Rare Disease Commercialization: Beyond Approval

In this episode of The Emerging Biotech Leader, host Kim Kushner sits down with Matt Trudeau, President of ITF Therapeutics, to discuss what it means to operate within a family-run pharmaceutical company and how that model shapes decision-making differently than a public or venture-backed organization.

With more than 25 years of experience spanning Genzyme, Biogen, Bluebird Bio, and now ITF, Matt shares how his career has been defined by following the science, building commercial strategies, and putting rare disease patients at the center of every decision. He reflects on ITF’s journey in rare disease, exploring the unique challenges of commercializing therapies in underserved communities, from gaining regulatory approval to ensuring meaningful patient access and payer alignment.

Matt also offers insights into:

• The evolving role of the patient voice post-approval and why commercialization is only the starting line.
• The balance of working across U.S. and European markets to deliver consistent value to small, global patient communities.
• Why the real challenge in rare diseases isn’t competing products, but overcoming complacency - ensuring patients gain access, education, and true choice among therapies.

Approval may mark a milestone, but in rare disease the real work begins afterward: breaking through barriers of access, evidence, and complacency.