Table 2.
Overview of inclusion of carers' health-related quality of life (HRQL)
| Carers: | CADTH | HAS | ICER | NICE | NCPE | PBAC | Pharmac | SMC | TLV | ZiN | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Elosulfase alfa | Includeda | Scenario analysis [30] | No published assessment | No published assessment | Base case[31] | Not mentioned [32] | Not mentioned [33] | No published assessment | Not mentioned [34] | Scenario analysis [35] | No published assessment |
| Methodb | Disutilities | Disutilities | Disutilities | ||||||||
| Numberc | NR | 1 | 1 | ||||||||
| Nusinersen | Includeda | Not mentioned [36] | Not mentioned [37] | Thinking presented, excluded in analysis [38] | Base case[39] [40] [41] | No published assessment | Not mentioned [42] | Not mentioned [43] | Scenario analysis [44] | Scenario analysis [45] | Scenario analysis [46] |
| Methodb | Disutilities | Disutilities | NR | Utilities | Utilities | ||||||
| Numberc | 2 |
Early onset: 3 Late onset: 2 (3 in worst) |
NR | 1 | 1 | ||||||
| Ocrelizumab | Includeda | Scenario analysis [47] | No published assessment | Not mentioned [48] | Base case [49] [50] [51] | Not mentioned [52] | Not mentioned [53] | No published assessment | Not mentioned [54] | Scenario analysis [55] | No published assessment |
| Methodb | Disutilities | Disutilities | NR | ||||||||
| Numberc | NR | 1 | NR | ||||||||
| Patisiran | Includeda | Excluded [56] | No published assessment | Scenario analysis [57] | Base case [58] [59, 60] | Not mentioned [61] | No published assessment | No published assessment | Not mentioned[62] | Scenario analysis [63] | No published assessment |
| Methodb | Disutilities | Disutilities | Disutilities | ||||||||
| Numberc | NR |
Stage 1-2: 1 Stage 3: 2 |
1 | ||||||||
| Voretigene neparvovec | Includeda | Scenario analysis [64] | No published assessment | Not mentioned [65] | Base case [66] [67] [68] | Not mentioned [69] | No published assessment | No published assessment | Base case [70] | Scenario analysis [71] | Scenario analysis [72] |
| Methodb | NR | Disutilities | NR | Disutilities | Disutilities | ||||||
| Numberc | NR |
Company: children: 1, adults: 0.5 ERG: children 1.78, adults: 0 |
NR | NR |
Children: 1 Adults: 0.5 |
CADTH Canadian Agency for Drugs and Technologies in Health, ERG evidence review group, HAS: Haute Autorité de Santé, ICER Institute for Clinical and Economic Reviews, NCPE National Centre for PharmacoEconomics, NICE National Institute for Health and Care Excellence, NR not reported, PBAC Pharmaceutical Benefits Advisory Committee, SMC Scottish Medicines Consortium, TLV Tandvårds- och läkemedelsförmånsverket, ZiN Zorginstituut Nederlands
aRefers to whether carers’ HRQL was included in the model. Base case/scenario analysis/excluded refers to HTA body’s final analysis and not company submission
bRefers to the method used for including carers’ HRQL in the model. Please refer to Sect. 4.1 in the text for further information
cRefers to the number of carers whose HRQL was included in the model