Annette Von Drygalski, MD, PharmD

A forward-looking analysis of how nonfactor therapies are transforming patient quality of life, clinician-patient collaboration, and the broader healthcare ecosystem for hemophilia management.

Focus on payer dynamics, cost, prior authorization processes, and the logistical aspects of transitioning patients from factor-based to nonfactor therapies, including adherence and at-home treatment implications.

Detailed discussion of Fitusiran, Marstacimab, and Concizumab, their mechanisms of action, differentiation from factor replacement, and highlights from key clinical trials (BASIS, ATLAS, Explorer 8).

Exploration of the emergence of nonfactor treatment classes, explaining how these agents function independently of missing clotting factors and their relevance to patient convenience, dosing, and efficacy.

A historical perspective on traditional factor replacement therapies, tracing their development, successes, and ongoing limitations such as short half-life, infusion requirements, and inhibitor formation.

An overview of the biological and clinical distinctions between hemophilia A and B, including factor deficiencies, prevalence, diagnostic nuances, and implications for treatment strategy.