Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products; Draft Guidance for Industry; Availability

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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products." This guidance complements and expands on the 1998 guidance entitled "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products" (the 1998 guidance). Although FDA's evidentiary standard for effectiveness has not changed since 1998, the evolution of drug development and science has led to changes in the types of drug development programs submitted to the Agency. Specifically, there are more programs studying serious diseases lacking effective treatment, more programs in rare diseases, and more programs for therapies targeted at disease subsets. There is a need for more Agency guidance on the flexibility in the amount and type of evidence needed to meet the substantial evidence standard in these circumstances.