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Study identification Bensmail, D, Ward, Ab, Wissel, J, Motta, F, Saltuari, L, Lissens, J, Cros, S, Beresniak, A. Cost-effectiveness modeling of intrathecal baclofen therapy versus other interventions for disabling spasticity. Neurorehabilitation and Neural Repair 2009 | ||
|---|---|---|
| Guidance topic: Cerebral palsy in adults | Question no: A.2 | |
| 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 |
| 1.1 Is the study population appropriate for the review question? | Partly | Patients with disabling spasticity and functional dependence caused by any neurological disease |
| 1.2 Are the interventions appropriate for the review question? | Yes | Intrathecal baclofen (ITB) therapy compared with conventional medical treatments |
| 1.3 Is the system in which the study was conducted sufficiently similar to the current UK context? | Yes | France |
| 1.4 Are the perspectives clearly stated and are they appropriate for the review question? | Yes | Only direct costs of treatment included according to French Guidelines for economic evaluations |
| 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 2 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 | Successful treatment (defined as a combination of: the increased patient and caregiver satisfaction as assessed by goal attainment scaling (GAS), and a decrease of at least 1 point on the Ashworth score) |
| 1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Yes | |
| 1.9 Overall judgement: Partially applicable | ||
| Other comments: | ||
| Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
| 2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | Yes | Decision tree structure. An expert panel was used to define the treatment sequences (model structure) and review parameters estimates of the historical databases. |
| 2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Partly | Time horizon 2 years |
| 2.3 Are all important and relevant outcomes included? | Yes | See 2.1 |
| 2.4 Are the estimates of baseline outcomes from the best available source? | Unclear | Clinical effectiveness data is included in the model but sources and values are not reported |
| 2.5 Are the estimates of relative intervention effects from the best available source? | Unclear | See 2.4 |
| 2.6 Are all important and relevant costs included? | Yes | Treatment costs were calculated based on hospital costs in France including drug costs, physician visits, procedure costs, hospitalization, nursing care, physical treatments, surgery, transportation services, device acquisition costs (ITB), complication costs of ITB treatment, cost of managing pressure sores, and severe muscle contractions. |
| 2.7 Are the estimates of resource use from the best available source? | Unclear | Not all cost sources reported. Direct medical costs were measured in Euros (2006) and based on a French retrospective cost survey at Raymond Poincaré Hospital. |
| 2.8 Are the unit costs of resources from the best available source? | Unclear | See 2.7 |
| 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 (5,000 iterations) |
| 2.11 Is there any potential conflict of interest? | No | Not reported |
| 2.12 Overall assessment: Potentially serious limitations | ||
| Other comments: | ||
|
Study identification Sampson, F. C., Hayward, A., Evans, G., Morton, R., Collett, B. Functional benefits and cost/benefit analysis of continuous intrathecal baclofen infusion for the management of severe spasticity. Journal of Neurosurgery 2002 | ||
| Guidance topic: Cerebral palsy in adults | Question no: A.2 | |
| 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 |
| 1.1 Is the study population appropriate for the review question? | Partly | Severe spasticity, not all papers included in the meta-analysis included participants with CP |
| 1.2 Are the interventions appropriate for the review question? | Yes | Continuous intrathecal baclofen infusion |
| 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 | Only direct costs of treatment included. |
| 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 |
Time horizon 5 years. Costs and benefits were discounted 6% per annum (NICE reference case 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: Partially applicable | ||
| Other comments: | ||
| Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
| 2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | NA | DAM not developed |
| 2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon 5 years (the lifespan of ITB equipment) |
| 2.3 Are all important and relevant outcomes included? | Yes | |
| 2.4 Are the estimates of baseline outcomes from the best available source? | Yes | Estimation of benefits identified from a systematic review of the literature |
| 2.5 Are the estimates of relative intervention effects from the best available source? | Yes | See 2.4 |
| 2.6 Are all important and relevant costs included? | Yes | A separate literature search was performed to identify existing economic analyses or cost studies relating to continuous intrathecal baclofen infusion. Key cost elements were identified from the literature and from semi structured interviews with clinicians from hospitals in the UK. |
| 2.7 Are the estimates of resource use from the best available source? | Yes | See 2.6 |
| 2.8 Are the unit costs of resources from the best available source? | Yes | See 2.6 |
| 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? | No | One threshold sensitivity analysis was performed to examine the annual gains in health state values (QALYs) required to provide specific cost-effectiveness ratios of between £5000 and £25,000 per QALY |
| 2.11 Is there any potential conflict of interest? | No | Not reported |
| 2.12 Overall assessment: Potentially serious limitations | ||
| Other comments: | ||
|
Study identification Saulino, M., Guillemette, S., Leier, J., Hinnenthal, J. Medical cost impact of intrathecal baclofen therapy for severe spasticity. Neuromodulation 2015 | ||
| Guidance topic: Cerebral palsy in adults | Question no: A.2 | |
| 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 |
| 1.1 Is the study population appropriate for the review question? | Yes | Informed by data that included participants with multiple sclerosis (N=124), cerebral palsy (N=131) and spinal cord injury (N=40) |
| 1.2 Are the interventions appropriate for the review question? | Yes | Intrathecal baclofen compared to continued conventional medical management (pre-pump implantation) |
| 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? | Partly | US third party payer. Indirect costs are not reported but the perspective is not explicitly stated. |
| 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 30 years. 3% annual discount rate applied (note slight deviation from NICE’s preferred 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). | No | |
| 1.8 Are costs and outcomes from other sectors fully and appropriately measured and valued? | Yes | |
| 1.9 Overall judgement: Partially applicable | ||
| Other comments: | ||
| Section 2: Study limitations (the level of methodological quality) | Yes/partly/no/unclear/NA | Comments |
| 2.1 Does the model structure adequately reflect the nature of the topic under evaluation? | NA | DAM not developed. Cost-benefit analysis. |
| 2.2 Is the time horizon sufficiently long to reflect all important differences in costs and outcomes? | Yes | Time horizon 30 years |
| 2.3 Are all important and relevant outcomes included? | Partly | It was assumed that future costs would follow a reasonable trend rate based on healthcare industry standards. Device-related complications were included. |
| 2.4 Are the estimates of baseline outcomes from the best available source? | Partly | Model informed by retrospective analysis of commercial administrative claims data. A systematic review of the literature was not undertaken. |
| 2.5 Are the estimates of relative intervention effects from the best available source? | Partly | See 2.4 |
| 2.6 Are all important and relevant costs included? | Yes | |
| 2.7 Are the estimates of resource use from the best available source? | Yes | See 2.4 |
| 2.8 Are the unit costs of resources from the best available source? | Yes | See 2.4 |
| 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? | Partly |
SA for different values of three variables: 1) the drug delivery system’s battery life, 2) the length of the pre implant experience period used to establish average starting costforprojectionpurposes,and3)the medical cost trend assumptions. PSA not undertaken |
| 2.11 Is there any potential conflict of interest? | Yes | Funded by Medtronic, Inc. |
| 2.12 Overall assessment: Potentially serious limitations | ||
| Other comments: | ||
From: Management of abnormal muscle tone: neurosurgical procedures to reduce spasticity
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