Abstract：
OBJECTIVE: 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.
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.
摘要：
目的：先进治疗药物（ATMPs）的卫生技术评估（HTA），例如高成本，一次性细胞和基因疗法，特别具有挑战性。基于结果的协议（OBA）是一种潜在的解决方案，可以减轻风险，同时为患者提供服务，但并未在欧洲广泛使用。这项研究旨在制定政策建议，以支持OBAs在欧洲的可接受性和实施。
方法：使用政策沙盒方法与利益相关者互动，并探讨HTA组织如何支持ATMP关于OBA的报销决策。在两个讲习班上召集了来自欧洲地区的38名专家组成的小组，代表付款人，HTA组织，病人,登记册，和一个工业贸易机构。
结果：制定了政策建议，以支持OBA适当考虑偿还高度不确定的技术，比如ATMP。如果无法以建议的价格提出积极的HTA建议，那么反映不确定性的简单价格折扣比OBA等复杂解决方案更受欢迎。如果追求OBA，应与所有利益相关者合作设计，以了解数据收集的可行性，并最大程度地减少患者和提供者的负担。鼓励付款人将OBA作为明智决策的工具，包括准备根据不利的证据做出否定的报销决定。
结论：该研究提出了在报销决策中使用OBAs的政策框架。OBA必须精心设计，注重适当性和执行负担。有关当局应致力于根据由此产生的证据做出决定。
方法：使用政策沙盒方法与利益相关者互动，并探讨HTA组织如何支持ATMP关于OBA的报销决策。在两个讲习班上召集了来自欧洲地区的38名专家组成的小组，代表付款人，HTA组织，病人,登记册，和一个工业贸易机构。
结果：制定了政策建议，以支持OBA适当考虑偿还高度不确定的技术，比如ATMP。如果无法以建议的价格提出积极的HTA建议，那么反映不确定性的简单价格折扣比OBA等复杂解决方案更受欢迎。如果追求OBA，应与所有利益相关者合作设计，以了解数据收集的可行性，并最大程度地减少患者和提供者的负担。鼓励付款人将OBA作为明智决策的工具，包括准备根据不利的证据做出否定的报销决定。
结论：该研究提出了在报销决策中使用OBAs的政策框架。OBA必须精心设计，注重适当性和执行负担。有关当局应致力于根据由此产生的证据做出决定。
