PDF(Pubmed)
Abstract：
BACKGROUND: Juvenile idiopathic arthritis (JIA) refers to a heterogeneous group of rheumatic conditions in children. Novel drugs have greatly improved disease outcomes; however, outcomes are impacted by limited awareness of the importance of early diagnosis and adequate treatment, and by differences in access across health systems. As a result, patients with JIA continue to be at risk for short- and long-term morbidity, as well as impacts on virtually all aspects of life of the child and family.
METHODS: Literature on the socioeconomic burden of JIA is largely focused on healthcare costs, and the impact of JIA on patients, families, and communities is not well understood. High quality evidence on the impact of JIA is needed to ensure that patients are receiving necessary support, timely diagnostics, and adequate treatment, and to inform decision making and resource allocation. This commentary introduces the European Joint Programme on Rare Diseases: Producing an Arthritis Value Framework with Economic Evidence: Paving the Way for Rare Childhood Diseases (PAVE) project, which will co-develop a patient-informed value framework to measure the impact of JIA on individuals and on society. With a patient-centered approach, fundamental to PAVE is the involvement of three patient advocacy organizations from Canada, Israel, and Europe, as active research partners co-designing all project phases and ensuring robust patient and family engagement. The framework will build on the findings of projects from six countries: Canada, Germany, Switzerland, Spain, Israel, and Belgium, exploring costs, outcomes (health, well-being), and unmet needs (uveitis, mental health, equity).
CONCLUSIONS: This unique international collaboration will combine evidence on costs (from family to societal), outcomes (clinical, patient and family outcomes), and unmet needs, to co-design and build a framework with patients and families to capture the full impact of JIA. The framework will support the development of high-quality evidence, encompassing economic and clinical considerations, unmet needs, and patient perspectives, to inform equitable resource allocation, health system planning, and quality of care better aligned with the needs of children with JIA, their families, and communities. Knowledge gained from this novel approach may pave the way forward to be applied more broadly to other rare childhood diseases.
METHODS: Literature on the socioeconomic burden of JIA is largely focused on healthcare costs, and the impact of JIA on patients, families, and communities is not well understood. High quality evidence on the impact of JIA is needed to ensure that patients are receiving necessary support, timely diagnostics, and adequate treatment, and to inform decision making and resource allocation. This commentary introduces the European Joint Programme on Rare Diseases: Producing an Arthritis Value Framework with Economic Evidence: Paving the Way for Rare Childhood Diseases (PAVE) project, which will co-develop a patient-informed value framework to measure the impact of JIA on individuals and on society. With a patient-centered approach, fundamental to PAVE is the involvement of three patient advocacy organizations from Canada, Israel, and Europe, as active research partners co-designing all project phases and ensuring robust patient and family engagement. The framework will build on the findings of projects from six countries: Canada, Germany, Switzerland, Spain, Israel, and Belgium, exploring costs, outcomes (health, well-being), and unmet needs (uveitis, mental health, equity).
CONCLUSIONS: This unique international collaboration will combine evidence on costs (from family to societal), outcomes (clinical, patient and family outcomes), and unmet needs, to co-design and build a framework with patients and families to capture the full impact of JIA. The framework will support the development of high-quality evidence, encompassing economic and clinical considerations, unmet needs, and patient perspectives, to inform equitable resource allocation, health system planning, and quality of care better aligned with the needs of children with JIA, their families, and communities. Knowledge gained from this novel approach may pave the way forward to be applied more broadly to other rare childhood diseases.
摘要：
背景：幼年特发性关节炎（JIA）是指一组异质性的儿童风湿性疾病。新型药物极大地改善了疾病的预后；然而，结果受到对早期诊断和适当治疗重要性的认识有限的影响，以及不同卫生系统的准入差异。因此,JIA患者仍然有短期和长期发病的风险，以及对儿童和家庭生活的几乎所有方面的影响。
方法：关于JIA的社会经济负担的文献主要集中在医疗保健成本上，以及JIA对患者的影响，家庭,和社区不是很好的理解。需要有关JIA影响的高质量证据，以确保患者获得必要的支持，及时诊断,和适当的治疗，并为决策和资源分配提供信息。本评论介绍了欧洲罕见疾病联合计划：用经济证据制定关节炎价值框架：为罕见儿童疾病铺平道路（PAVE）项目，这将共同开发一个病人知情的价值框架，以衡量JIA对个人和社会的影响。以患者为中心的方法，PAVE的基础是来自加拿大的三个患者倡导组织的参与，以色列,和欧洲,作为积极的研究合作伙伴，共同设计所有项目阶段，并确保强大的患者和家庭参与。该框架将以六个国家的项目成果为基础：加拿大，德国,瑞士,西班牙,以色列,比利时,探索成本，结果(健康，福祉)，和未满足的需求(葡萄膜炎，心理健康,股本)。
结论：这种独特的国际合作将结合成本证据（从家庭到社会），结果(临床，患者和家庭结果)，和未满足的需求，与患者和家属共同设计和建立一个框架，以捕捉JIA的全部影响。该框架将支持高质量证据的发展，包括经济和临床考虑，未满足的需求，和病人的观点，为公平的资源分配提供信息，卫生系统规划，护理质量更好地符合JIA儿童的需求，他们的家人,和社区。从这种新方法中获得的知识可能会为更广泛地应用于其他罕见的儿童疾病铺平道路。
方法：关于JIA的社会经济负担的文献主要集中在医疗保健成本上，以及JIA对患者的影响，家庭,和社区不是很好的理解。需要有关JIA影响的高质量证据，以确保患者获得必要的支持，及时诊断,和适当的治疗，并为决策和资源分配提供信息。本评论介绍了欧洲罕见疾病联合计划：用经济证据制定关节炎价值框架：为罕见儿童疾病铺平道路（PAVE）项目，这将共同开发一个病人知情的价值框架，以衡量JIA对个人和社会的影响。以患者为中心的方法，PAVE的基础是来自加拿大的三个患者倡导组织的参与，以色列,和欧洲,作为积极的研究合作伙伴，共同设计所有项目阶段，并确保强大的患者和家庭参与。该框架将以六个国家的项目成果为基础：加拿大，德国,瑞士,西班牙,以色列,比利时,探索成本，结果(健康，福祉)，和未满足的需求(葡萄膜炎，心理健康,股本)。
结论：这种独特的国际合作将结合成本证据（从家庭到社会），结果(临床，患者和家庭结果)，和未满足的需求，与患者和家属共同设计和建立一个框架，以捕捉JIA的全部影响。该框架将支持高质量证据的发展，包括经济和临床考虑，未满足的需求，和病人的观点，为公平的资源分配提供信息，卫生系统规划，护理质量更好地符合JIA儿童的需求，他们的家人,和社区。从这种新方法中获得的知识可能会为更广泛地应用于其他罕见的儿童疾病铺平道路。
