Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products

1st July 2026

Summary: This FDA guidance explains the agency's current approach to demonstrating substantial evidence of effectiveness to support the approval of human drug and biological products. It outlines how a range of scientific evidence, including data from adequate and well-controlled clinical investigations, may be used to establish effectiveness, while recognising that different development programmes may require different evidential approaches depending on the product and disease area. The guidance is intended to promote flexibility in drug development while maintaining the scientific standards needed to demonstrate that medicines are safe and effective for their intended use.

Link: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products

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