Study identification Wolter, J. M., Bowler, S. D., Nolan, P. J., McCormack, J. G., Home intravenous therapy in cystic fibrosis: a prospective randomized trial examining clinical, quality of life and cost aspects, European Respiratory Journal, 10, 896–900, 1997 |
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Guidance topic: Cystic Fibrosis | Question no: 16 |
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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 | Home IV vs. hospital IV |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | Australia |
1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Partly | Includes costs borne by participants (societal perspective) |
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? | 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 | Cost-consequence analysis, but quality of life assessed |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Unclear | |
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? | NA | Cost-consequence analysis alongside RCT |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Unclear | Time horizon not defined, 1 course of IV inferred |
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 small RCT |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | From small 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 small 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 | Could be calculated from data reported |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Partly | SDs reported but sensitivity analysis not performed |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Serious limitations |
Other comments: Have all important and relevant costs and outcomes for each alternative been quantified, where appropriate? Yes Were any assumptions of materiality made to restrict the number of consequences considered? None reported 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 number of hospital admissions 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? No |
Study identification Thornton, J., Elliott, R. A., Tully, M. P., Dodd, M., Webb, A. K., Clinical and economic choices in the treatment of respiratory infections in cystic fibrosis: comparing hospital and home care, Journal of Cystic Fibrosis, 4, 239–47, 2005 |
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Guidance topic: Cystic Fibrosis | Question no: 16 |
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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 | Home IV vs. hospital IV, but not exclusive |
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: 1 year |
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 | ICER reported. Outcome measure: FEV1 |
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 cost-effectiveness. |
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 | |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: 1 year |
2.3 Are all important and relevant outcomes included? | Yes | QoL outcomes not considered |
2.4 Are the estimates of baseline outcomes from the best available source? | Partly | From retrospective observational study |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | From retrospective observational study |
2.6 Are all important and relevant costs included? | Yes | Details provided |
2.7 Are the estimates of resource use from the best available source? | Yes | From retrospective observational study |
2.8 Are the unit costs of resources from the best available source? | Yes | Recognised UK databases |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | Difference in FEV1 / difference in cost |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | 95% Cis reported and probabilistic analysis performed |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Minor limitations |
Other comments:
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Study identification Elliott, R. A., Thornton, J., Webb, A. K., Dodd, M., Tully, M. P., Comparing costs of home- versus hospital-based treatment of infections in adults in a specialist cystic fibrosis center, International Journal of Technology Assessment in Health Care, 21, 506–10, 2005 |
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Guidance topic: Cystic Fibrosis | Question no: 16 |
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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 | Home IV vs. hospital IV, but not exclusive |
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: 1 year |
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 |
1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Unclear | |
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? | NA | |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: 1 year |
2.3 Are all important and relevant outcomes included? | Yes | QoL outcomes not considered |
2.4 Are the estimates of baseline outcomes from the best available source? | Partly | From retrospective observational study |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | From retrospective observational study |
2.6 Are all important and relevant costs included? | Yes | Details provided |
2.7 Are the estimates of resource use from the best available source? | Yes | From retrospective observational study |
2.8 Are the unit costs of resources from the best available source? | Yes | Recognised UK databases |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | No | Could be calculated from data reported |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Partly | 95% CIs reported |
2.11 Is there any potential conflict of interest? | No | |
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? None implied |