Study identification Tappenden, P., Harnan, S., Uttley, L., Mildred, M., Walshaw, M., Taylor, C., Brownlee, K., The cost effectiveness of dry powder antibiotics for the treatment of Pseudomonas aeruginosa in patients with cystic fibrosis, Pharmacoeconomics, 32, 159–72, 2014 |
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Guidance topic: Cystic Fibrosis | Question no: 13 |
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Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5) | Yes/partly/no/unclear/NA | Comments |
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1.1 Is the study population appropriate for the review question? | Yes | People with CF |
1.2 Are the interventions appropriate for the review question? | Yes | Coli DPI vs. NT Tobi DPI vs. NT |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | UK |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes | NHS |
1.5 Are all direct effects on individuals included, and are all other effects included where they are material? | Yes | |
1.6 Are all future costs and outcomes discounted appropriately? | Yes | Time horizon: lifetime Discount rate: 3.5% |
1.7 Is QALY used as an outcome, and was it derived using NICE’s preferred methods? If not, describe rationale and outcomes used in line with analytical perspectives taken (item 1.4 above). | Yes | |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Yes | |
1.9 Overall judgement: Applicable |
Other comments:
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Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
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2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | Yes | Treatment switching occurs in clinical practice but no data on this |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: lifetime and within trial analysis |
2.3 Are all important and relevant outcomes included? | Yes | |
2.4 Are the estimates of baseline outcomes from the best available source? | Yes | From RCT |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | From RCT |
2.6 Are all important and relevant costs included? | Yes | Full details on costs provided |
2.7 Are the estimates of resource use from the best available source? | Yes | From RCT |
2.8 Are the unit costs of resources from the best available source? | Yes | From UK recognised sources |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | OWSA and PSA |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: No limitations |
Other comments:
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Study identification Tappenden, P., Harnan, S., Uttley, L., Mildred, M., Carroll, C., Cantrell, A., Colistimethate sodium powder and tobramycin powder for inhalation for the treatment of chronic Pseudomonas aeruginosa lung infection in cystic fibrosis: systematic review and economic model, Health Technology Assessment (Winchester, England), 17, v-xvii, 2013 |
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Guidance topic: Cystic Fibrosis | Question no: 13 |
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Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5) | Yes/partly/no/unclear/NA | Comments |
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1.1 Is the study population appropriate for the review question? | Yes | People with CF |
1.2 Are the interventions appropriate for the review question? | Yes | Coli DPI vs. NT |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | UK |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes | NHS |
1.5 Are all direct effects on individuals included, and are all other effects included where they are material? | Yes | |
1.6 Are all future costs and outcomes discounted appropriately? | Yes | Time horizon: lifetime Discount rate: 3.5% |
1.7 Is QALY used as an outcome, and was it derived using NICE’s preferred methods? If not, describe rationale and outcomes used in line with analytical perspectives taken (item 1.4 above). | Yes | |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Yes | |
1.9 Overall judgement: Directly applicable |
Other comments:
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Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
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2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | Yes | Treatment switching occurs in clinical practice but no data on this |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: lifetime and within trial analysis |
2.3 Are all important and relevant outcomes included? | Yes | |
2.4 Are the estimates of baseline outcomes from the best available source? | Yes | From RCT |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | From RCT |
2.6 Are all important and relevant costs included? | Yes | Full details on costs provided |
2.7 Are the estimates of resource use from the best available source? | Yes | From RCT |
2.8 Are the unit costs of resources from the best available source? | Yes | From UK recognised sources |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | OWSA and PSA |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: No limitations |
Other comments:
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Study identification Iles, R., Legh-Smith, J., Drummond, M., Prevost, A., Vowler, S., Economic evaluation of Tobramycin nebuliser solution in cystic fibrosis, Journal of Cystic Fibrosis, 2, 120–8, 2003 |
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Guidance topic: Cystic Fibrosis | Question no: 13 |
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Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5) | Yes/partly/no/unclear/NA | Comments |
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1.1 Is the study population appropriate for the review question? | Yes | People with CF |
1.2 Are the interventions appropriate for the review question? | Yes | NT |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | UK |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes | NHS |
1.5 Are all direct effects on individuals included, and are all other effects included where they are material? | Partly | HRQoL not considered |
1.6 Are all future costs and outcomes discounted appropriately? | NA | Time horizon: 12 months |
1.7 Is QALY used as an outcome, and was it derived using NICE’s preferred methods? If not, describe rationale and outcomes used in line with analytical perspectives taken (item 1.4 above). | No | Outcome measure: cost savings from reduced resources |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Unclear | |
1.9 Overall judgement: Directly applicable |
Other comments: This study does not include the preferred measure of effects (QALYs), but is still thought to be useful for decision making given that all other criteria are applicable and the alternative outcome measure reported is unlikely to change the conclusions about costeffectiveness. |
Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
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2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | NA | Cost-benefit analysis |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon: 12 months |
2.3 Are all important and relevant outcomes included? | Partly | QoL outcomes not considered |
2.4 Are the estimates of baseline outcomes from the best available source? | Partly | Before and after study |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | Before and after study |
2.6 Are all important and relevant costs included? | Partly | Insufficient detail |
2.7 Are the estimates of resource use from the best available source? | Partly | Before and after study, but resource use not described in detail |
2.8 Are the unit costs of resources from the best available source? | Unclear | Not all sources reported |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | No | |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | No | 95% CIs reported |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Serious limitations |
Other comments: Are money-costs and ‘benefits’ which are savings of future money-costs evaluated? No Have all important and relevant costs and outcomes for each alternative been quantified in money terms? Yes Has at least 1 of net present value, benefit/cost ratio and payback period been estimated? No, only net present value Were any assumptions of materiality made? None implied |
Study identification Schechter, M. S., Trueman, D., Farquharson, R., Higuchi, K., Daines, C. L., Inhaled Aztreonam Lysine versus Inhaled Tobramycin in Cystic Fibrosis. An Economic Evaluation, Annals of the American Thoracic Society, 12, 1030–8, 2015 |
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Guidance topic: Cystic Fibrosis | Question no: 13 |
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Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5) | Yes/partly/no/unclear/NA | Comments |
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1.1 Is the study population appropriate for the review question? | Yes | People with CF |
1.2 Are the interventions appropriate for the review question? | Yes | Aztreonam vs. NT |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | No | US |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes and no | Stated but inappropriate – US third party payer |
1.5 Are all direct effects on individuals included, and are all other effects included where they are material? | Yes | |
1.6 Are all future costs and outcomes discounted appropriately? | Yes | Time horizon: lifetime Discount rate: 3.0% |
1.7 Is QALY used as an outcome, and was it derived using NICE’s preferred methods? If not, describe rationale and outcomes used in line with analytical perspectives taken (item 1.4 above). | Yes | |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Unclear | Cost sources not described |
1.9 Overall judgement: Partially applicable |
Other comments:
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Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
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2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | Yes | |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: 3 years |
2.3 Are all important and relevant outcomes included? | Yes | |
2.4 Are the estimates of baseline outcomes from the best available source? | Yes | From RCT with an open label extension |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | But note estimates of effectiveness not reproducible |
2.6 Are all important and relevant costs included? | Unclear | Insufficient detail |
2.7 Are the estimates of resource use from the best available source? | Unclear | Insufficient detail |
2.8 Are the unit costs of resources from the best available source? | Unclear | Insufficient detail and US based sources |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | Scenario analysis, univariate and PSA |
2.11 Is there any potential conflict of interest? | Partly | Supported by Gilead Sciences |
2.12 Overall assessment: Serious limitations |
Other comments:
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