Table 7.
Relevance of identified HSUVs to HTA body reference cases
| Study | Questions to assess relevance of identified HSUVs to HTA body reference cases | Is the study consistent with HTA bodya reference cases? | ||||||
|---|---|---|---|---|---|---|---|---|
| (A) Instrument selection | (B) Did patients describe the health states? | (C) Were appropriate societal preferences used to value health states? | (D) Was the TTO/SG method used to value health states? | HTA body | Consistent with reference case (Yes [with justification]/no/unclear) | If no/unclear, specify which question | ||
| (A1) Was a generic preference-based instrument used to describe health states? | (A2) Was the selected instrument age appropriate? | |||||||
| Belter et al. [51] | Yes: utilities were derived using the HUI3 | Yes: parents completed HUI3 on behalf of paediatric patients and adult patients self-completed the questionnaire | Partially: a proportion of questionnaires were completed by patients (adults), but the majority were completed by a parent/caregiver | Unclear | Unclear | CADTH | Unclear | C |
| US ICER Group | Unclear | C | ||||||
| NICE | Unclear | C/D | ||||||
| Binz et al. [52] | Yes: utilities were derived using the EQ-5D-5L | Yes: study considered adult patients (aged ≥18 years) only | Yes: utilities were derived directly from patients | Yes: German tariff | No: EQ-VTv2.0 was assumed from a published reference [92] | CADTH | Unclear | C |
| US ICER Group | No | C | ||||||
| NICE | No | C/D | ||||||
| Chambers et al. [53] | Yes: utilities were derived using the EQ-5D-Y | Yes: youth version appropriate for proxy respondents for a paediatric population | Partially: questionnaires were completed by patients or by caregivers on behalf of their child | Yes: Australian tariff | Yes: TTO | CADTH | Unclear | C |
| US ICER Group | No | C | ||||||
| NICE | No | C | ||||||
| Lloyd et al. [46] | Yes: utilities were derived using the EQ-5D-Y | Yes: youth version appropriate for proxy respondents for a paediatric population | No: utilities were derived from clinical experts on behalf of patients | Yes: UK tariff | NR | CADTH | Yes—with justification | – |
| US ICER Group | No | C | ||||||
| NICE | Yes—with justification | – | ||||||
| Lo et al. [56] | No: a DCE was used to estimate disutilities | NA | No: the sample included members of the UK general population | NA | NA | CADTH | No | A1/C |
| US ICER Group | No | A1/C | ||||||
| NICE | No | A1/C/D | ||||||
| Lo et al. [57] | Yes: utilities were derived using the EQ-5D-5L | Yes: appropriate for adult caregivers | No: utilities were derived for caregivers of patients with Type 2 SMA | Yes: UK tariff | Yes: values mapped to EQ-5D-3L TTO value | CADTH | Yes | – |
| US ICER Group | No | C | ||||||
| NICE | Yes | – | ||||||
| López-Bastida et al. [47] | Yes: utilities were derived using the EQ-5D (3L and 5L) | No: youth version more appropriate for paediatric population | No: utilities were derived from caregivers on behalf of patients | Unclear | Yes: TTO | CADTH | Unclear | A2/C |
| US ICER Group | Unclear | A2/C | ||||||
| NICE | Unclear | A2/C | ||||||
| Love et al. [58] | Yes: utilities were derived using the HUI3 | Yes: HUI3 completed by patients aged 13–18 years and by parents of younger children | Yes: utilities were derived from both patients and their parents | Unclear | Unclear | CADTH | Unclear | C |
| US ICER Group | Unclear | C | ||||||
| NICE | Unclear | C | ||||||
| Malone et al. [48] | No: PedsQL data were mapped to the EQ-5D-Y using a published algorithm [36] | Yes: PedsQL appropriate for paediatric population | Yes: pts completed the PedsQL, which was mapped to the EQ-5D | NA | NA | CADTH | No | A1/C |
| US ICER Group | No | A1/C | ||||||
| NICE | No | A1/C/D | ||||||
| McMillan et al. [54] | Yes: utilities were derived using the EQ-5D-5L | Partially: appropriate for self-completing adults; youth version available for children | Partially: surveys were self-completed by adult patients or proxy-completed by caregivers (children or adults needing assistance) | Unclear | Unclear | CADTH | Unclear | A2/C |
| US ICER Group | Unclear | A2/C | ||||||
| NICE | Unclear | A2/C/D | ||||||
| Peña-Longobardo et al. [55] | Yes: utilities were derived using the EQ-5D (3L and 5L) | No: youth version more appropriate for paediatric/adolescent population | Yes: utilities were derived directly from patients and caregivers | Yes: UK tariff was assumed from a published reference [93] | Yes: TTO | CADTH | Yes | – |
| US ICER Group | No | C | ||||||
| NICE | Yes | – | ||||||
| Rowell et al. [59] | Yes: utilities were derived using the EQ-5D-3L | Yes: appropriate for adult caregivers | No: utilities were derived for caregivers of patients with Type 2 SMA | Yes: UK tariff (assumed; refers to crosswalk value set) | Yes: TTO (assumed; refers to crosswalk value set) | CADTH | Yes | – |
| US ICER Group | No | C | ||||||
| NICE | Yes | – | ||||||
| Sampson and Garau. [49] | Yes: utilities were derived using the EQ-5D-3L | No: youth version more appropriate for paediatric population | No: utilities were derived from caregivers on behalf of patients | Unclear | Unclear | CADTH | Unclear | A2/C |
| US ICER Group | Unclear | A2/C | ||||||
| NICE | Unclear | A2/C/D | ||||||
| Thompson et al. [50] | Yes: utilities were derived using the EQ-5D-3L | No: youth version more appropriate for paediatric population | No: utilities were derived from parents on behalf of patients | Unclear | Unclear | CADTH | Unclear | A2/C |
| US ICER Group | Unclear | A2/C | ||||||
| NICE | Unclear | A2/C/D | ||||||
CADTH Canadian Agency for Drugs and Technologies in Health, DCE discrete choice experiment, EQ-VTv2.0 EQ-Valuation Technology Vision 2.0, HSUV health state utility value, HTA health technology assessment, HUI3 Health Utilities Index Mark 3, ICER Institute for Clinical and Economic Review, NA not applicable, NICE National Institute for Health and Care Excellence, NR not reported, PedsQL Pediatric Quality of Life Inventory, SG standard gamble, SMA spinal muscular atrophy, TTO time trade-off
aHTA body reference cases were NICE [34], CADTH [44], and the US ICER Group [45]