Julian Elaine, Pavlovic Mira, Sola-Morales Oriol, Gianfrate Fabrizio, Toumi Mondher, Bucher Heiner C, Dierks Christian, Greiner Wolfgang, Mol Peter, Bergmann Jean-François, Salmonson Tomas, Hebborn Ansgar, Grande Mathilde, Cardone Antonella, Ruof Jörg
R-Connect Ltd, Basel, Switzerland.
Medicines Development and Training (MDT) Services, Paris, France.
Health Econ Rev. 2022 Nov 5;12(1):54. doi: 10.1186/s13561-022-00402-x.
Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny.
Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority).
Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multi-national stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new endpoints; ii) patient preferences on endpoints; iii) position of HTA and other stakeholders; iv) long-term generation and secondary use of data; v) endpoint challenges in RCTs.
The implementation of a joint European HTA assessment is a unique opportunity for a stronger European Health Union. We identified 19 domains related to the four key areas, processes, uncertainty, comparator choice and endpoint selection that urgently need to be addressed for this regulation to become a success.
欧洲联合卫生技术评估（HTA）面临的主要挑战包括针对选择适当对照物的统一方法、选择与特定疾病患者相关的终点、应对剩余的不确定性以及对相关流程进行透明且一致的管理。我们旨在进一步明确这四个领域内值得进一步研究和审查的相关核心领域。
基于之前进行的问卷调查结果，确定了四个关键领域、流程、不确定性、对照物选择和终点选择。在欧洲准入学院的首届大会上，成立了专门的工作组，为这四个领域中的每一个领域定义核心领域并确定其优先级。每个工作组由约10名参与者组成，代表所有相关利益相关者群体（患者/临床医生/监管机构/卫生技术评估与支付方/学术界/行业）。提前分享了确定工作任务的案例集。每个工作组有两名负责人和一名记录员协助推进该过程。所有排名均采用从1（低优先级）到7（高优先级）的有序李克特量表评分进行。
确定的关键领域包括流程方面：i）应对多个PICO（患者/干预措施/对照物/结局）的（资源）挑战，ii）时间和能力挑战，iii）整合所有相关利益相关者，iv）不同跨国利益相关者之间的冲突与协调，v）与卫生技术开发者的互动；不确定性方面：i）早期和包容性协作，ii）关于随机对照试验可行性的共识以及对不确定性的接受，iii）在弥合证据差距方面的协调，iv）能力差距；对照物选择方面：i）在日益碎片化的治疗格局中选择对照物的标准，ii）PICO中对照物的合理数量，iii）塑造早期建议，使对照物同时满足监管和卫生技术评估需求，iv）间接治疗比较（ITC）的可接受性，v）确保广泛的利益相关者参与对照物选择；终点选择方面：i）接近新的终点，ii）患者对终点的偏好，iii）卫生技术评估和其他利益相关者的立场，iv）数据的长期生成和二次使用，v）随机对照试验中的终点挑战。
实施欧洲联合卫生技术评估是建立更强大的欧洲卫生联盟的独特机遇。我们确定了与流程、不确定性、对照物选择和终点选择这四个关键领域相关的19个领域，要使该法规取得成功，这些领域亟待解决。
