FDA’s June 2026 draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, provides a formal framework for one of the year’s most significant regulatory policy developments: one robust pivotal trial, supported by confirmatory evidence, is emerging as the default pathway for establishing effectiveness — shifting the focus away from the long-standing assumption that two pivotal trials are required.

For sponsors, this shift introduces important flexibility but also raises critical questions. What constitutes sufficient evidence? How should sponsors adapt their clinical development and evidence-generation strategies?

In this webinar, Cytel experts will examine the draft guidance in detail and explore what FDA’s evolving approach means for clinical development and evidence-generation strategies.

Key Topics: 

• The different pathways for providing substantial evidence of effectiveness and FDA’s expectations for each.
• How the robustness of trial design and execution contributes to the strength of the evidence
• The role external data can play in providing confirmatory evidence, including considerations for real-world evidence and external controls.

Speakers: 

• Jürgen Hummel – Vice President, Innovative Statistics
• Nathalie Horowicz-Mehler – Vice President, Global Head HEOR and RWE