OBJECTIVES : Our research aims to understand payer acceptance of value attributes of therapies beyond clinical outcomes and determine their impact on pricing and access outcomes in the EU5.

METHODS : Three recently launched products (ixazomib, ravulizumab, and onasemnogene abeparvovec) were selected as case studies. HTA reports were analyzed to understand how key attributes, including oral route of administration vs. infusion, decreased frequency of administration vs. competitors, and a decrease in need for continued care, was perceived by payers and contributed to the overall HTA outcomes.

RESULTS : Attributes of a medicine beyond clinical efficacy and safety can provide value by addressing unmet needs for patients and the healthcare system, such as improved convenience or decreased healthcare resource utilization. However, challenges remain in demonstrating the value of these improvement to HTA bodies across countries.

In GBR and FRA, potential quality of life gains and cost savings from a less frequent dosing schedule vs. other alternative treatments and patient preference for the fewer infusions were noted as value drivers in assessments.

On the other hand, assessments in DEU, ITA, and ESP largely focused on clinical criteria and the direct budget impact of each drug to inform decision-making. Payers in these countries did not see the value in potential cost-savings from reduced resource use or the benefit to patients who preferred the less frequent dosing or less intensive administration of the selected therapies.

CONCLUSIONS : Challenges in quantifying and formally demonstrating value drivers beyond clinical outcomes negatively impact the perception of these attributes and decrease their contribution to overall outcomes. While outcomes impacting, economic outcomes can be incorporated within modelling and leveraged in some markets, more qualitative outcomes (e.g., patient preferences) are not likely to be accepted by payers. Development of standardized metrics to better outline and communicate these attributes can increase their acceptance across the EU markets.