E6-1433. Draft Guidance for Industry on Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims; Availability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The Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.” The draft guidance was prepared by the Office of New Drugs and the Office of Medical Policy in the Center for Drug Evaluation and Research (CDER) in cooperation with the Center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 The draft guidance describes how FDA evaluates PRO instruments used as effectiveness endpoints in clinical trials.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Submit written or electronic comments on the draft guidance by April 4, 2006.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Submit written requests for single copies of the draft guidance to the Division of Drug Information (HFD-240), Center for Drug Evaluation and Research, Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857;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 Submit written comments on the draft guidance to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.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 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.