[Federal Register Volume 71, Number 23 (Friday, February 3, 2006)]
[Notices]
[Pages 5862-5863]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E6-1433]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2006D-0044]


Draft Guidance for Industry on Patient-Reported Outcome Measures: 
Use in Medical Product Development to Support Labeling Claims; 
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availability of a draft guidance for industry entitled ``Patient-
Reported Outcome Measures: Use in Medical Product Development to 
Support Labeling Claims.'' The draft guidance was prepared by the 
Office of New Drugs and the Office of Medical Policy in the Center for 
Drug Evaluation and Research (CDER) in cooperation with the Center for 
Biologics Evaluation and Research (CBER) and the Center for Devices and 
Radiological Health (CDRH) at FDA. This document provides guidance to 
industry on the measurement of patient-reported outcomes (PROs) in 
studies to support medical product claims in approved labeling. The 
draft guidance describes how FDA evaluates PRO instruments used as 
effectiveness endpoints in clinical trials. It also describes our 
current thinking on how sponsors can develop and use PRO instruments to 
support claims in approved product labeling. By explicitly addressing 
the review issues identified in this guidance, sponsors can increase 
the efficiency of their endpoint discussions with FDA during the 
product development process, streamline FDA's review of PRO endpoint 
adequacy, and provide optimal information about the patient's 
perspective of treatment benefit at the time of product approval.

DATES:  Submit written or electronic comments on the draft guidance by 
April 4, 2006. General comments on agency guidance documents are 
welcome at any time.

ADDRESSES:  Submit written requests for single copies of the draft 
guidance to the Division of Drug Information (HFD-240), Center for Drug 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; or the Office of Communication, Training, 
and Manufacturers Assistance (HFM-40), Center for Biologics Evaluation 
and Research, Food and Drug Administration, 1401 Rockville Pike, 
Rockville, MD 20852-1448. The guidance can also be obtained by mail by 
calling CBER at 1-800-835-4709 or 301-827-1800. Send one self-addressed 
adhesive label to assist that office in processing your requests. 
Submit written comments on the draft 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://www.fda.gov/dockets/ecomments. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the draft guidance 
document.

FOR FURTHER INFORMATION CONTACT: Laurie B. Burke, Center for Drug 
Evaluation and Research (6411), Food and Drug Administration, 10903 New

[[Page 5863]]

Hampshire Ave., Bldg. 22, rm. 6478, Silver Spring, MD 20993-0002, 301-
796-0700; or
    Toni Stifano, Center for Biologics Evaluation and Research (HFM-
600), Food and Drug Administration, 1401 Rockville Pike, suite 200N, 
Rockville, MD 20852, 301-827-6190.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Patient-Reported Outcome Measures: Use in Medical Product 
Development to Support Labeling Claims.'' The term ``PRO'' refers to 
one or more concepts about how patients feel or function as perceived 
and reported by study subjects (i.e., ``patients''). PROs may represent 
traditional aspects of health such as symptoms and activities of daily 
living, or broader concepts such as physical function, well-being 
related to health, and satisfaction with treatment. ``PRO instruments'' 
are the tools for measuring PROs.
    Generally, sponsors can use study results measured by PRO 
instruments to support claims in approved product labeling if the 
claims are derived from adequate and well-controlled investigations 
using PRO instruments that reliably and validly measure the specific 
concepts claimed. The amount of evidence expected to support a labeling 
claim measured by a PRO instrument is the same as that required for any 
other labeling claim. As with other labeling claims, the determination 
of whether the endpoint is an adequate measure of effectiveness is 
specific to the intended population, the characteristics of the 
condition or disease treated, and the sensitivity of the clinical study 
used to measure the endpoint.
    This draft guidance presents our current thinking on the review 
process concerning the development, validation, and application of PRO 
instruments in the clinical study setting.
    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 patient-
reported outcome measures. 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 regarding this document. 
Submit a single copy of electronic comments or two paper copies of any 
mailed comments, except that individuals may submit one paper copy. 
Comments are to be identified 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 
http://www.fda.gov/ohrms/dockets/default.htm, http://www.fda.gov/cder/guidance/index.htm, or http://www.fda.gov/cber/guidelines.htm.

    Dated: January 26, 2006.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. E6-1433 Filed 2-2-06; 8:45 am]
BILLING CODE 4160-01-S