Evaluating National Healthcare Service Spending With Progression Free Survival-Based Models Versus Traditional Reimbursement Schemes for Breast and Lung Cancer in Italy

OBJECTIVES: Outcomes-based managed entry agreements (OBMEAs) can be used to mitigate clinical and financial uncertainty around the real-world drugs’ performance. However, their economic implications are not fully understood. This study assesses the scenarios making 12-month progression free survival (PFS)-based payment by result (PbR) schemes a possible convenient option for the Italian National Healthcare Service (NHS) in alternative to traditional reimbursement schemes.

METHODS: The study included phase-3 clinical trials of drugs reimbursed in Italy in the last three years (2021-2023) as first-line treatments for the deadliest solid tumors for women (breast cancer) and men (non-small cell lung cancer), which reported mature PFS data as primary endpoint. First, PFS curves were digitalized to retrieve their 12-month values. Then, NHS spending for each drug’s median treatment duration was estimated under both traditional and OBMEA reimbursement scheme. The former was estimated assuming complete reimbursement for the whole treatment duration. For the latter, probability of reimbursement of the first year of treatment was considered equal to the trial’s 12-month PFS value, while complete reimbursement was assumed for the remainder duration. Lastly, different scenarios of confidential discounts were applied to the traditional scheme to assess its convenience with respect to the OBMEA scheme.

RESULTS: Five clinical trials were selected. The OBMEA scheme was always cost-saving with respect to traditional reimbursement for discount values below 18.0%, while it emerged as cost-saving for at least 2 drugs out of 5 when increasing the discount up to 34.7%. Only for discount values above 61.7%, the traditional scheme was the most convenient for all five cases.

CONCLUSIONS: The analysis showed that 12-month PFS-based PbR schemes may reduce Italian NHS spending compared with traditional reimbursement schemes, suggesting potential opportunities for this type of agreements in the future. Further analyses on different indications may be needed to extend generalizability of results.