FDA Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products Draft

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Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products Draft[附网盘链接]是于过去发布的FDA标准，适用于US。

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Draft — Not for Implementation 1 Demonstrating Substantial Evidence of Effectiveness for 2 Human Drug and Biological Products 3 Guidance for Industry 4 5 6 This draft guidance, when finalized, will represent the current thinking of the Food and Drug 7 Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not 8 binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the 9 applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible 10 for this guidance as listed on the title page. 11 12 13 14 I. INTRODUCTION 15 16 This document is intended to provide guidance to applicants planning to file new drug 17 applications (NDAs), biologics license applications (BLAs), or applications for supplemental 18 indications on the evidence to be provided to demonstrate effectiveness. This guidance 19 complements and expands on the 1998 guidance entitled Providing Clinical Evidence of 1

20 Effectiveness for Human Drug and Biological Products (the 1998 guidance) . 21 22 The 1998 guidance was issued in response to the Food and Drug Administration Modernization 23 Act of 1997 (FDAMA) (Pub. L. 105–115), which stated that the substantial evidence 24 requirement for effectiveness, which had generally been interpreted as calling for two adequate 2

25 and well-controlled trials, could also be met by a single trial plus confirmatory evidence. The 26 1998 guidance, therefore, provided many examples of the types of evidence that could be 27 considered confirmatory evidence, with a specific focus on adequate and well-controlled trials of 28 the test agent in related populations or indications, as well as a number of illustrations of a single 29 adequate and well-controlled trial supported by convincing evidence of the drug’s mechanism of 30 action in treating a disease or condition. 31 32 FDAMA thus introduced a specific new area of flexibility in the evidence needed to support 33 effectiveness, but there are many other characteristics of the evidence supporting effectiveness 34 that can vary (notably, trial designs, trial endpoints, statistical methodology), and evidence that 35 varies in such ways potentially can provide substantial evidence of effectiveness but because of 36 these characteristics may provide greater or lesser certainty. These characteristics also deserve 37 consideration and were not discussed in the 1998 guidance. FDA’s consideration of these 38 various designs, endpoints, and analyses which can differ in the strength of evidence they 1

FDA updates guidances periodically. To make sure you have the most recent version of a guidance, check the FDA webpage. The guidances mentioned in this document are available on the guidance web page at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, and https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics. 2

In this guidance, the terms “trial” and “clinical trial” have the same meaning as the term “clinical investigation” as the latter is defined in FDA regulations (see, e.g., 21 CFR 312.3(b)).

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