June 24, 2026
The Food and Drug Administration issued three draft guidance documents addressing drug and biologic development, including product approval standards, innovative clinical trial designs and first-in-human dose selection.
The revised draft guidance, “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products,” updates the agency’s recommendations for demonstrating effectiveness. The guidance discusses factors that influence the strength of evidence supporting a product and clarifies how sponsors may rely on a single adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the substantial evidence standard.
The agency is accepting comments on the draft guidance through Sept. 22.
FDA also released a revised draft guidance, “Master Protocols for Drug and Biological Product Development,” which provides recommendations on the design, analysis and regulatory review of trials conducted under a master protocol. According to the agency, master protocols can improve efficiency by allowing multiple therapies, diseases or patient populations to be studied within a shared trial framework. The revised draft includes additional recommendations related to basket trials.
The agency is accepting comments on the draft guidance through Aug. 24.
A third draft guidance, “Quantitative Systems Pharmacology (QSP)-Based Dose Selection for Minimum Anticipated Biological Effect Level (MABEL) in First-in-Human Trials,” addresses the use of mechanistic modeling to support starting-dose selection in early clinical studies. FDA said the approach may be particularly useful for products associated with potent pharmacologic activity, including cytokine release and T-cell activation.
The agency is accepting comments on the draft guidance through July 24.
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