Prospects for research in haemophilia with real‐world data—An analysis of German registry and secondary data

Abstract

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.

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

“…Appropriate governance is key to clearly define data ownership, to facilitate data collection, data access, data sharing and data linkage. All these aspects can be difficult to implement due to limited funding and resources available to registries, but also due to restrictions linked to national data protection requirements ( Schopohl et al, 2018 ; McGettigan et al, 2019 ). A clear sustainability plan laying down short and long terms strategies on the development and maintenance of the registries is critical to ensuring their continuous viability, adaptability and suitability to support regulators’ decision making ( European Medicines Agency, 2021b ).…”