Table 1.
Model costs (in 2015 Great British pounds) and utility data
| Direct costs | Indirect (productivity) costsc | Total cost | Utilities | ||||
|---|---|---|---|---|---|---|---|
| Medicala | Non-medicalb | Patient | Caregiver | Patient | Caregiver | ||
| Model I (DMDSAT) | |||||||
| Initial scoree | 8340 (830) | 9120 (860) | 14,230 (1540)d | 6360 (740) | 23,870 (1580) | 0.879 (0.037) | 0.862 (0.016) |
| Per lost score (multiplier) | 1.057 (1.005) | 1.04 (1.006) | NA | 1.037 (1.006) | 1.053 (1.003) | 0.905 (1.003) | 0.995 (1.001) |
| Model II (ambulatory status) | |||||||
| Early ambulatory | 10,670 (140) | 9740 (50) | 0 (0) | 7180 (190) | 27,590 (350) | 0.699 (0.036) | 0.858 (0.017) |
| Late ambulatory | 11,190 (100) | 11,420 (50) | 0 (0) | 8340 (150) | 30,950 (260) | 0.607 (0.029) | 0.839 (0.017) |
| Early non-ambulatory | 16,490 (290) | 17,860 (110) | 0 (0) | 12,810 (370) | 47,160 (710) | 0.224 (0.014) | 0.784 (0.021) |
| Late non-ambulatory | 27,590 (340) | 16,810 (90) | 14,230 (1540)d | 11,240 (260) | 66,720 (1600) | 0.146 (0.010) | 0.810 (0.018) |
| Model III (ventilation status) | |||||||
| None | 11,520 (60) | 12,660 (60) | 14,230 (1540)d | 9160 (120) | 34,520 (440) | 0.518 (0.027) | 0.837 (0.014) |
| Night-time | 31,710 (590) | 14,610 (240) | 14,230 (1540)d | 10,490 (420) | 61,490 (2600) | 0.129 (0.017) | 0.775 (0.030) |
| Day- and night-time | 36,390 (840) | 15,500 (190) | 14,230 (1540)d | 12,860 (640) | 83,250 (2210) | 0.051 (0.010) | 0.774 (0.033) |
Data are presented as mean (standard error) (costs roundest to the nearest ten). Source cost estimates were converted from US dollars to Great British pounds using an exchange rate of 0.634 and inflated from 2012 to 2015 values using consumer price data from the Organisation for Economic Co-operation and Development (OECD). Patient utilities (ranging from 0 = dead to 1 = perfect health) were obtained from the Health Utilities Index Questionnaire and caregiver utilities from the EuroQol EQ-5D-3L
DMDSAT Duchenne muscular dystrophy Functional Ability Self-Assessment Tool, NA not applicable
aHospital admissions, emergency care, respite care, visits to physicians and other healthcare practitioners (i.e. nurses, general practitioners, specialist physicians, psychologists, therapists, physiotherapists, occupational therapists, care coordinators/care advisors, dentists, dietitians/nutritionists and speech/language/swallowing therapists), tests and assessments, medications, medical aids, devices and investments, and community services (e.g. home help and personal assistants)
bNon-medical aids, devices, and investments and cost associated with informal care (see Landfeldt et al. [10] for details)
cValued according to the human capital approach at the cost of employment
dMean per-patient annual indirect cost for patients 18 years of age or older. Total cost may not equal the sum of total direct and indirect costs since not all patients in the specified strata accrue indirect costs (because, for example, they are <18 years old, attend university or are employed) and because of rounding
ePatients start the model simulation at a DMDSAT score of 23 (i.e. at full functional ability)