FDA Biomarker Qualification Evidentiary Framework Draft Guidance for Industry and FDA Staff

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Draft — Not for Implementation 1 Biomarker Qualification: Evidentiary Framework 1

2 Guidance for Industry and FDA Staff 3 4 5 This draft guidance, when finalized, will represent the current thinking of the Food and Drug 6 Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not 7 binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the 8 applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible 9 for this guidance as listed on the title page. 10 11 12 13 I. INTRODUCTION 14 2

15 This guidance for biomarker development stakeholders and Food and Drug Administration 16 (FDA) staff provides recommendations on general considerations to address when developing a 17 biomarker for qualification under the 21st Century Cures Act (Cures Act) that added new section 18 507of the Federal Food, Drug, and Cosmetic Act (FD&C Act), Qualification of Drug 3

19 Development Tools. This guidance discusses the evidentiary framework that should be used to 20 support biomarker qualification, as that term is now used in section 507 of the FD&C Act, and it 21 was informed by public workshops that predated the Cures Act. 22 23 The evidentiary framework described in this guidance identifies the recommended components 24 of a biomarker development program, including determining the type and level of evidence 25 sufficient to support qualification, and addresses how these components interrelate to inform the 26 evidentiary framework. This evidentiary framework is broadly applicable to all biomarker 27 qualification submissions, regardless of the type of biomarker or context of use (COU). 28 Qualification of a biomarker is a determination that within the stated COU, the biomarker can be 29 relied on to have a specific interpretation and application in drug development and regulatory 4 5

30 review. Thus, a qualified biomarker can be used across multiple drug development programs 31 under the COU for which it was qualified. Requests for qualification of a biomarker should 32 address the evidentiary framework discussed in this document. 33 1

This guidance has been prepared by the Office of New Drugs and the Office of Translational Sciences in the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration in cooperation with the Center for Biologics Evaluation and Research (CBER). 2

Throughout this guidance, the term biomarker is intended to include both single entity and composite biomarkers (biomarkers consisting of several individual biomarkers whose measurements are combined in a defined algorithm to reach a single interpretive output). References in this guidance to the use of a biomarker in drug development imply making a decision in drug development based upon the measurement of the biomarker. 3

Section 507 of the FD&C Act (21 U.S.C. 357) was added by section 3011(a) of the Cures Act (Public Law 114-

255). 4

FD&C Act section 507(e)(7). 5

For the purposes of this guidance, all references to drugs or drug products include both human drugs and biological drug products regulated by CDER and CBER, unless otherwise specified. 1

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