IGT Hill Briefing: The Value of Gene Therapy: Protecting Children's Access, American Innovation
- Jun 23
- 1 min read
As Congress considers Most Favored Nation (MFN) pricing proposals, lawmakers are overlooking a critical consequence: children with rare diseases could lose access to potentially curative gene therapies.
The Institute for Gene Therapies (IGT) is raising concerns that MFN pricing mechanisms would effectively unravel the Medicaid Best Price reforms designed to protect vulnerable patient
populations. These reforms have enabled innovative payment models that make high-cost, one-time gene therapies more accessible. Applying MFN in this space could undermine those models and discourage manufacturers from bringing transformative treatments to the U.S. market.
Importantly, the proposal mirrors pricing approaches seen in several European countries, where restrictive policies have contributed to delayed or limited access to gene therapies. The result has been fewer treatment options for patients with urgent, unmet medical needs.
On June 18, IGT convened a briefing to examine these risks and highlight the potential impact on patients, providers, and the broader innovation ecosystem. The discussion brought together leading experts across policy, industry, and patient advocacy:
Speakers included:
Erik Paulsen, Chair, IGT (Moderator)
John Feore, Director of Health Policy & Advocacy, IGT
John M. O’Brien, PharmD, MPH, President and CEO, National Pharmaceutical Council; former Senior Advisor to the U.S. Secretary of Health and Human Services; former Deputy Assistant Secretary for Planning and Evaluation (Health Policy)
Christina M. Hartman, Head of U.S. Government Affairs, Orchard Therapeutics
Amera Bilal, Vice President of Programs and Policy, Caregiver Action Network
Watch the full discussion here and read our policy brief here to better understand what’s at stake for patients and innovation.
