Study identification Moodie et al. 2014. Costs of Bronchoalveolar Lavage-Directed Therapy in the First 5 Years of Life for Children with Cystic Fibrosis. The Journal of Pediatrics; 165 (3), pages 564–569 |
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Guidance topic: Cystic Fibrosis | Question no: 9 |
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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 | BAL & standard therapy |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | Australia & New Zealand |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes | Health care provider |
1.5 Are all direct effects on individuals included, and are all other effects included where they are material? | Partly | HRQoL and adverse events not considered |
1.6 Are all future costs and outcomes discounted appropriately? | NA | Time horizon: 5 years |
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 | All outcomes transformed into costs |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Unclear | Other sectors not stated |
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 alongside RCT |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: 5 years |
2.3 Are all important and relevant outcomes included? | Partly | QoL outcomes and adverse events not considered |
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 national databases |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | No | Cost-benefit analysis |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Partly | SDs & 95% CIs reported |
2.11 Is there any potential conflict of interest? | Partly | Tobramycin provided free by the manufacturer |
2.12 Overall assessment: Minor 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? No, all relevant costs included and described |
Study identification Etherington et al. 2008. Clinical impact of reducing routine susceptibility testing in chronic Pseudomonas aeruginosa infections in cystic fibrosis. Journal of Antimicrobial Chemotherapy; 61, pages 425–7. |
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Guidance topic: Cystic Fibrosis | Question no: 7 |
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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? | No | Number of routine susceptibility tests conducted on isolates of pseudomonas aeruginosa, frequency is not a comparison in the protocol |
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: 6 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: Not applicable |
Other comments: Still considered relevant for decision making given that the Committee could make recommendations about the frequency of testing |
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 | Not a costeffectiveness analysis |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | No | Time horizon: 6 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 | No detail regarding cost build up |
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 | Sources not reported |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | No | Report cost savings from new protocol from consumable and staff time. Other clinical outcomes also reported. |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | No | Only point estimates reported |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Very serious limitations |
Other comments: Have all important and relevant costs and outcomes for each alternative been quantified, where appropriate? No, not all relevant costs and outcomes included Were any assumptions of materiality made to restrict the number of consequences considered? Unclear, insufficient detail regarding cost build-up Was an analysis of correlations between consequences carried out to help control for double counting? No Was there any indication of the relative importance of the difference consequence and suggested weighting of them? No Were there any theoretical relationships between consequences that could have been taken into account in determining weights? Final outcomes associated with a cost a QoL weight such as the duration of IV antibiotics Were the consequences considered one by one to see if a decision could be made based on a single consequence or a combination of a small number of consequences? No Were the consequences considered in subgroups of all consequences in the analysis to see if a decision could be made based on a particular subgroup? No Was an MCDA (multiple criteria decision analysis) or other published method of aggregation of consequences attempted? |