title: Cost-of-illness of scleroderma: the case for rare diseases.
abstract: OBJECTIVE
  To determine the societal costs of scleroderma (SSc), a rare chronic connective tissue disease that affects approximately 98,000 Americans. Lack of reliable national databases limit rare disease cost studies, and this study suggests methods of using multiple data sources to assess the costs of rare diseases.
  
  
  METHODS
  Primary and secondary data sources were used to calculate direct and indirect costs of SSc, including discounted lifetime mortality and morbidity costs. A prevalence-based, human capital approach was used. Sensitivity analyses were used to vary parameters that are uncertain, such as prevalence, mortality, and labor costs.
  
  
  RESULTS
  Annual direct and indirect costs of SSc in the United States are $1.5 billion. Morbidity represents the major cost burden, with costs of $819 million (56%) of total costs. The current value of lifetime earnings lost was $179 million (12%) or $300,000 per death. Direct costs were $462 million (32%) or $4,731 per person annually, indicating that costs are spread over the long disease duration.
  
  
  CONCLUSIONS
  This study provides one model for the assessment of rare disease costs. Triangulation of data sources and sensitivity analyses are important for determining the costs of rare diseases. The high cost of SSc, despite its low prevalence, suggests that the burden of rare chronic diseases can be high. The high morbidity costs reflect the young age of onset of the disease as well as the need for treatments to decrease morbidity costs. Local shared databases and national surveys are needed to improve cost estimates of rare diseases.
authors.affiliation: School of Pharmacy, University of California, San Francisco 94143, USA.
authors.forename: L
authors.initials: L
authors.lastname: Wilson
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D017281:Cost of Illness; D003362:Cost-Benefit Analysis; D003365:Costs and Cost Analysis; D004180:Direct Service Costs / Q000706:statistics & numerical data; D006233:Persons with Disabilities / Q000706:statistics & numerical data; D005260:Female; D017048:Health Care Costs / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D009017:Morbidity; D012106:Research / Q000191:economics / Q000706:statistics & numerical data; D012595:Scleroderma, Systemic / Q000191:economics* / Q000453:epidemiology / Q000401:mortality; D012680:Sensitivity and Specificity; D018582:Sick Leave / Q000706:statistics & numerical data; D014478:Unemployment / Q000706:statistics & numerical data; D014481:United States / Q000453:epidemiology