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Schopohl, D.* ; Bidlingmaier, C.* ; Herzig, D.* ; Klamroth, R.* ; Kurnik, K.* ; Rublee, D.* ; Schramm, W.* ; Schwarzkopf, L. ; Berger, K.*

Prospects for research in haemophilia with real-world dataAn analysis of German registry and secondary data.

Haemophilia 24, 584-594 (2018)
DOI PMC
Open Access Green as soon as Postprint is submitted to ZB.
Introduction and aimOpen 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.MethodsSystematic 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 elementsstratified by use in epidemiology, safety, outcomes and health economics researchand accessibility were investigated by desk research.ResultsScreening 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.ConclusionRigorous 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.
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Publication type Article: Journal article
Document type Scientific Article
Keywords German ; Haemophilia ; Open Questions ; Real-world Data ; Registry ; Secondary Database; Claims Data; Task-force; Insurance; Therapy; Quality
ISSN (print) / ISBN 1351-8216
e-ISSN 1365-2516
Journal Haemophilia
Quellenangaben Volume: 24, Issue: 4, Pages: 584-594 Article Number: , Supplement: ,
Publisher Wiley
Publishing Place 111 River St, Hoboken 07030-5774, Nj Usa
Reviewing status Peer reviewed
Institute(s) Institute of Health Economics and Health Care Management (IGM)