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 | Â |
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 | Â |
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%) |