Presentation (CTI)

OBJECTIVES: Following the recent shift toward Most Favored Nation (MFN)-type price benchmarking in the U.S., policymakers and analysts are increasingly debating what could come next to further regulate pharmaceutical spending. One emerging hypothesis is that cost-effectiveness analysis (CEA) may become a complementary or alternative policy tool. While the U.S. has historically resisted formal use of cost-utility metrics, the combination of rising specialty-drug spending, cross-market price interactions, and the limitations of MFN rules has reignited interest in more structured value assessment frameworks.
METHODS: A targeted review of U.S. policy proposals, Congressional reports, and payer-driven value assessment activities (e.g., state-level affordability boards, employer coalitions, payer consortiums) was conducted. Selected European HTA systems were analyzed to identify transferable policy instruments. Thematic analysis evaluated feasibility barriers in the U.S. context across legal, methodological, political, and institutional domains.
RESULTS: The potential adoption of CEA in the U.S. faces four major challenges:(1) absence of a federal authority responsible for economic evaluation, and restrictions on QALY use in federal programs;(2) heterogeneous payer landscape, limiting standardized implementation;(3) methodological and ethical concerns; and(4) strong political resistance to any mechanism perceived as constraining patient access or pricing autonomy.At the same time, multiple catalysts have emerged: cost-growth pressures on Medicare and commercial plans, diffusion of value-assessment tools by private HTA entities, and the recognition that MFN pricing alone may not fully address structural inefficiencies.
CONCLUSIONS: While a nationwide CEA mandate remains unlikely in the short term, targeted pathways may emerge: voluntary CEA-informed contracting, state-level affordability reviews, or indication-based pricing pilots. As MFN mechanisms expose system-wide inconsistencies, structured value assessment could serve as a next-step policy lever—if paired with safeguards around equity, transparency, and methodological standardization. Early stakeholder engagement and incremental pilot programs may provide a politically feasible route toward broader integration of CEA principles in U.S. pharmaceutical pricing.