Patient-centred value framework for haemophilia
Patient-centred value framework for haemophilia
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DOI：
10.1111/hae.13456
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发表时间：
2018-11-01
期刊：
影响因子：
3.9
通讯作者：
Goodman, C.
中科院分区：
文献类型：
--
作者：
O'Mahony, B.;Dolan, G.;Goodman, C.
不断增长的预算需求导致对罕见病医疗支出的审查增加，导致血友病社区内统一的目标，客观地定义以患者为中心的血友病护理价值。开发一个以患者为中心的结果框架，具有全球适用性，用于评估血友病医疗保健的价值。方法结果召集了一个国际多学科专家小组，以确定血友病医疗保健对患者的影响范围，并根据波特的评估价值模型将这些组织成一个三层，以患者为中心的结果框架。除了其他慢性病常见的措施（如生存和生活质量）外，第1层，即达到或保持的健康状况，包括血友病特有的出血频率、肌肉骨骼并发症和危及生命的出血，以及功能或活动的措施。第2层，康复过程，包括初始治疗时间、康复时间和错过的教育/工作时间等结果；还包括通过抑制剂发展、病原体传播/感染、骨科干预和静脉通路困难来衡量的护理的负效用。第三层，健康的可持续性，是通过避免流血、长期维持有生产力的生活和良好的健康来衡量的；潜在的长期负面后果包括治疗不足或不适当以及与年龄相关的并发症。结果框架对不同类型血友病医疗干预措施的适用性进行了描述。结论血友病医疗保健可影响不同患者类型和医疗保健系统的多种以患者为中心的结果。该框架将这些结果组织起来，为多个利益相关者的基于价值的决策提供信息，并为进一步细化和开发标准化的结果集提供基础。
Introduction Aim Growing budgetary demands have led to increased scrutiny of healthcare spending for rare diseases, leading to a unified goal within the haemophilia community to define objectively patient-centred value in haemophilia care. To develop a patient-centred outcomes framework with global applicability for assessing value in haemophilia healthcare. Methods Results An international, multidisciplinary panel of experts convened to identify the range of patient impacts of haemophilia health care and organize these into a three-tiered, patient-centred outcomes framework based on Porter's model for assessing value. In addition to measures common to other chronic diseases (eg survival and quality of life), Tier 1, health status achieved or retained, includes haemophilia-specific outcomes of bleeding frequency, musculoskeletal complications and life-threatening bleeds, as well as measures of function or activity. Tier 2, process of recovery, includes such outcomes as time to initial treatment, time to recovery and time missed at education/work; also included are disutility of care, measured by inhibitor development, pathogen transmission/infections, orthopaedic intervention and difficult venous access. Tier 3, sustainability of health, is measured by bleed avoidance, maintenance of productive lives and good health over time; potential long-term negative consequences include insufficient or inappropriate therapy and age-related complications. The applicability of the outcomes framework for different types of haemophilia healthcare interventions is described. Conclusion Haemophilia health care can affect multiple patient-centred outcomes across diverse patient types and healthcare systems. This framework organizes those outcomes for informing value-based decision making by multiple stakeholders and provides the basis for further refinement and development of a standardized outcomes set.