Table 9.
Model uncertainty and validation: health technology assessment submissions (31 submissions)
| Study | Sensitivity analyses reported | Face validity | Verification of internal validity | Cross-validity | External validity | Predictive validity |
|---|---|---|---|---|---|---|
| CADTH PC0164-000 (2019) [38] | NR | NR | NR | NR | NR | NR |
| CADTH PC0165-000 (2019) [39] | Scenarios | NR | NR | NR | NR | NR |
| CADTH PC0220-000 (2021) [58] | DSA | NR | NR | NR | NR | NR |
| CADTH PC0256-000 (2022) [41] | DSA, PSA, scenarios | NR | NR | NR | NR | NR |
| CADTH PC0276-000 (2022) [40] | Scenarios | NR | NR | NR | NR | NR |
| Institute for Clinical and Economic Review (ide-cel, cilta-cel, belamaf assessment) (2021) [67] | DSA, PSA, scenarios | Model input parameters were varied to evaluate face validity of changes in results | Validation of the model by independent modelers | NR | Literature review findings | NR |
| NICE TA171 (2019) [42] | NR | NR | NR | NR | NR | NR |
| NICE TA573 (2019) [22] | DSA, PSA, scenarios | NR | Validation of the model structure, calculations, and inputs | NR | Multiple advisory boards | NR |
| NICE TA586 (2019) [59] | DSA, PSA, scenarios | NR | NR | NR | NR | NR |
| NICE TA657 (2020) [43] | DSA, PSA, scenarios | NR | NR | NR | Feedback from clinical experts | NR |
| NICE TA658 (2020) [60] | DSA, PSA, scenarios | NR | Validation by an independent group | NR | Feedback from clinical and economic modeling experts | NR |
| NICE TA695 (2021) [44] | DSA, PSA, scenarios | Validation against trial and literature data | Internal validation | NR | Advisory board | NR |
| NICE TA783 (2022) [61] | DSA, PSA, scenarios | NR | NR | NR | NR | NR |
| NICE TA870 (2023) [62] | DSA, PSA, scenarios | Validation against trial and RWE data | NR | NR | Advisory board | NR |
| PBAC 5.02 July 2021, Darzalex [23] | NR | NR | NR | NR | NR | NR |
| PBAC 5.04 November 2020, Empliciti [63] | Scenarios | NR | NR | NR | NR | NR |
|
PBAC 5.07 July 2021, Xpovio [48] Resubmission: PBAC 7.07 November 2022, Xpovio [98] Resubmission: PBAC 7.10 March 2022, Xpovio [99] |
Scenarios | NR | NR | NR | NR | NR |
| PBAC 5.07 November 2020, Ninlaro [49] | Scenarios | NR | NR | NR | NR | NR |
|
PBAC 5.08 July 2021, Xpovio [64] Resubmission: PBAC 7.09 March 2022, Xpovio [100] |
Scenarios | NR | NR | NR | NR | NR |
|
PBAC 5.09 July 2019, Aplidin [65] Resubmission: PBAC 7.06 March 2020, Aplidin [101] |
DSA, scenarios | NR | NR | NR | NR | NR |
|
PBAC 6.08 July 2019, Pomalyst [24] Resubmission: PBAC 7.13 November 2019, Pomalyst [73] |
NR | NR | NR | NR | NR | NR |
| PBAC 6.08 July 2020, Kyprolis [50] | NR | NR | NR | NR | NR | NR |
| PBAC 7.01 November 2019, Darzalex [25] | Scenarios | NR | NR | NR | NR | NR |
| PBAC 7.03 March 2019, Darzalex [51] | Scenarios | NR | NR | NR | NR | NR |
| PBAC 7.03 March 2022, Kyprolis [52] | Scenarios | NR | NR | NR | NR | NR |
| PBAC 7.05 July 2021, Emplicit [53] | NR | NR | NR | NR | NR | NR |
| PBAC 7.06 March 2022, Ninlaro [66] | Scenarios | NR | NR | NR | NR | NR |
| PBAC 7.11 July 2020, Darzalex [26] | Scenarios | NR | NR | NR | NR | NR |
| SMC 2180 (2019) [45] | NR | NR | NR | NR | NR | NR |
| SMC 2290 (2020) [46] | DSA, scenarios | NR | NR | NR | NR | NR |
| SMC 2303 (2021) [47] | DSA, scenarios | NR | NR | NR | NR | NR |
belamaf belantamab-mafodotin, CADTH Canadian Agency for Drugs and Technologies in Health, cilta-cel ciltacabtagene autoleucel, DSA deterministic sensitivity analysis, ide-cel idecabtagene vicleucel, NICE National Institute for Health and Care Excellence, NR not reported, PBAC Pharmaceutical Benefits Advisory Committee, PSA probabilistic sensitivity analysis, RWE real-world evidence, SMC Scottish Medicines Consortium, TA technology appraisal