Presentation (CTI)

OBJECTIVES: When launching new products into therapeutic areas dominated by off-patent standards of care, companies often face significant hurdles in securing favourable HTA outcomes and pricing and market access decisions. These challenges stem from the high bar set by payers who expect clear added value over existing, low-cost treatments. This study aims to assess the main drivers of success and failure in such settings by examining the types of evidence submitted, the nature of payer feedback, and the resulting HTA and pricing decisions across France, Germany, England, Canada, and Brazil.
METHODS: An analogue analysis was conducted, focusing on non-oncology products approved since 2011 in single indications where the SoC had already genericised. The analysis reviewed HTA and pricing outcomes, and reimbursement status. Products were evaluated based on payer-perceived value, with higher ratings classified as ASMR III or “considerable added benefit” (or better), and lower ratings as ASMR V/VI or “no added benefit.”
RESULTS: Only a few products (e.g., Camzyos and Xenpozyme) achieved positive P&MA outcomes in off-patent spaces, largely due to high unmet need and meaningful improvements in clinical or quality-of-life outcomes. A favourable safety profile was also critical, with concerns often leading to negative assessments. In the US, access was broader compared to ex-US, although prior authorisations were common. Payers generally preferred head-to-head trials, though placebo-controlled studies with accepted endpoints were also considered (except in Germany, where the absence of H2H data was often penalised). Common payer concerns included limited long-term data, short study durations, and questions around generalisability.
CONCLUSIONS: Success in off-patent environments remains rare and hinges on clear differentiation, high unmet need, and robust clinical evidence. Payers expect incremental benefit demonstrated through well-designed trials, with quality-of-life and secondary endpoints viewed as supportive but not sufficient. Trial design limitations remain a major barrier to positive HTA and pricing outcomes.