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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%)