Linking Reimbursement to Patient Benefits for Advanced Therapy Medicinal Products and Other High-Cost Innovations: Policy Recommendations for Outcomes-Based Agreements in Europe

IF 4.9 2区 医学 Q1 ECONOMICS Value in Health Pub Date : 2024-11-01 DOI:10.1016/j.jval.2024.07.007
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Abstract

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.
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将先进治疗药物产品和其他高成本创新产品的报销与患者利益挂钩:欧洲基于结果的协议的政策建议。
目的:对高成本、一次性细胞和基因疗法等高级治疗药物产品(ATMP)进行卫生技术评估(HTA)尤其具有挑战性。基于结果的协议 (OBA) 是一种潜在的解决方案,既能降低风险,又能为患者提供治疗机会,但在欧洲尚未得到广泛应用。本研究旨在制定政策建议,以支持欧洲接受和实施基于结果的协议:方法:采用政策沙盒的方法与利益相关者接触,探讨 HTA 组织如何支持有关 ATMP 的 OBA 的报销决策。在两次研讨会上,来自欧洲地区的 38 位专家组成了一个专家小组,他们分别代表支付方、HTA 组织、患者、注册机构和一个行业贸易机构:制定了政策建议,以支持在对 ATMP 等高度不确定的技术进行报销时适当考虑 OBA。如果无法按照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 等复杂的解决方案更可取。如果要采用 OBA,则应与所有利益相关者合作设计,以了解数据收集的可行性,并尽量减轻患者和医疗服务提供者的负担。鼓励支付方将 OBAs 作为知情决策的工具,包括随时准备根据不利证据做出否定的报销决定:本研究提出了在报销决策中使用 OBA 的政策框架。必须精心设计 OBA,重点关注适宜性和执行负担。相关部门应致力于根据所获得的证据做出决策。
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来源期刊
Value in Health
Value in Health 医学-卫生保健
CiteScore
6.90
自引率
6.70%
发文量
3064
审稿时长
3-8 weeks
期刊介绍: Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
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