PSY56 REAL-WORLD DATA FOR HAEMOPHILIA: A SYSTEMATIC APPROACH TO IDENTIFY AND ASSESS DATA SOURCES

Abstract

Evidence from real-world data (RWD) sources provides valuable insights into the characteristics of patients, and the safety, effectiveness and use of treatments for haemophilia. However, there is limited information on the specific data captured and the completeness of these sources for haemophilia. Using a systematic approach, we sought to identify and assess RWD sources that include patients with haemophilia. Relevant articles (2012‒2018) containing information on RWD sources for patients with haemophilia were screened following searches of Embase and MEDLINE. Congress and supplementary searches (2016–2018) were undertaken to identify RWD sources that are not referenced in full publications. For each data source, completeness of data was assessed for: patient demographics; diagnosis; treatment and management; laboratory results; patient-reported outcomes (PROs); clinical and safety outcomes; and economic burden. A bespoke assessment tool was used to rate each data source as ‘excellent’ (3 points), ‘good’ (2 points) or ‘fair’ (1 point) for data completeness and to give them an overall data completeness score (maximum of 21 points). Of 1941 articles identified, 834 referring to 95 RWD sources met inclusion criteria. In total, 30 RWD sources (described in 175 articles) were prioritized for assessment; 24 focused on a single country, and 6 were multinational. Excellent/good information was provided for: patient demographics (n=22); diagnosis (n=16); treatment and management (n=15); laboratory results (n=9); PROs (n=5); clinical and safety outcomes (n=8); and economic burden (n=15). Three sources scored well (≥16 points) overall: the Australian Bleeding Disorders Registry; the National Haemophilia Database; and the American Thrombosis and Hemostasis Network. The analysis provides a comprehensive list of useful RWD sources for conducting real-world studies of patients with haemophilia. There are several robust RWD sources providing complete information, but further sources collecting data on laboratory results, PROs, and clinical and safety outcomes are required.