Presentation (CTI)

OBJECTIVES: US, “value” has frequently been operationalized through rebate economics and formulary leverage. Market and policy signals increasingly favor evidence-defined value, including comparative effectiveness, patient outcomes, real-world, affordability, and explicit uncertainty management. In parallel, EU-style assessment mechanisms continue to mature (e.g., Joint Clinical Assessments initiated for oncology medicines and ATMPs). This work proposes an operating model to build EU-style value propositions for the US that is evolving beyond rebates as the dominant access tool.
METHODS: We developed a framework synthesizing current affordability/policy pressures and evolving assessment expectations with payer decision needs. The framework translates EU-style value assessment logic into a practical toolkit and lifecycle timing gates spanning early development, pivotal evidence design, launch planning, and post-launch reassessment.
RESULTS: The proposed model reframes “value” from net price achieved via rebates to demonstrated incremental benefit supported by decision-grade evidence. The toolkit includes: (1) a value-target product profile (claims, endpoints, comparators, and target subpopulations); (2) patient-relevant outcome and comparator strategies; (3) a combined economic package (budget impact/affordability plus cost-effectiveness when appropriate) with explicit scenario and threshold analyses; (4) an RWE strategy designed for credibility and reassessment; (5) an uncertainty reduction plan linked to post-launch evidence commitments; and (6) contracting approaches aligned to measurable performance rather than rebate magnitude alone. Optional accelerators include Product Value Testing (PVT) to validate minimum evidence thresholds and value story clarity with decision-makers, and Integrated Evidence Generation Planning (IEGP) to govern cross-functional evidence delivery across the lifecycle. The framework provides timing gates to prevent late-cycle evidence gaps and to align launch strategies to EU-style assessment expectations.
CONCLUSIONS: As the definition of value shifts from rebates toward EU-style evidence tools, manufacturers require a model that builds credible, ready value propositions. A structured toolkit—governed through IEGP and selectively validated through PVT—can reduce access uncertainty and enable sustainable pricing and coverage strategies in a post-rebate paradigm.