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Table 3 Characteristics of the cost-of-illness studies included in the review

From: Cost-of-illness studies in rare diseases: a scoping review

Characteristic Number of studies (%)
Prevalence or incidence-based estimation
Prevalence 59 (94%)
Incidence 4 (6%)
Design
Cross-sectional 40 (63%)
Cohort 10 (16%)
Cohort compared with a control cohort 7 (11%)
Mathematical model 5 (8%)
Cost study nested in a clinical trial 1 (2%)
Prospective or retrospective
Retrospective 55 (87%)
Prospective 4 (6%)
Source of patients  
Registries or databases 32 (51%)
Hospital or other centres 24 (38%)
Patients’ organisations 18 (29%)
Source of data
Questionnaires 42 (67%)
Registries or databases 30 (48%)
Other 4 (6%)
Bottom-up or top-down approaches
Bottom-up 59 (94%)
Top-down 0 (0%)
Mixed 4 (6%)
Time horizon
A year 52 (83%)
Lifetime 4 (6%)
Other 11 (17%)
Perspective  
Societal 40 (63%)
Third payer/health system/government 17 (27%)
Patients and families 7 (11%)
Hospital 3 (5%)
Costs included in the analysis
Medical costs 63 (100%)
Non-medical costs 38 (60%)
Lost productivity costs 43 (68%)
Informal care costs 27 (43%)
Method for the estimation of lost productivity costs
Human capital approach 34 (54%)
Friction cost approach 2 (3%)
Other 8 (13%)