Study identification Suri R, Grieve R, Normand C, Metcalfe C, Thompson S, Wallis C, Bush A. “Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis.” Thorax. 2002: Oct;57(10):841–6 |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase & HS |
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 and adverse events not considered |
1.6 Are all future costs and outcomes discounted appropriately? | NA | Time horizon: 12 weeks |
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 | Outcomes associated with a resource use transformed into costs |
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 benefit analysis alongside crossover trial |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon: 12 week crossover trial |
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 randomised crossover trial |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | From randomised crossover trial |
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 randomised crossover trial |
2.8 Are the unit costs of resources from the best available source? | Yes | UK recognised sources |
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 | 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? No, all relevant costs included and described |
Study identification Grieve R, Thompson S, Normand C, Suri R, Bush A, Wallis C. “A cost-effectiveness analysis of rhDNase in children with cystic fibrosis.” Int J Technol Assess Health Care. 2003: 19(1):71–9. |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase & HS |
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 and adverse effects not considered |
1.6 Are all future costs and outcomes discounted appropriately? | NA | Time horizon: 12 weeks |
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 | Outcomes associated with a resource use transformed into costs |
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 | Results taken from crossover trial |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon: 12 week crossover |
2.3 Are all important and relevant outcomes included? | Partly | QoL outcomes and adverse effects not considered |
2.4 Are the estimates of baseline outcomes from the best available source? | Yes | From randomised crossover trial |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | From randomised crossover trial |
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 randomised crossover trial |
2.8 Are the unit costs of resources from the best available source? | Yes | UK recognised sources |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | ICER £ per 1% gain in FEV1 |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | PSA |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Minor limitations |
Other comments:
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Study identification Christopher F, Chase D, Stein K, Milne R.J. rhDNase therapy for the treatment of cystic fibrosis patients with mild to moderate lung disease. Clin Pharm Ther. 1999 Dec;24(6):415–26. |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Partly | UK analysis based on US clinical effectiveness data |
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? | Yes | Time horizon: lifetime |
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 | LYG |
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? | Yes | Risk of death dependent on FEV1 |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon: 12 week crossover trial used to inform analysis |
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 | From US RCT |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | From US RCT |
2.6 Are all important and relevant costs included? | Partly | From US RCT |
2.7 Are the estimates of resource use from the best available source? | Partly | From US RCT |
2.8 Are the unit costs of resources from the best available source? | Yes | UK recognised sources |
2.9 Is an appropriate incremental analysis presented or can it be calculated from the data? | Yes | ICER cost per LYG |
2.10 Are all important parameters whose values are uncertain subjected to appropriate sensitivity analysis? | Yes | OWSA on FEV1 parameters and subgroup analysis |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Very serious limitations |
Other comments:
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Study identification Menzin J, Oster G, Davies L, Drummond MF, Greiner W, Lucioni C, Merot JL, Rossi F, vd Schulenburg JG, Souêtre E. “A multinational economic evaluation of rhDNase in the treatment of cystic fibrosis.” Int J Technol Assess Health Care. 1996: 12(1):52–61. |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Partly | UK analysis based on US clinical effectiveness data |
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: 24 weeks |
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-benefit analysis |
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-benefit analysis |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon: 24 weeks |
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 | From US RCT |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | From US RCT |
2.6 Are all important and relevant costs included? | No | From US RCT adapted to UK setting, insufficient detail reported on translation, cost of rhDNase not included |
2.7 Are the estimates of resource use from the best available source? | Partly | From US RCT adapted to UK setting, insufficient detail reported on translation |
2.8 Are the unit costs of resources from the best available source? | Unclear | Cost of care taken from 3 UK CF centres |
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? | No | Not assessed |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Very 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? Unclear, cost of rhDNase not included 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? Unclear |
Study identification McIntyre AM. “Dornase alpha and survival of patients with cystic fibrosis.” Hosp Med. 1999: 60(10):736–9. |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase |
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Partly | UK analysis based on non-UK clinical effectiveness data |
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? | Partly | Discount rate: 6% Time horizon: lifetime |
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-benefit analysis |
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-benefit analysis |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: lifetime |
2.3 Are all important and relevant outcomes included? | Yes | |
2.4 Are the estimates of baseline outcomes from the best available source? | Partly | From non-UK studies |
2.5 Are the estimates of relative intervention effects from the best available source? | Partly | From non-UK studies |
2.6 Are all important and relevant costs included? | Unclear | CF costs categorised into mild, moderate or severe CF |
2.7 Are the estimates of resource use from the best available source? | Unclear | From non-UK studies, insufficient detail on how CF care is costed according to severity |
2.8 Are the unit costs of resources from the best available source? | Partly | Cost of care taken from a UK study (Robson 1992), based on severity |
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? | Yes | Improvement with rhDNase varied |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: Very 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? Unclear 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? Unclear |
Study identification Mannitol HTA |
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Guidance topic: Cystic Fibrosis | Question no: 11 |
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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 | rhDNase & mannitol |
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 |
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 | |
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon: lifetime |
2.3 Are all important and relevant outcomes included? | Yes | |
2.4 Are the estimates of baseline outcomes from the best available source? | Yes | Patient level data |
2.5 Are the estimates of relative intervention effects from the best available source? | Yes | Patient level data, note the effect of mannitol was assumed to be the same in rhDNase users and non-users in the manufacturer’s initial submission |
2.6 Are all important and relevant costs included? | Yes | Patient level data |
2.7 Are the estimates of resource use from the best available source? | Yes | Patient level data |
2.8 Are the unit costs of resources from the best available source? | Yes | 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 | PSA |
2.11 Is there any potential conflict of interest? | No | |
2.12 Overall assessment: No limitations |