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
Guidance topic: Cystic FibrosisQuestion no: 13
Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5)Yes/partly/no/unclear/NAComments
1.1 Is the study population appropriate for the review question?YesPeople with CF
1.2 Are the interventions appropriate for the review question?YesColi 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?YesUK
1.4 Are the perspectives clearly stated and are they appropriate for the review question?YesNHS
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?YesTime 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:
Section 2: Study limitations (the level of methodological quality)Yes/partly/no/unclear/NAComments
2.1 Does the model structure adequately reflect the nature of the topic under evaluation?YesTreatment 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?YesTime 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?YesFrom RCT
2.5 Are the estimates of relative intervention effects from the best available source?YesFrom RCT
2.6 Are all important and relevant costs included?YesFull details on costs provided
2.7 Are the estimates of resource use from the best available source?YesFrom RCT
2.8 Are the unit costs of resources from the best available source?YesFrom 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?YesOWSA and PSA
2.11 Is there any potential conflict of interest?No
2.12 Overall assessment: No limitations
Other comments:
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
Guidance topic: Cystic FibrosisQuestion no: 13
Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5)Yes/partly/no/unclear/NAComments
1.1 Is the study population appropriate for the review question?YesPeople with CF
1.2 Are the interventions appropriate for the review question?YesColi DPI vs. NT
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context?YesUK
1.4 Are the perspectives clearly stated and are they appropriate for the review question?YesNHS
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?YesTime 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:
Section 2: Study limitations (the level of methodological quality)Yes/partly/no/unclear/NAComments
2.1 Does the model structure adequately reflect the nature of the topic under evaluation?YesTreatment 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?YesTime 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?YesFrom RCT
2.5 Are the estimates of relative intervention effects from the best available source?YesFrom RCT
2.6 Are all important and relevant costs included?YesFull details on costs provided
2.7 Are the estimates of resource use from the best available source?YesFrom RCT
2.8 Are the unit costs of resources from the best available source?YesFrom 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?YesOWSA and PSA
2.11 Is there any potential conflict of interest?No
2.12 Overall assessment: No limitations
Other comments:
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
Guidance topic: Cystic FibrosisQuestion no: 13
Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5)Yes/partly/no/unclear/NAComments
1.1 Is the study population appropriate for the review question?YesPeople with CF
1.2 Are the interventions appropriate for the review question?YesNT
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context?YesUK
1.4 Are the perspectives clearly stated and are they appropriate for the review question?YesNHS
1.5 Are all direct effects on individuals included, and are all other effects included where they are material?PartlyHRQoL not considered
1.6 Are all future costs and outcomes discounted appropriately?NATime 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).NoOutcome 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/NAComments
2.1 Does the model structure adequately reflect the nature of the topic under evaluation?NACost-benefit analysis
2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes?PartlyTime horizon: 12 months
2.3 Are all important and relevant outcomes included?PartlyQoL outcomes not considered
2.4 Are the estimates of baseline outcomes from the best available source?PartlyBefore and after study
2.5 Are the estimates of relative intervention effects from the best available source?PartlyBefore and after study
2.6 Are all important and relevant costs included?PartlyInsufficient detail
2.7 Are the estimates of resource use from the best available source?PartlyBefore and after study, but resource use not described in detail
2.8 Are the unit costs of resources from the best available source?UnclearNot 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?No95% 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
Guidance topic: Cystic FibrosisQuestion no: 13
Section 1: Applicability (relevance to specific review questions and the NICE reference case as described in section 7.5)Yes/partly/no/unclear/NAComments
1.1 Is the study population appropriate for the review question?YesPeople with CF
1.2 Are the interventions appropriate for the review question?YesAztreonam vs. NT
1.3 Is the system in which the study was conducted sufficiently similar to the current UK context?NoUS
1.4 Are the perspectives clearly stated and are they appropriate for the review question?Yes and noStated 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?YesTime 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?UnclearCost sources not described
1.9 Overall judgement: Partially applicable
Other comments:
Section 2: Study limitations (the level of methodological quality)Yes/partly/no/unclear/NAComments
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?YesTime 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?YesFrom RCT with an open label extension
2.5 Are the estimates of relative intervention effects from the best available source?YesBut note estimates of effectiveness not reproducible
2.6 Are all important and relevant costs included?UnclearInsufficient detail
2.7 Are the estimates of resource use from the best available source?UnclearInsufficient detail
2.8 Are the unit costs of resources from the best available source?UnclearInsufficient 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?YesScenario analysis, univariate and PSA
2.11 Is there any potential conflict of interest?PartlySupported by Gilead Sciences
2.12 Overall assessment: Serious limitations
Other comments:

From: Appendix M, Health economics quality assessment

Cover of Cystic Fibrosis
Cystic Fibrosis: Diagnosis and management.
NICE Guideline, No. 78.
National Guideline Alliance (UK).
Copyright © NICE 2017.

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