Cost-effectiveness of using Polyheal compared with surgery in the management of chronic wounds with exposed bones and/or tendons due to trauma in France, Germany and the UK.

The objective of this study was to assess the cost-effectiveness of Polyheal compared with surgery in treating chronic wounds with exposed bones and/or tendons (EB&T) due to trauma in France, Germany and the UK, from the perspective of the payers. Decision models were constructed depicting the management of chronic wounds with EB&T and spanned the period up to healing or up to 1 year. The models considered the decision by a plastic surgeon to treat these wounds with Polyheal or surgery and was used to estimate the relative cost-effectiveness of Polyheal at 2010/2011 prices. Using Polyheal instead of surgery is expected to increase the probability of healing from 0·93 to 0·98 and lead to a total health-care cost of €7984, €7517 and €8860 per patient in France, Germany and the UK, respectively. Management with surgery is expected to lead to a total health-care cost of €12 300, €18 137 and €11 330 per patient in France, Germany and the UK, respectively. Hence, initial treatment with Polyheal instead of surgery is expected to lead to a 5% improvement in the probability of healing and a substantial decrease in health-care costs of 35%, 59% and 22% in France, Germany and the UK, respectively. Within the models' limitations, Polyheal potentially affords the public health-care system in France, Germany and the UK a cost-effective treatment for chronic wounds with EB&T due to trauma, when compared with surgery. However, this will be dependent on Polyheal's healing rate in clinical practice when it becomes routinely available.

Cost-effectiveness of using an extensively hydrolysed formula compared to an amino acid formula as first-line treatment for cow milk allergy in the UK.

OBJECTIVE: To estimate the cost-effectiveness of using an extensively hydrolysed formula (eHF; Nutramigen) compared with an amino acid formula (AAF; Neocate) as first-line treatment for cow milk allergy (CMA) in the UK, from the perspective of the National Health Service (NHS). METHOD: A decision model was constructed depicting the treatment paths and associated resource use attributable to first-line management of CMA with the two formulae. The model was based on the case records of 145 AAF-treated infants and 150 matched eHF-treated patients from The Health Improvement Network (THIN) database [a nationally representative database of patients registered with general practitioners (GPs) in the UK]. The model estimated the costs and consequences of patient management over 12 months following their initial GP visit for CMA. RESULTS: Patients presenting with a combination of gastrointestinal symptoms and eczema accounted for 44% of all patients in both groups. Those with gastrointestinal symptoms alone and eczema alone accounted for up to a further 39% and 13%, respectively. Those with urticaria and faltering growth accounted for <5% and ≤6% of all patients, respectively. Patients' age and weight at presentation was a mean 2.6-2.8 months and 4.4 kg, respectively. It took a mean 2.2 months to start a formula after initially seeing a GP. Time to symptom resolution following the start of treatment was 1.2 months in both groups; hence, the mean number of symptom-free months during the 12 months following the initial GP visit was estimated to be 8.6 months in both groups. Patients treated with an eHF had a mean 13.1 GP visits over the 12 months compared to 17.5 visits made by AAF-treated patients (p < 0.001). The NHS cost of managing a CMA infant over the first 12 months following initial presentation to a GP was estimated to be £1853 and £3161 for an eHF-treated and AAF-treated patient, respectively. CONCLUSION: Starting treatment for CMA with an eHF was the cost-effective option, as there were no significant differences in clinical outcomes between the two groups. A prospective, randomized controlled trial would allow a definitive confirmation of these findings.

Clinical outcomes and cost-effectiveness of continuous positive airway pressure to manage obstructive sleep apnea in patients with type 2 diabetes in the U.K.

OBJECTIVE: To assess clinical outcomes and cost-effectiveness of using continuous positive airway pressure (CPAP) to manage obstructive sleep apnea (OSA) in patients with type 2 diabetes (T2D) from the perspective of the U.K.'s National Health Service (NHS). RESEARCH DESIGN AND METHODS: Using a case-control design, 150 CPAP-treated patients with OSA and T2D were randomly selected from The Health Improvement Network (THIN) database (a nationally representative database of patients registered with general practitioners in the U.K.) and matched with 150 OSA and T2D patients from the same database who were not treated with CPAP. The total NHS cost and outcomes of patient management in both groups over 5 years and the cost-effectiveness of CPAP compared with no CPAP treatment were estimated. RESULTS: Using CPAP was associated with significantly lower blood pressure at 5 years and increasingly lower HbA1c levels over 5 consecutive years compared with untreated OSA patients. At 5 years, the HbA1c level in the CPAP-treated group was 8.2% (66.0 mmol/mol) vs. 12.1% (108.4 mmol/mol) in the control group (P < 0.03). Use of CPAP significantly increased patients' health status by 0.27 quality-adjusted life years (QALYs) per patient over 5 years (P < 0.001) and NHS management costs by £4,141 per patient over 5 years; the cost per QALY gained with CPAP was £15,337. CONCLUSIONS: Initiating treatment with CPAP in OSA patients with T2D leads to significantly lower blood pressure and better controlled diabetes and affords a cost-effective use of NHS resources. These observations have the potential for treatment modification if confirmed in a prospective study.

Utility values for advanced soft tissue sarcoma health States from the general public in the United kingdom.

Soft tissue sarcomas are a rare type of cancer generally treated with palliative chemotherapy when in the advanced stage. There is a lack of published health utility data for locally advanced "inoperable"/metastatic disease (ASTS), essential for calculating the cost-effectiveness of current and future treatments. This study estimated time trade-off (TTO) and standard gamble (SG) preference values associated with four ASTS health states (progressive disease, stable disease, partial response, complete response) among members of the general public in the UK (n = 207). The four health states were associated with decreases in preference values from full health. Complete response was the most preferred health state (mean utility of 0.60 using TTO). The second most preferred health state was partial response followed by stable disease (mean utilities were 0.51 and 0.43, respectively, using TTO). The least preferred health state was progressive disease (mean utility of 0.30 using TTO). The utility value for each state was significantly different from one another (P < 0.001). This study demonstrated and quantified the impact that different treatment responses may have on the health-related quality of life of patients with ASTS.

Cost effectiveness of first-line treatment with doxorubicin/ifosfamide compared to trabectedin monotherapy in the management of advanced soft tissue sarcoma in Italy, Spain, and sweden.

Background. Doxorubicin/ifosfamide is a first-line systemic chemotherapy for the majority of advanced soft tissue sarcoma (ASTS) subtypes. Trabectedin is indicated for the treatment of ASTS after failure of anthracyclines and/or ifosfamide; however it is being increasingly used off-label as a first-line treatment. This study estimated the cost effectiveness of these two treatments in the first-line management of ASTS in Italy, Spain, and Sweden. Methods. A Markov model was constructed to estimate the cost effectiveness of doxorubicin/ifosfamide compared to trabectedin monotherapy, defined as the cost per QALY gained, in each country. Results. First-line treatment with doxorubicin/ifosfamide resulted in lower two-year healthcare costs and more QALYs than first-line treatment with trabectedin monotherapy in all three countries. Probabilistic sensitivity analysis showed that at a cost per QALY threshold of €35,000, >90% of a cohort would be cost effectively treated with doxorubicin/ifosfamide compared to trabectedin monotherapy in all three countries. Conclusion. Within the model's limitations, first-line treatment of patients with ASTS with doxorubicin/ifosfamide instead of trabectedin monotherapy affords a cost-effective use of publicly funded healthcare resources in Italy, Spain, and Sweden and is therefore the preferred treatment in all three countries. These findings support the recommendation that trabectedin should remain a second-line treatment.

Utility values for chronic myelogenous leukemia chronic phase health states from the general public in the United Kingdom.

This study estimated time trade-off preference values associated with the four chronic myelogenous leukemia (CML) chronic phase-related health states (i.e. untreated, hematologic response, cytogenetic response and molecular response) among members of the general public in the UK (n = 241). All four health states were associated with decreases in preference values from full health. The molecular response to treatment was the most preferred health state (mean utility of 0.94). The second-most preferred health state was cytogenetic response followed by hematologic response (mean utilities were 0.89 and 0.80, respectively). The least preferred health state was untreated chronic phase CML (mean utility of 0.72). The utility values for each state were significantly different from one another (p < 0.001). This study demonstrated and quantified the impact that more robust treatment responses have on the health-related quality of life of patients with chronic phase CML.

Budget impact of managing cow milk allergy in the Netherlands.

OBJECTIVE: To assess the resource implications and budget impact of managing cow milk allergy (CMA) in the Netherlands from the perspective of the healthcare insurers. METHODS: A model was constructed depicting the management of CMA in the Netherlands using information obtained from interviews with youth healthcare doctors (n = 14), general practitioners (n = 6) and paediatricians (n = 11) with relevant clinical experience of managing CMA. The model was used to estimate the expected level of healthcare resource use and corresponding cost (at 2007/08 prices) attributable to managing 4,382 new CMA sufferers. RESULTS: The expected cost of healthcare resource use attributable to managing 4,382 new CMA sufferers up to 1 year of age following initial consultation with a community-based physician at a mean 3 months of age was estimated to be €11.28 (95% CI: €7.82; €14.33) million. Clinical nutrition preparations emerged as the primary cost driver accounting for 91% of the total cost and clinician visits collectively accounted for a further 5%. The time taken for CMA sufferers to be put on an appropriate diet and achieve symptom resolution was estimated to be 30 (95% CI: 27; 32) days. Sensitivity analysis showed that the costs would increase by approximately 16% if all new CMA sufferers were to undergo a double-blind placebo-controlled cow milk challenge in a hospital setting, as is currently being proposed. It is not clear how this proposal would affect time to symptom resolution since this would depend on the efficiency of hospitals being able to deal with the increased workload. LIMITATIONS: The intolerance rates were derived from a 1-year follow-up study among 1,000 infants with CMA in the UK, healthcare resource use was not collected prospectively and the study period was censured at 1 year of age and does not consider the impact of CMA in subsequent years. However, most children outgrow this form of allergy during their second year. CONCLUSION: Within the model's limitations, CMA imposes a substantial burden on the Dutch healthcare system. Moreover, initiating a double-blind placebo-controlled cow milk challenge for all CMA sufferers will potentially increase clinicians' workload and use of limited resources within paediatric hospital departments in the Netherlands.

Modelling the health economic impact of managing cow milk allergy in South Africa.

OBJECTIVE: To quantify the health economic impact of managing cow milk allergy (CMA) in South Africa, from the perspective of healthcare insurers in both the private and public sectors and parents/carers of CMA sufferers. METHODS: A decision model depicting the management of CMA in South Africa was constructed, using information obtained from interviews with paediatric specialists in the private and public sectors with relevant clinical experience. The model was used to estimate the expected 12-monthly levels of healthcare resource use and corresponding costs (at 2007/08 prices) attributable to managing CMA sufferers following an initial consultation with a paediatrician. RESULTS: The expected 12-monthly cost incurred by an insurer attributable to managing a CMA sufferer following an initial consultation with a paediatrician was estimated to be R2,430.4 (€202.0) and R1,073.7 (€89.0) in the private and public sector, respectively. The expected 12-monthly cost incurred by parents/carers following an initial consultation with a paediatrician was estimated to be R43,563.1 (€3,634.0) and R24,899.9 (€2,076.9) in the private and public sector, respectively. The time taken for a CMA sufferer to be put on an appropriate diet and achieve symptom resolution was estimated to be 24 days in the private sector and 18 days in the public sector. The total cost to manage an annual cohort of 18,270 newly diagnosed infants with CMA in South Africa in the first year following presentation to a paediatrician was estimated to be R22.1 (€1.8) million for healthcare insurers and R489.1 (€40.8) million for parents/carers. The expected costs to insurers were driven by visits to general paediatricians and prescriptions for dermatological drugs in both the private and public sectors. The expected costs to parents/carers were driven by over-the-counter (OTC) purchases of clinical nutrition preparations. LIMITATIONS: The intolerance rates were derived from a study among 1,000 infants with CMA in the UK, healthcare resource use was not collected prospectively and the study period was limited to 1 year following presentation to a paediatrician and does not consider the impact of CMA in subsequent years. However, most children outgrow this form of allergy by the time they reach 2 years of age. CONCLUSION: CMA imposes a substantial socio-economic burden in South Africa, especially on parents/carers of CMA sufferers. Any strategy that reduces this burden should potentially lead to higher compliance with clinicians' recommendations, thereby improving health outcomes associated with treatment and should also release healthcare resource use for alternative use.

Cost effectiveness of establishing a neonatal screening programme for phenylketonuria in Libya.

BACKGROUND: Inborn errors of metabolism (IEM) are a significant cause of morbidity and mortality in North Africa and the Middle East. With the evident success of neighbouring countries in initiating neonatal screening for IEM, the Libyan Authorities are now considering introducing neonatal screening for phenylketonuria (PKU) in Libya in the first instance, with the prospect of expanding the programme to cover other IEM in the future. OBJECTIVE: To estimate the cost effectiveness of neonatal screening for PKU compared with no neonatal screening in Libya. METHODS: A decision model was constructed to estimate the cost effectiveness of neonatal screening for PKU, from the perspective of Libyan society. Healthcare resource use and other input parameters were based on expert opinion. RESULTS: The expected discounted cost to Libyan society of screening over 15 years and managing ∼374 patients with detected PKU over their lifetime was estimated to be $US213.6 (95% CI 211.9, 214.3) million (year 2007-8 values). The current expected discounted cost of managing these same PKU patients over their lifetime as a result of not screening was estimated to be $US321.2 (95% CI 318.0, 322.7) million. Hence, screening would save Libyan society $US107.6 (95% CI 105.5, 109.1) million over the lifetime of PKU patients and lead to an additional 6947 life-years (95% CI 6837, 7056). The expected cost per undiscounted life-year gained was estimated to be -$US15,500 (95% CI -16,600, 1100). There would be a 90% return on investment in the screening programme since society would gain $US1.9 for every $US1 invested. Probabilistic sensitivity analysis demonstrated that the screening programme has a 0.95 probability of being cost effective even at a willingness-to-pay threshold of $US4000 per life-year gained. CONCLUSIONS: Within the model's limitations, neonatal screening for PKU appears to offer Libyan society a strategy that is cost effective compared with no neonatal screening.

Cost-effectiveness of pentostatin compared with cladribine in the management of hairy cell leukemia in the United Kingdom.

OBJECTIVE: This article assesses the cost-effectiveness of pentostatin compared with cladribine in the management of hairy cell leukemia (HCL) in the United Kingdom. METHODS: A systematic literature search for papers on HCL was performed using MEDLINE, EMBASE, Current Contents, NHS Economic Evaluation Database, and the Cochrane computerized database. Search terms were HCL plus 1 of the following: incidence, prevalence, epidemiology, cladribine, interferon, pentostatin, rituximab, splenectomy, utility, quality of life, cost-effectiveness, cost-utility, resource utilization, economic, or cost. Published clinical outcomes and estimates of health care resource use obtained from 10 consultant hematologists across the United Kingdom were used to construct a 5-year Markov model depicting the current management of HCL in the United Kingdom. Utilities for health states in the model were obtained from the general public using standard gamble, time tradeoff, and visual analog scale techniques. The model was used to consider the decision by a clinician to initially treat an HCL patient with either pentostatin or cladribine and to estimate the relative cost-effectiveness of pentostatin over 5 years (at 2007/2008 prices) from the perspective of the UK's National Health Service (NHS). RESULTS: According to the model, 64% of all pentostatin-treated patients are expected to be in relapse-free remission at 5 years compared with 49% of cladribine-treated patients (P = 0.04). Repeat treatment of initial partial responders, nonresponders, and those who relapse during the 5 years is expected to result in complete remission in 92% of pentostatintreated patients and 90% of cladribine-treated patients at 5 years. Using pentostatin instead of cladribine is expected to lead to a minimal cost increase (from 21,325 pounds to 21,609 pounds) and an improvement in health status (from 3.64 to 3.77 quality-adjusted life-years [QALYs]) over 5 years. Hence, the cost per QALY gained from using pentostatin is expected to be 5000 pounds. Moreover, pentostatin has a 0.90 probability of being cost-effective for a threshold of 20,000 pounds per QALY. Accordingly, using pentostatin as a first-line treatment for patients with HCL is an effective use of NHS resources. CONCLUSION: Based on current practice, this model predicts that pentostatin is a cost-effective treatment compared with cladribine in the management of HCL from the perspective of the UK's NHS.