Table 8.
Model uncertainty and validation: peer-reviewed articles (19 articles)
| Study (year) | Sensitivity analyses reported | Face validity | Verification of internal validity | Cross validity | External validity | Predictive validity |
|---|---|---|---|---|---|---|
| Aceituno et al. (2018) [20] | PSA, DSA | NR | NR | NR | All model inputs validated by two hematologists with extensive experience in the treatment of multiple myeloma in Chile | NR |
| Borg et al. (2016) [54] | PSA, DSA, scenarios | NR | NR | NR | Validated through the submission and the reimbursement application for pomalidomide in Sweden | NR |
| Cai et al. (2019) [27] | PSA, DSA | NR | NR | NR | NR | NR |
| Campioni et al. (2020) [28] | PSA, DSA, scenarios | NR | NR | NR | In a recent retrospective analysis of long-term PFS and OS data of Rd patients in the RMG registry, the median PFS and OS values are very closely in line with the predictions of our model | NR |
| Carlson et al. (2018) [29] | PSA, DSA, scenarios | For curve fitting and drug cost estimates (compared against an analysis performed by Potluri et al. 2015) | NR | NR | NR | NR |
| Dolph et al. (2021) [30] | PSA, DSA, scenarios | Clinical and health economic expert guidance throughout the development of the model | Quality-control validation using a cost-effectiveness model verification checklist | Cross-validation performed via an assessment of observed PFS and OS outcomes, against which model outputs were compared | Systematic examination of calculations and VBA coding accuracy by external reviewers; results and model approaches compared with previously published relevant economic analyses; validation of external data sources | NR |
| Gueneau et al. (2018) [55] | NR | NR | NR | NR | NR | NR |
| Jakubowiak et al. (2016) [31] | PSA, DSA, scenarios | NR | NR | NR | Comparing the estimated total survival for Rd based on the K-GEM to that estimated in a life-time CE model from the UK NHS perspective | NR |
| Jakubowiak et al. (2017) [32] | PSA, DSA, scenarios | NR | Using data from a well-conducted phase III randomized trial that showed superiority of Kd56 over Vd in PFS and OS and the estimation of long-term survival for the Vd arm from matched registry patients | NR | Using SEER registry data to validate results | NR |
| Kapinos et al. (2023) [33] | PSA, DSA | NR | NR | NR | NR | NR |
| Karampampa et al. (2023) [56] | PSA, DSA, scenarios | NR | NR | NR | Validated for long-term estimates of OS and PFS with standard parametric models by clinical experts | NR |
| Matsela et al. (2022) [21] | DSA, scenarios | NR | NR | NR | NR | NR |
| Nikolaou et al. (2021) [57] | PSA, DSA, scenarios | Conceptual validity through critical literature review and validated by two key opinion leaders | The model was validated according to the AdViSHE checklist by an independent modeling expert | NR | NR | NR |
| Patel et al. (2021) [34] | PSA, DSA, scenarios | NR | NR | NR | NR | NR |
| Pelligra et al. (2017) [68] | PSA, DSA, scenarios | The results of the model were compared with what was expected for each input | Model validation and verification was undertaken using the AdViSHE checklist. The conceptual model was reviewed by health economists and clinicians at Celgene | NR | Validation of the results by comparing the health outcomes for Pd to a recent CE study published in a similar population and comparing the cost results to a study of patients with RRMM in the USA | NR |
| Wong et al. (2021) [35] | PSA, DSA, scenarios | NR | NR | NR | NR | Validity of the chosen distributions by comparing the extrapolated outcomes with the updated survival analysis based on the latest follow-up data and comparing the extrapolated hazard estimates with the observed hazard estimates based on the Kaplan–Meier curves |
| Zeng et al. (2020) [36] | PSA, DSA | The Weibull survival model that was selected matched the PFS curves of the trial satisfactorily. In this evaluation, the uncertainty of PFS estimation was verified adequately by the sensitivity analyses | NR | NR | NR | NR |
| Zhang et al. (2018) [37] | PSA, DSA, scenarios | NR | NR | NR | NR | The estimated rate of PFS at 1 or 2 years according to the model were compared to the clinical results of the RCTs |
AdViSHE Assessment of the Validation Status of Health-Economic Decision Models, CE cost-effectiveness, DSA deterministic sensitivity analysis, K-GEM Kyprolis Global Economic Model, NHS National Health Service, NR not reported, OS overall survival, Pd pomalidomide plus dexamethasone, PFS progression-free survival, PSA probabilistic sensitivity analysis, RCT randomized controlled trial, Rd lenalidomide and dexamethasone, RMG Registry of Monoclonal Gammopathies, SEER Surveillance, Epidemiology, and End Result, VBA Visual Basic for Applications, Vd bortezomib and dexamethasone