RESUMEN
OBJECTIVES: Health technology assessment (HTA) of advanced therapy medicinal products (ATMPs), such as high-cost and one-time cell and gene therapies, is particularly challenging. Outcomes-based agreements (OBAs) are a potential solution to mitigate the risks while providing access to patients but are not widely used across Europe. This study aimed to develop policy recommendations to support the acceptability and implementation of OBAs in Europe. METHODS: A policy sandbox approach was used to engage with stakeholders and explore how HTA organizations can support reimbursement decisions regarding OBAs for ATMPs. A panel of 38 experts from across the European region was convened in 2 workshops, representing payers, HTA organizations, patients, registries, and an industry trade body. RESULTS: Policy recommendations were developed to support the appropriate consideration of OBAs for reimbursing highly uncertain technologies, such as ATMPs. If a positive HTA recommendation cannot be made at the proposed price, then a simple price discount reflecting the uncertainty is preferred over complex solutions such as OBAs. If an OBA is pursued, it should be designed collaboratively with all stakeholders to understand data collection feasibility and minimize burden to patients and providers. Payers are encouraged to approach OBAs as a tool for informed decision making, including a readiness to make negative reimbursement decisions based on unfavorable evidence. CONCLUSIONS: The study presents a policy framework for using OBAs in reimbursement decisions. OBAs must be carefully designed, focusing on appropriateness and the burden of implementation. The relevant authorities should be committed to making decisions in light of the resulting evidence.