Guidance for Industry on Postmarketing Studies and Clinical Trials-Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act; Availability

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA) is announcing the availability of a guidance for industry entitled “Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act.” The Food and Drug Administration Amendments Act of 2007 (FDAAA) added new provisions to the Federal Food, Drug, and Cosmetic Act (the FD&C Act) authorizing FDA to require certain postmarketing studies and clinical trials for prescription drugs approved under the FD&C Act and biological products approved under the Public Health Service Act (the PHS Act). This guidance provides information on the implementation of the new provisions and a description of the types of postmarketing studies and clinical trials that will generally be required under the new legislation (postmarketing requirements (PMRs)) and the types that will generally be agreed-upon commitments (postmarketing commitments (PMCs)) because they do not meet the new statutory criteria for required postmarketing studies and clinical trials.

DATES:

Submit either electronic or written comments on Agency guidances at any time.

ADDRESSES:

Submit written requests for single copies of this 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 (CBER), Food and Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448. The guidance may 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 the office in processing your requests.

Submit electronic comments 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:

Nancy Dickinson, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 6226, Silver Spring, MD 20993-0002, 301-796-5400; or

Stephen Ripley, Center for Biologics Evaluation and Research (HFM-17), Food and Drug Administration, 1401 Rockville Pike, Rockville, MD 20852-1448, 301-827-6210.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a guidance for industry entitled “Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act.” In the past, FDA has used the term “PMC” to refer to studies (including clinical trials), conducted by an applicant after FDA has approved a drug for marketing or licensing, that were intended to further refine the safety, efficacy, or optimal use of a product, or to ensure consistency and reliability of product quality. These commitments were either agreed upon by FDA and the applicant or, in certain circumstances, required by FDA. Prior to the passage of FDAAA, FDA required PMCs in the following situations:

• Subpart H and subpart E accelerated approvals, which require postmarketing studies to demonstrate clinical benefit (21 CFR 314.510 and 601.41);
• Deferred pediatric studies, where studies are required under the Pediatric Research Equity Act (section 505, FD&C Act); and
• Animal Efficacy Rule approvals, where studies to demonstrate safety and efficacy in humans are required at the time of use (21 CFR 314.610(b)(1) and 601.91(b)(1)).

Title IX, section 901 of FDAAA (Pub. L. 110-85) amended the FD&C Act by adding new section 505(o) (21 U.S.C. 355(o)). Section 505(o)(3) of the FD&C Act authorizes FDA to require certain postmarketing studies or clinical trials for prescription drugs approved under section 505(b) of the FD&C Act and biological products approved under section 351 of the PHS Act (42 U.S.C. 262). Section 505(o)(3)(B) of the FD&C Act states that postmarketing studies and clinical trials may be required for one of three purposes:

• To assess a known serious risk related to the use of the drug;
• To assess signals of serious risk related to the use of the drug; or
• To identify an unexpected serious risk when available data indicates the potential for a serious risk.

This guidance provides information on the implementation of new section 505(o)(3) of the FD&C Act. The guidance also describes which types of postmarketing studies and clinical trials will be required (PMRs) under section 505(o)(3) and which types will be agreed-upon commitments because they do not meet the statutory criteria for required studies and trials (PMCs).

In the Federal Register of July 15, 2009 (74 FR 34358), FDA announced the availability of a draft guidance for industry entitled “Postmarketing Studies and Clinical Trials—Implementation of Section 505(o)(3) of the Federal Food, Drug, and Cosmetic Act.” The notice gave interested persons the opportunity to comment by October 13, 2009. The draft guidance was revised in response to comments submitted to the docket requesting that the guidance clearly distinguish PMRs required under section 505(o)(3) of the FD&C Act from risk evaluation and mitigation strategies. The revisions also provide additional detail in the examples of PMRs and PMCs and clarify reporting requirements.

This guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents the Agency's current thinking on the implementation of section 901 of FDAAA on postmarketing studies and clinical trials. 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. It is no longer necessary to send two copies of mailed 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. Paperwork Reduction Act of 1995

This guidance provides information on the implementation of section 901 of FDAAA. The collections of information requested in the draft guidance would be submitted under 21 CFR 314.80, 314.81, and 601.70. These collections of information are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520) and are approved under OMB control numbers 0910-0230, 0910-0001, and 0910-0338. Section VI of the guidance refers to procedures in the guidance entitled “Formal Dispute Resolution: Appeals Above the Division Level,” which contains collections of information approved under OMB control number 0910-0430.

IV. 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,, or http://www.regulations.gov .