Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.
Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, LMU Munich, Munich, Germany.
Haemophilia. 2018 Jul;24(4):584-594. doi: 10.1111/hae.13443. Epub 2018 Feb 28.
Open questions in haemophilia, such as effectiveness of innovative therapies, clinical and patient-reported outcomes (PROs), epidemiology and cost, await answers. The aim was to identify data attributes required and investigate the availability, appropriateness and accessibility of real-world data (RWD) from German registries and secondary databases to answer the aforementioned questions.
Systematic searches were conducted in BIOSIS, EMBASE and MEDLINE to identify non-commercial secondary healthcare databases and registries of patients with haemophilia (PWH). Inclusion of German patients, type of patients, data elements-stratified by use in epidemiology, safety, outcomes and health economics research-and accessibility were investigated by desk research.
Screening of 676 hits, identification of four registries [national PWH (DHR), national/international paediatric (GEPARD, PEDNET), international safety monitoring (EUHASS)] and seven national secondary databases. Access was limited to participants in three registries and to employees in one secondary database. One registry asks for PROs. Limitations of secondary databases originate from the ICD-coding system (missing: severity of haemophilia, presence of inhibitory antibodies), data protection laws and need to monitor reliability.
Rigorous observational analysis of German haemophilia RWD shows that there is potential to supplement current knowledge and begin to address selected policy goals. To improve the value of existing RWD, the following efforts are proposed: ethical, legal and methodological discussions on data linkage across different sources, formulation of transparent governance rules for data access, redefinition of the ICD-coding, standardized collection of outcome data and implementation of incentives for treatment centres to improve data collection.
血友病领域仍存在一些开放性问题，例如创新疗法的疗效、临床和患者报告结局（PRO）、流行病学和成本等，亟待解答。本研究旨在确定所需的数据属性，并调查德国登记处和二级数据库中的真实世界数据（RWD）的可用性、适宜性和可及性，以回答上述问题。
通过在 BIOSIS、EMBASE 和 MEDLINE 中进行系统检索，确定了非商业性二级医疗保健数据库和血友病患者登记处（PWH）。通过案头研究调查了纳入德国患者、患者类型、数据元素（根据在流行病学、安全性、结局和卫生经济学研究中的用途进行分层）以及可及性。
对 676 个命中记录进行筛选，确定了 4 个登记处[全国性 PWH（DHR）、全国性/国际性儿科（GEPARD、PEDNET）、国际性安全性监测（EUHASS）]和 7 个全国性二级数据库。仅三个登记处的参与者和一个二级数据库的员工可以访问。一个登记处询问 PRO。二级数据库的局限性源于 ICD 编码系统（缺失：血友病严重程度、抑制性抗体的存在）、数据保护法以及对可靠性监测的需求。
对德国血友病 RWD 的严格观察性分析表明，有潜力补充现有知识并开始解决选定的政策目标。为了提高现有 RWD 的价值，建议做出以下努力：在不同来源之间进行数据链接的伦理、法律和方法学讨论、制定透明的数据访问治理规则、重新定义 ICD 编码、标准化结局数据的收集以及激励治疗中心改善数据收集。
