trials to Support Approval of Human Drugs and 
Biological Products.'' The purpose of this document is to provide 
guidance to industry on enrichment strategies that can be used in 
clinical trials intended to support effectiveness and safety claims in 
new drug applications (NDAs) and biologics license applications (BLAs). 
This document defines several types of enrichment strategies, provides 
examples of various potential clinical trial designs, and discusses 
potential regulatory considerations when using enrichment strategies in 
clinical trials.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by February 15, 2013.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 2201, Silver Spring, MD 20993-0002; or 
the Office of Communication, Outreach and Development (HFM-40), Center 
for Biologics Evaluation and Research, Food and Drug Administration, 
1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448 (1-800-835-
4709 or 301-827-1800), or the Division of Small Manufacturers, 
International and Consumer Assistance, Center for Devices and 
Radiological Health, Food and Drug Administration, 10903 New Hampshire 
Ave., Bldg. 66, Rm. 4613, Silver Spring, MD 20993-0002, or fax your 
request to 301-847-8149. Send one self-addressed adhesive label to 
assist that office in processing your requests. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the draft guidance 
document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Robert Temple, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 4212, Silver Spring, MD 20993-0003, 301-
796-2270; or Stephen Ripley, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 1401 Rockville Pike, Suite 
200N, Rockville, MD 20852-1448, 301-827-6210; or Robert L. Becker, 
Center for Device and Radiological Health, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 5674, Silver 
Spring, MD 20993-0003, 301-796-5450.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Enrichment Strategies for Clinical Trials to Support 
Approval of Human Drugs and Biological Products.'' This document 
provides guidance to industry on enrichment strategies that can be used 
in clinical trials intended to support effectiveness and safety claims 
in new drug applications (NDAs) and biologics license applications 
(BLAs). Similar approaches could be used in clinical trials in earlier 
phases of drug development. As part of the reauthorization of the 
Prescription Drug User Fee Act (PDUFA IV), FDA committed to certain 
performance goals (see letters from the Secretary of Health and Human 
Services to the Chairman of the Committee on Health, Education, Labor, 
and Pensions of the Senate and the Chairman of the Committee on Energy 
and Commerce of the House of Representatives, as set forth in the

[[Page 74671]]

Congressional Record).\1\ This draft guidance addresses one of these 
goals with the creation of a guidance document that addresses enriched 
trial designs. The guidance defines and discusses three enrichment 
strategies: Decreasing heterogeneity, predictive enrichment, and 
prognostic enrichment. The guidance also discusses general clinical 
trial design considerations, provides examples of potential clinical 
trial designs, and discusses regulatory considerations when using 
enrichment strategies.
---------------------------------------------------------------------------

    \1\ See ``Section A: PDUFA Reauthorization Performance Goals and 
Procedures Fiscal Years 2008 Through 2012'' (http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm119243.htm).
---------------------------------------------------------------------------

    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 clinical 
trial designs employing enrichment strategies to support approval of 
human 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 statutes and regulations.

II. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) either electronic or written comments regarding this 
document. It is only necessary to send one set of comments. Identify 
comments with the docket number found in brackets in the heading of 
this document. Received comments may be seen 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://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/default.htm, or http://www.regulations.gov.

    Dated: November 20, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-30274 Filed 12-14-12; 8:45 am]
BILLING CODE 4160-01-P


