title: Measuring the quality of haemophilia care across different settings: a set of performance indicators derived from demographics data.
abstract: BACKGROUND
  Haemophilia is a rare disease for which quality of care varies around the world. We propose data-driven indicators as surrogate measures for the provision of haemophilia care across countries and over time.
  
  
  METHODS
  The guiding criteria for selection of possible indicators were ease of calculation and direct applicability to a wide range of countries with basic data collection capacities. General population epidemiological data and haemophilia A population data from the World Federation of Hemophilia (WFH) Annual Global Survey (AGS) for the years 2013 and 2010 in a sample of 10 countries were used for this pilot exercise.
  
  
  RESULTS
  Three indicators were identified: (i) the percentage difference between the observed and the expected haemophilia A incidence, which would be close to null when all of the people with haemophilia A (PWHA) theoretically expected in a country would be known and reported to the AGS; (ii) the percentage of the total number of PWHA with severe disease; and (iii) the ratio of adults to children among PWHA standardized to the ratio of adults to children for males in the general population, which would be close to one if the survival of PWHA is equal to that of the general population. Country-specific values have been calculated for the 10 countries.
  
  
  CONCLUSIONS
  We have identified and evaluated three promising indicators of quality of care in haemophilia. Further evaluation on a wider set of data from the AGS will be needed to confirm their value and further explore their measurement properties.
authors.affiliation: Departments of Clinical Epidemiology and Biostatistics and Medicine, McMaster University, Hamilton, ON, Canada.
authors.forename: A
authors.identifier: http://orcid.org/0000-0002-3331-8766
authors.initials: A
authors.lastname: Iorio
authors.affiliation: Poole College of Management, North Carolina State University, Raleigh, NC, USA.
authors.forename: J S
authors.initials: JS
authors.lastname: Stonebraker
authors.affiliation: World Federation of Hemophilia, Montreal, QC, Canada.
authors.forename: M
authors.initials: M
authors.lastname: Brooker
authors.affiliation: Division of Blood Disorders, Center for Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA.
authors.forename: J M
authors.initials: JM
authors.lastname: Soucie
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003710:Demography; D005260:Female; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D011787:Quality of Health Care; D055815:Young Adult
keywords: haemophilia; health care; incidence; prevalence