Table 6.
Study (year) | Model structure | Health states | Structure justification | Time horizon | Cycle length | Source of clinical inputs | Source of utility and disutility values | Source of HCRU inputs |
---|---|---|---|---|---|---|---|---|
Aceituno et al. (2018) [20] | Markov model |
1. Progression free (on treatment) 2. Progression free (off treatment) 3. Progressive disease 4. Death |
Markov structure was argued to be appropriate when disease conceptualization necessitated a long-term perspective and entails transitions between distinct health states | 25 years | 4 weeks | Clinical trials and retrospective study | Literature | Clinical trials |
Borg et al. (2016) [54] | Simple discrete event simulation model |
3 events 1. Stable disease 2. Progressive disease 3. Death |
NR | Lifetime | NR | Clinical trials | Clinical trials, literature | Literature |
Cai et al. (2019) [27] | Markov model |
1. Progression free 2. Progressive disease 3. Death |
NR | 10 years | NR | NMA | Literature | RWE |
Campioni et al. (2020 [28] | PSM |
1. Progression free 2. Progressive disease 3. Death |
NR | Lifetime (40 years) | 4 weeks | Clinical trials and real-world data | Clinical trials, literature | Literature |
Carlson et al. (201)8 [29] | PSM |
1. Progression free 2. Progressive disease 3. Death |
NR | Lifetime | 1 week | NMA | Literature | NR |
Dolph et al. (2021) [30] | PSM |
1. Progression free (on treatment) 2. Progression free (off treatment) 3. Progressive disease 4. Death |
NR | Lifetime (40 years) | 1 week | Clinical trials, NMA | Clinical trials, literature | Literature |
Gueneau et al. (2018) [55] | NR | NR | NR | NR | NR | Clinical trials | NR | NR |
Jakubowiak et al. (2016) [31] | PSM |
1. Progression free (on treatment) 2. Progression free (off treatment) 3. Progressive disease 4. Subsequent lines 5. Best supportive care 6. Death |
NR | 30 years | 4 weeks | Clinical trials and real-world data | Clinical trials, literature | Literature |
Jakubowiak et al. (2017) [32] | PSM |
1. Progression free (on treatment) 2. Progression free (off treatment) 3. Progressive disease 4. Death |
NR | 30 years | 4 weeks | Clinical trials | Clinical trials, literature | NR |
Kapinos et al. (2023) [33] | Monte Carlo Markov Chain |
For CAR-T: 1. Complete response 2. Minimal response 3. No response 4. Progressive disease For belamaf: 1. Complete response 2. Minimal response 3. Progressive disease Within each state, patients could either die or survive |
NR | Lifetime | Monthly | Clinical trialsa | Literature | NR |
Karampampa et al. (2023) [56] | PSM |
1. Progression free 2. Progressive disease 3. Death |
In line with previous economic evaluations and health technology assessments of treatments for RRMM | Lifetime (15 years) | Monthly | Clinical trials and real-world data | Clinical trials, literature | Clinical expert opinion |
Matsela et al. (2022) [21] | Markov model |
1. Progression free 2. Progressive disease 3. Death |
NR | Lifetime (15 years) | 4 weeks | Clinical trials, NMA | Literature | Literature |
Nikolaou et al. (2021) [57] | PSM |
1. Progression free (on treatment) 2. Progression free (off treatment) 3. Progressive disease 4. Death |
NR | 10 years | 1 week | Clinical trials, MAIC | Clinical trials, literature | Clinical expert opinion |
Patel et al. (2021) [34] | Markov model (treatment sequencing) |
1. Second-line treatment (either SVd or Vd) 2. Third-line treatment 3. Fourth-line treatment 4. Best supportive care 5. Death |
NR | Lifetime | Monthly | Clinical trials | Literature | Literature |
Pelligra et al. (2017) [68] | NR |
1. Progression free 2. Progressive disease 3. Death |
NR | 3 years | 4 weeks | Clinical trials and MAIC | Clinical trial and mixed-effect model | Literature |
Wong et al. (2021) [35] | PSM |
1. Progression free 2. Progressive disease 3. Death |
NR | 10 years | 1 week | Clinical trials | Literature | Clinical trials, literature |
Zeng et al. (2020) [36] | Markov model |
1. Stable disease 2. Progressive disease 3. Death |
NR | Lifetime | 3 weeks | Clinical trials | Literature | NR |
Zhang et al. (2018) [37] | Semi-Markov model |
1. Progression free 2. Progressive disease 3. Death |
NR | 10 years | 4 weeks | Clinical trialsa | Literature | NR |
belamaf belantamab-mafodotin, CAR-T chimeric antigen receptor T-cell, HCRU healthcare resource use, MAIC matching-adjusted indirect comparison, NMA network meta-analysis, NR not reported, PSM partitioned survival model, RRMM relapsed/refractory multiple myeloma, RWE real-world evidence, SVd selinexor plus bortezomib and dexamethasone, Vd bortezomib and dexamethasone
aModels that provided more than one clinical trial as sources for clinical inputs but did not provide any further information regarding how these sources were incorporated in the calculations