Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products; Guidance for Industry; Draft Guidance

The EHE Foundation, an advocacy organization for patients with an ultra-rare cancer, supports the revised draft guidance because it provides a realistic pathway for developing treatments for rare diseases with small patient populations. They request further clarification on evidentiary bars for natural history data, the scope of mechanistic-evidence pathways for gene-fusion-driven cancers, and dedicated engagement mechanisms for small sponsors.

Truveta, a healthcare data company, supports the FDA's efforts to accelerate the transition from drug discovery to first-in-human studies. They argue that their longitudinal real-world data and AI-enabled analytics can improve disease characterization, protocol design, patient recruitment, and site selection for clinical trials.