The growing number of hemophilia registries: Quantity vs. quality

Abstract

Registries for rare diseases provide a tool for obtaining an overview of the clinical situation and can be used to discover points of improvement and to monitor long-term safety. Registries could also become a powerful tool to provide supporting information for marketing authorization. There is an urgent need for a pan-European or global strategy that supports consistent data. Therefore, transparency in data collection, harmonization of the database structures, and the convergence of scientific approaches are required.

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

“…In most of the evaluated registries, only limited information on outcome is provided, predominantly on comorbidities and arthropathy . The conclusions of the three articles on registries in haemophilia are similar: (i). Registries have a high potential for clinical research.…”

“…Several articles have been published which discuss the available evidence from registries in haemophilia. Evaluations concerned publicly available information on national haemophilia registries in Europe, registries on haemophilia with inhibitors worldwide and the incorporation of outcome assessment in haemophilia registries . For registries investigating inhibitor formation, which is a major safety aspect in haemophilia A, the following recommendations for a minimum set of variables for inhibitor predictors were proposed: FVIII mutation type, mode or treatment prior to inhibitor development, type of concentrate, ethnicity, family history for inhibitor and adverse event and surgery prior to inhibitor development .…”

World bleeding disorders registry: The pilot study

“…Despite the numerous registries that exist for patients with bleeding disorders, most are regional, country or HTC specific with the exception of a few international registries. 2,13,14 They are predominantly found in high-income settings 13,14 where patients have access to high quality of care, and are heterogeneous in terms of patients included, data collected and outcomes measured, 2,14 limiting the ability to merge or link data between registries. Additionally, evidence stemming from registry data is lacking.…”

“…Most higher-income countries already have some form of a patient registry. 2,13 A recent survey of 45 European countries confirmed that 78% of the respondent countries (n = 29/37) reported having a national registry. 13 In contrast, patient disease registries in low-income settings are lacking, 14 with the unfortunate consequence of making patients with bleeding disorders invisible to public countries.…”

Haemophilia registries to complement clinical trial data: a pious hope or an urgent necessity?

“…Once a significant number of patients have been entered, registries are able to minimize selection bias and to collect long-term outcome data. A lack of transparency complicates the discussion on whether existing registries already provide the data that regulators are seeking [15]. Even if the collected parameters are known, the quality of the data is often unknown.…”