The growing number of hemophilia registries: Quantity vs. quality. Issue 5 (3rd April 2015)

• Record Type: Journal Article
• Authors: Keipert, C; Hesse, J; Haschberger, B; Heiden, M; Seitz, R; van den Berg, HM; Hilger, A; on behalf of the ABIRISK Consortium
• Is Part Of: Clinical pharmacology & therapeutics. Volume 97:Issue 5(2015:May)
• Page Start: 492
• Page End: 501
• Publication Date: 2015-04-03
• Subjects: Pharmacology -- Periodicals; Therapeutics -- Periodicals; 615.5
• DOI: 10.1002/cpt.83
• Languages: English
• ISSNs: 0009-9236

Abstract

<abstract abstract-type="main"> <title> <x xml:space="preserve">Abstract</x> </title> <p>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.</p> </abstract>