Rare cancers, defined as those with an annual incidence of fewer than six cases per 100,000 individuals, are associated with significant health inequalities. This study aimed to assess the knowledge, attitudes, and perceptions of healthcare providers with expertise in rare cancers regarding the effectiveness of enacted or planned rare cancer policies across Europe. Between 25 March 2023 and 5 March 2024, we conducted an online survey targeting 738 healthcare providers affiliated with the European Reference Networks and the Organization of European Cancer Institutes, yielding 92 complete responses from 28 European countries (response rate: 12.5%). While a significant portion of respondents were unaware of their country’s legal definitions for rare cancers, 67.4% acknowledged that national cancer plans prioritized rare cancers. These plans received the highest ratings for their evidence-based interventions and monitoring efforts. The integration of rare cancer policies into broader oncology frameworks was the preferred policy model. National cancer registries were highly rated for confidentiality and validity but scored the lowest for cost-effectiveness. Government funding was deemed crucial for cancer screening programs. The disease burden and unmet health needs primarily influenced reimbursement decisions in the field of rare cancers. Respondents rated palliative care as more effective in adults with rare cancers compared to in children, particularly regarding symptom management. We confirmed significant variability in rare cancer policy evaluations across Europe, the necessity for a common EU-level definition for rare cancers, and a shift in reimbursement and policy framework models, highlighting the importance of policy integration and enhanced collaboration. However, given the limitations of the study, such as small sample size and possible unstudied confounding factors, we should interpret our findings with caution. A systematic policy review and multistakeholder assessment in the future could complement our results.
罕见癌症，即年发病率低于每10万人6例的癌症，与显著的健康不平等现象相关。本研究旨在评估欧洲罕见癌症专业医疗保健提供者对已实施或计划中的罕见癌症政策有效性的认知、态度和看法。2023年3月25日至2024年3月5日期间，我们针对欧洲参考网络和欧洲癌症研究所组织的738名医疗保健提供者进行了在线调查，共收到来自28个欧洲国家的92份完整回复（回复率：12.5%）。尽管相当一部分受访者不了解本国对罕见癌症的法律定义，但67.4%的受访者承认国家癌症计划将罕见癌症列为优先事项。这些计划在基于证据的干预措施和监测工作方面获得了最高评价。将罕见癌症政策纳入更广泛的肿瘤学框架是首选的政策模式。国家癌症登记处在保密性和有效性方面获得高度评价，但在成本效益方面得分最低。政府资助被认为是癌症筛查计划的关键。疾病负担和未满足的健康需求主要影响罕见癌症领域的报销决策。受访者认为姑息治疗在成人罕见癌症患者中比在儿童中更有效，特别是在症状管理方面。我们证实了欧洲各国在罕见癌症政策评估方面存在显著差异，需要欧盟层面制定统一的罕见癌症定义，以及报销和政策框架模式的转变，凸显了政策整合和加强合作的重要性。然而，鉴于本研究的局限性，如样本量小和可能存在未研究的混杂因素，我们应谨慎解释研究结果。未来的系统性政策审查和多利益相关方评估可以补充我们的研究结果。
An Evaluation of Rare Cancer Policies in Europe: A Survey Among Healthcare Providers
肿瘤(癌症)患者之家