The growing number of hemophilia registries: Quantity vs. quality

Clin Pharmacol Ther. 2015 May;97(5):492-501. doi: 10.1002/cpt.83. Epub 2015 Apr 3.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Access to Information
  • Database Management Systems
  • Databases, Factual* / standards
  • Evidence-Based Medicine / methods*
  • Evidence-Based Medicine / standards
  • Guidelines as Topic
  • Hemophilia A* / diagnosis
  • Hemophilia A* / epidemiology
  • Hemophilia A* / therapy
  • Humans
  • Registries* / standards