OBJECTIVES : Many high-cost orphan and oncology agents require some form of managed entry agreement (MEA) to help EU payers marry the clinical value with the financial costs of such therapies. In the US, manufacturers launching high-cost therapies with weight-based dosing and/or a need for titration have implemented patient utilization adjustment (PUA) contracts to mitigate the risk of escalating or variable costs associated with individualized dosing. We aim to understand what potential value EU4 payers assign to a PUA contract and how this compares to existing contract structures.

METHODS : We surveyed national payers from FR, DE, IT, and ES to understand the attractiveness and feasibility of managed entry agreements, including PUA, simple discounting, responder-driven outcomes arrangement, and event-based outcomes arrangements. Additionally, we asked about the top benefits and challenges of such agreements. Survey findings were supplemented with phone interviews for a deeper understanding of the decisions.

RESULTS : Of the four contract structures tested for feasibility and acceptability, simple discounts scored highest on average (attractiveness: 4.8/5; feasibility: 5/5) while PUA-style agreements scored the lowest (2.1/5, 2.1/5). The main barrier of a PUA-style agreement was the preference to conduct such tracking at an aggregate level (similar to a budget cap), rather than at an individual level. While alternative outcomes-linked VBAs were viewed more favorably than a PUA, they still trailed simple discounting.

CONCLUSIONS : EU4 payers did not see viability in a PUA-style contract, opting to implement a simple budget cap in the event there is uncertainty around associated cost due to dosing variability. Payers generally prefer simple discounts, which are easily implemented; however, outcome-based contracts can create sustainable opportunities for manufacturers when launching agents that may command premium prices in a future environment where national payers increasingly demand demonstrated clinical and financial value.