Cost effectiveness of treatment of Parkinson's disease with entacapone in the United States

OBJECTIVE: To determine the cost effectiveness of adjunctive therapy with entacapone versus standard treatment (levodopa) without entacapone for patients in the US with Parkinson's disease (PD) who experience 'off-time' (re-emergence of the symptoms of PD) while receiving levodopa. STUDY DESIGN: A Markov model was used to estimate 5-year costs and effectiveness of standard treatment with and without entacapone. METHODS: Probabilities, unit costs, resource utilisation data and utilities were obtained from published literature, clinical trial reports, a national database, and clinical experts. PD disability was measured using the daily proportion of off-time and Hoehn and Yahr scale scores. The analysis measured costs from a societal and third-party payer perspective, and effectiveness as gains in quality-adjusted life-years (QALYs) and years without progression to >25% off-time. RESULTS: From a societal perspective, entacapone therapy resulted in an incremental cost of US dollars 9327 per QALY gained compared with standard treatment. Treatment with entacapone also provided an additional 7.6 months with < or =25% off-time/day compared with standard treatment. Sensitivity analyses indicated that the model is sensitive to changes in rates of improvement/deterioration of off-time, and to the number of doses per day of levodopa with adjunctive entacapone. CONCLUSIONS: The addition of entacapone to standard treatment for patients receiving levodopa who experience off-time provides additional QALYs and gain in time with minimal fluctuations. Results of this modelling exercise suggest that therapy with entacapone may be cost effective when compared with standard treatment for PD.     

Cost Effectiveness of Eplerenone for the Treatment of Systolic Heart Failure with Mild Symptoms in Alberta,Canada

Background

Eplerenone has been demonstrated as being cost effective for the treatment of patients with systolic heart failure (HF) and mild symptoms in several jurisdictions; however, its cost effectiveness is unknown in the context of Alberta, Canada.

Methods

We used a discrete-event simulation model to compare costs and outcomes between standard care and standard care plus eplerenone for the treatment of HF with mild symptoms. We used Alberta data (whenever possible) together with a healthcare perspective, a lifetime horizon, and 3 % annual discount rate for analyses.

Results

Clinically, eplerenone prevented HF hospitalizations, atrial fibrillations, and cardiovascular (CV) deaths, but incurred more adverse events and device implantations than standard care. The remaining life of patients receiving eplerenone was 7.08 versus 5.83 years for those receiving standard care. Eplerenone gained 1.25 life-years and 1.18 quality-adjusted life-years (QALYs), with an incremental cost of $Can7200. Therefore, the incremental cost-effectiveness ratio (ICER) was $Can5700 per life-year gained and $Can6100 per QALY gained.

Conclusions

Given the most cited ICER threshold is $Can50,000, the use of eplerenone as an adjunct to standard care for treating patients with systolic HF and mild symptoms is cost effective in the context of Alberta. Eplerenone would cost the Alberta health system about $Can4.6 million a year in drug costs. Incorporating reductions in health services utilization associated with eplerenone, the budget impact is smaller. For the first year, the use of eplerenone is cost saving and for 5 years the cost is approximately $Can6 million.

     

Cost effectiveness of recombinant human insulin-like growth factor I therapy in patients with ALS.

OBJECTIVE: Amyotrophic lateral sclerosis (ALS) is a fatal, degenerative neuromuscular disease characterised by a progressive loss of voluntary motor activity. Recombinant human insulin-like growth factor I (rhIGF-I) has been shown to be useful in treating ALS. The purpose of this study was to examine the cost effectiveness of rhIGF-I therapy in patients who have ALS. DESIGN: We performed a cost-effectiveness analysis from the societal perspective on 177 patients who received treatment with rhIGF-I or placebo in a North American randomised clinical trial. We estimated the incremental cost-effectiveness ratio of rhIGF-I using resource utilisation and functional status measurements from the clinical trial. Costs were estimated from 1996 US Medicare reimbursement schedules. Utility weights were elicited from ALS healthcare providers using the standard gamble technique. MAIN OUTCOME MEASURES AND RESULTS: The overall cost per quality-adjusted life-year (QALY) gained for rhIGF-I therapy compared with placebo was $US67,440. For the subgroups of patients who were progressing rapidly or were in earlier stages of disease at enrolment, rhIGF-I cost $US52,823 and $US43,197 per QALY gained, respectively. CONCLUSIONS: Treatment with rhIGF-I is most cost effective in ALS patients who are either in earlier stages of the disease or progressing rapidly. The cost effectiveness of rhIGF-I therapy compares favourably with treatments for other chronic progressive diseases.     

The long-term cost effectiveness of treatments for benign prostatic hyperplasia

INTRODUCTION: Excellent treatment outcomes with long-term durability and few adverse effects are expectations of treatments for chronic conditions. The long-term cost effectiveness of newer treatments for benign prostatic hyperplasia (BPH), including high-energy transurethral microwave thermotherapy (TUMT) and combination pharmaceutical therapy, has not been sufficiently studied against existing alternatives. The objective of this study was to estimate the incremental cost effectiveness of BPH treatment alternatives. METHODS: We employed a Markov model over a 20-year time horizon and the payer's perspective to evaluate the cost effectiveness of watchful waiting (WW), pharmaceuticals (alpha-adrenoceptor antagonists [alpha-blockers], 5-alpha-reductase inhibitors [5-ARIs], combination therapy), TUMT and transurethral resection of the prostate (TURP) in treating BPH. Markov states included improvement in symptoms, no improvement in symptoms, adverse effects and death.We used data from the published literature for outcomes, including systematic reviews whenever possible. Costs were estimated using a managed-care claims database and Medicare fee schedules, and were reported in Dollars US, 2004 values. Costs and effectiveness outcomes were discounted at a rate of 3% per year. Men (aged > or =45 years) with moderate to severe lower urinary tract symptoms and uncomplicated BPH were included in the analysis, and results were stratified by age and BPH symptom levels.Outcomes included costs, QALYs, incremental cost-utility ratios and cost-effectiveness acceptability curves. Sensitivity analysis was performed on important parameters, with an emphasis on probabilistic sensitivity analysis. RESULTS: alpha-Blockers and TUMT were cost effective for treating moderate symptoms using the threshold of Dollars US 50,000 per QALY. For example, at 65 years of age, the cost per QALY was Dollars US 16,018 for alpha-blockers compared with WW and Dollars US 30,204 for TUMT versus alpha-blockers. TURP was the most cost-effective treatment for severe symptoms (Dollars US 5824 per QALY ) versus WW. Model results were robust to changes in costs and sensitive to the assumed probabilities, utility weights, extent of improvement and life expectancy. Nevertheless, acceptability curves consistently demonstrated the same alternatives as most likely to be cost effective. CONCLUSIONS: Our model suggests that alpha-blockers and TURP appear to be the most cost-effective alternatives, from a US payer perspective, for BPH patients with moderate and severe symptoms, respectively. TUMT was promising for patients with moderate symptoms and the oldest patients with severe symptoms, but otherwise was dominated. Value of information analysis could be used to determine the net benefit of additional research.     

Cost effectiveness of combination therapy with pioglitazone for type 2 diabetes mellitus from a german statutory healthcare perspective

BACKGROUND: Pioglitazone has been approved in Europe for oral combination therapy for type 2 diabetes mellitus. Along with other agents of the thiazolidinedione class, it has a novel intracellular mechanism of action. Clinical trials with pioglitazone have confirmed a strong product profile in terms of control of blood glucose and lipids. However, the drug acquisition cost for pioglitazone is greater than standard medications for type 2 diabetes. Long-term data regarding the cost effectiveness of pioglitazone-based combination therapy are not available. OBJECTIVE: To evaluate, using a decision analysis model, the cost effectiveness of pioglitazone-based combination therapy compared with relevant alternative medications for the treatment of type 2 diabetes in Germany. METHODS: This study compared the clinical effects and costs of pioglitazone 30 mg added to metformin in patients who failed metformin monotherapy and pioglitazone added to a sulphonylurea in patients who failed sulphonylurea monotherapy, with the most relevant treatment alternatives. A published and validated Markov model was adapted to reflect the management of type 2 diabetes. This simulated the number of severe complications occurring and the mean life expectancy of a diabetic cohort, which was based on the overweight group of the UK Prospective Diabetes Study at year 6 of follow-up. Drug treatment costs, other costs for general management of type 2 diabetes and the costs of complications were combined to compute overall lifetime treatment costs from the perspective of the German statutory healthcare system in 2002. RESULTS: Combination therapy with pioglitazone/metformin was associated with a higher life expectancy (15.2 years) relative to sulphonylurea/metformin (14.9 years) or acarbose/metformin (14.7 years). Likewise, pioglitazone/sulphonylurea (15.5 years) was superior to metformin/sulphonylurea (14.9 years) and acarbose/sulphonylurea (14.8 years). Undiscounted incremental cost-effectiveness ratios in comparison to the next best strategy were euro20,002 per life-year gained (LYG) for pioglitazone/metformin versus sulphonylurea/metformin, and euro8707 per LYG for pioglitazone/sulphonylurea versus metformin/sulphonylurea. After discounting costs and life expectancy at 5% per year, the incremental cost-effectiveness ratio was euro47 636 per LYG for pioglitazone/metformin versus sulphonylurea/metformin, and euro19 745 per LYG for pioglitazone/sulphonylurea versus metformin/sulphonylurea. CONCLUSIONS: In this model, with its underlying assumptions and data, combination therapy with pioglitazone increased life expectancy in overweight type 2 diabetes patients at acceptable cost compared with other well established medications in Germany. These findings should be re-evaluated as soon as additional evidence becomes available from the currently ongoing long-term clinical and economic studies.     

Cost effectiveness of oseltamivir treatment for patients with influenza-like illness who are at increased risk for serious complications of influenza: illustration for the Netherlands

BACKGROUND: Oseltamivir is effective in the treatment of influenza. Utilisation in The Netherlands is limited, but increasing. OBJECTIVE: To estimate the cost effectiveness of oseltamivir treatment (vs symptom relief only) for patients with influenza-like illness (ILI) who are at increased risk for serious complications of influenza. METHODS: A cost-effectiveness analysis was used, building on a previously developed model (decision tree) that was applied for evaluating influenza vaccination and pandemic preparedness plans. Three patient subgroups were assessed (elderly patients [aged > or = 65 years] without chronic disease, elderly patients with chronic disease, and chronically ill, non-elderly patients). Inputs for the model were taken from various sources including a meta-analysis. A societal perspective was adopted and costs were expressed in euro per life-year gained (year 2003 values). Life-years lost were discounted at 4% in accordance with Dutch guidelines. Deterministic and probabilistic sensitivity analyses were employed to assess the robustness of the results. RESULTS: For chronically ill patients with ILI, visits to the GP for oseltamivir treatment were cost saving. For non-chronically ill elderly patients, incremental cost-effectiveness was estimated at 1759 euros per life-year gained. Cost savings and favourable cost effectiveness were robust in a deterministic and stochastic sensitivity analysis. CONCLUSION: Our model-based analysis suggests that at-risk people presenting with ILI to a GP could be offered oseltamivir at favourable cost effectiveness or even cost savings in the Dutch setting compared with symptom relief with analgesics only.     

A pharmacoeconomic analysis of patients with symptoms of benign prostatic hyperplasia

A pharmacoeconomic analysis of therapies for patients with benign prostatic hyperplasia (BPH) was conducted. The therapies compared were androgenic hormone inhibition (finasteride) and alpha-blockade (doxazosin, prazosin and terazosin). This was a cost-effectiveness analysis from the perspective of the US military. The 36-month decision-tree model considered the aforementioned drugs as initial therapy for BPH following an unsuccessful period of watchful waiting. Therapy was continued toward a successful response. All patients who did not respond to therapy received secondary interventions, including transurethral resection of the prostate (TURP). The main outcome measures were clinical effectiveness and incurred costs. A Monte Carlo sensitivity analysis was performed on all cost-effectiveness ratios. The model and sensitivity analysis supported prazosin as the most cost effective alpha-blocker over finasteride: the mean difference was $US381.65 (1994 values) per successfully treated patient, with a range of $US57.83 to $US675.53, in favour of prazosin. If prazosin was used as initial drug therapy after watchful waiting for a man over 50 years of age with classical symptoms of prostatism and no other severe or confounding comorbid conditions, a cost of $US578.15 per treatment could be expected, with clinical effectiveness of 70.3%. Patients who cannot tolerate prazosin should be considered for terazosin therapy before moving on from alpha-blockers. Subsequent treatment with finasteride would cost $US1426.53, with an additional clinical effectiveness of 9.9%. For the small number of patients who fail both therapies, the cost effectiveness of a first TURP as 'third-line' intervention [$US4321.36 for an additional effectiveness of 8.62% and a repeat TURP as 'fourth-line' ($US7650.54 for 0.59%) interventional] was calculated in a similar manner. Costs were cumulative, and effectiveness was derived from the total number of patients who started prazosin therapy. Pharmacological therapy was more cost effective than surgical intervention, and alpha-blockers were more cost effective than finasteride. Among the alpha-blockers, prazosin was by far the most cost effective followed by terazosin, then doxazosin.     

Cost effectiveness of letrozole in the treatment of advanced breast cancer in postmenopausal women in the UK

OBJECTIVE: To simulate the treatment of postmenopausal women with advanced breast cancer from second-line hormone therapy to death, and to generate estimates of the cost and effectiveness of letrozole and megestrol in order to determine the incremental cost effectiveness of letrozole, expressed as cost per life-years gained. DESIGN: A decision-analytic model, using Markov process techniques, was designed to evaluate the lifetime clinical and economic consequences of treatment with letrozole compared with standard care with megestrol. The model was based on clinical trial results showing a clear advantage of letrozole in terms of time to progression and duration of response. SETTING: The setting of the study was that of the UK healthcare system in 1996. PATIENTS AND PARTICIPANTS: A hypothetical cohort of patients, identical to the patients recruited for the AR/BC2 clinical trial, who were postmenopausal women with advanced breast cancer who had previously failed to respond to first-line or adjuvant anti-estrogen therapy. INTERVENTIONS: The dosages of medications were 2.5 and 160 mg/day for letrozole and megestrol, respectively. The analysis covered the period from treatment initiation until death (lifetime model). Effectiveness was expressed as survival and time without progression, and the model also included all relevant economic measures. MAIN OUTCOME MEASURES AND RESULTS: Based on the model, the average survival time of the letrozole group was 2.1 years (25.3 months) versus 1.9 years (21.5 months) for the megestrol group, a gain in survival of 2.4 months (10.5%). The average time without progression, cumulatively calculated over the different treatment options, amounted to 20.2 months for letrozole and 17.8 months for megestrol, an increase of 13.7% for the former patients. The total average cost per patient for the treatment of advanced breast cancer starting from second-line hormone therapy until death was higher in the letrozole group at 7547 Pounds versus 6820 Pounds for the megestrol group (discounted at an annual rate of 5%), leading to an incremental cost-effectiveness ratio of 3588 Pounds per life-year gained (1996 values). CONCLUSIONS: Based on the assumptions used in this model, letrozole offers a suitable alternative to megestrol in the treatment of second-line hormone therapy.     

Cost effectiveness of entecavir versus lamivudine with adefovir salvage in HBeAg-positive chronic hepatitis B

OBJECTIVE: To evaluate the cost effectiveness of treatment of hepatitis B e antigen (HBeAg)-positive chronic hepatitis B (CHB) with entecavir compared with lamivudine with adefovir salvage, based primarily on the results of a recent 2-year, randomised, multicentre, clinical trial (n = 709). Previous economic analyses have been limited by the lack of comparative clinical data for entecavir and lamivudine beyond 1-year duration and for salvage therapy. METHODS: We conducted a cost-utility analysis using a Markov model from a US-payer perspective over a lifetime time horizon. The hypothetical cohort was 35-year-old patients with HBeAg-positive CHB. We evaluated 2 years of treatment with entecavir 0.5mg/day versus lamivudine 100mg/day, plus addition of adefovir 10mg/day for patients who developed virologic breakthrough due to resistance to either drug. In a scenario analysis, we considered adefovir plus lamivudine combination therapy for treatment-naive patients. Clinical and economic inputs ($US, year 2006 values) were derived from publicly available data, and probabilistic sensitivity analyses were conducted to evaluate uncertainty in the results. RESULTS: The estimated 10-year cumulative incidence of cirrhosis for patients initiated on entecavir was 2.3% lower than for those on lamivudine (20.5% vs 22.8%). The discounted incremental cost per QALY gained was $US7600 in the base-case analysis, and the 95% central range from probabilistic sensitivity analysis was $US2500-$US19 100. Combination therapy for treatment-naive patients led to an increase in costs without improvement in patient outcomes compared with entecavir monotherapy. CONCLUSIONS: Our analysis suggests entecavir improves health outcomes in a cost-effective manner compared with lamivudine with adefovir salvage or combination therapy, and highlights the importance of using evidence-based effectiveness estimates in economic studies of CHB therapies.     

Modelling the long term cost effectiveness of clopidogrel for the secondary prevention of occlusive vascular events in the UK

OBJECTIVE: To assess the long term cost effectiveness of clopidogrel monotherapy compared with acetylsalicylic acid (aspirin; ASA) monotherapy in patients at risk of secondary occlusive vascular events (OVEs) in the UK. DESIGN: Cost utility analysis based on clinical data from CAPRIE (a multicentre randomised controlled trial, involving 19185 patients); long-term effects were extrapolated beyond the trial period using a Markov model populated with data from UK observational studies. Health economic evaluation carried out from the perspective of the UK National Health Service. PARTICIPANTS: A representative cohort of 1000 UK patients aged 60 years (approximate mean age of the CAPRIE population), with the qualifying diagnoses of myocardial infarction, ischaemic stroke and peripheral arterial disease, who are at risk of secondary OVEs (non-fatal myocardial infarction, non-fatal stroke or vascular death). INTERVENTIONS: Patients were assumed to receive treatment with either clopidogrel (75 mg/day) for 2 years followed by ASA (325 mg/day, average) for their remaining lifetime, or ASA alone (325 mg/day, average) for life. MAIN OUTCOME MEASURES: Incremental cost per life year gained and incremental cost per quality-adjusted life year (QALY) gained. RESULTS: In the base case, the incremental cost effectiveness of clopidogrel versus ASA in this population is estimated at 18888 pounds per life year gained and 21 489 pounds per QALY gained. Multiple deterministic and probabilistic sensitivity analyses suggest the model is robust to variations in a wide range of input parameters. CONCLUSION: Two years of treatment with clopidogrel can be considered a cost effective intervention in patients at risk of secondary OVEs in the UK.     

Long-acting risperidone compared with oral olanzapine and haloperidol depot in schizophrenia: a Belgian cost-effectiveness analysis

Patients with schizophrenia suffer numerous relapses and rehospitalizations that are associated with high direct and indirect medical expense. Suboptimal therapeutic efficacy and, in particular, problems with compliance are major factors leading to relapse. Atypical antipsychotic agents offer improved efficacy and a lower rate of extrapyramidal adverse effects compared with conventional antipsychotic drugs. Long-acting intramuscular risperidone combines these benefits with improvements in compliance associated with depot injections. To assist decision making regarding the place of long-acting risperidone in therapy, a cost-effectiveness analysis of strategies involving first-line treatment with long-acting risperidone, oral olanzapine or depot haloperidol was performed from the perspective of the Belgian healthcare system. A decision tree model was created to compare the cost effectiveness of three first-line treatment strategies in a sample of young schizophrenic patients who had been treated for 1 year and whose disease had not been diagnosed for longer than 5 years. The model used a time horizon of 2 years, with health state transition probabilities, resource use and cost estimates derived from clinical trials, expert opinion and published prices. The four health states in the model were derived from an analysis of the literature. The principal efficacy measure was the proportion of patients successfully treated, defined as those who responded to initial treatment and who had none to two episodes of clinical deterioration without needing a change of treatment over the 2-year period. Comprehensive sensitivity analysis was carried out to test the robustness of the model. A greater proportion of patients were successfully treated with long-acting risperidone (82.7%) for 2 years, compared with those treated with olanzapine (74.8%) or haloperidol (57.3%). Total mean costs per patient over 2 years were 16,406 Euro with long-acting risperidone, 17,074 Euro with olanzapine and 21,779 Euro with haloperidol (year of costing 2003). The mean cost-effectiveness ratios were 19,839 Euro, 22,826 Euro and 38,008 Euro per successfully treated patient for long-acting risperidone, olanzapine and haloperidol, respectively. Results of the sensitivity analysis confirmed that the results were robust to a wide variation of different input variables (effectiveness, dosing distribution, patient status according to healthcare system). Long-acting risperidone was the dominant strategy, being both more effective and less costly than either oral olanzapine or depot haloperidol. Long-acting risperidone appears to represent a favourable first-line strategy for patients with schizophrenia requiring long-term maintenance treatment.     

Cost effectiveness of increasing the dose intensity of chemotherapy with granulocyte colony-stimulating factor in small-cell lung cancer: based on data from the Medical Research Council LU19 trial

BACKGROUND: The use of granulocyte colony-stimulating factor (G-CSF) can enable dose intensification of chemotherapy in small-cell lung cancer (SCLC). However, given its acquisition cost, it is important to assess its cost effectiveness within a resource-constrained health service. OBJECTIVE: To assess the cost effectiveness, from the UK NHS perspective, of G-CSF given in addition to doxorubicin, cyclophosphamide and etoposide (ACE) versus ACE alone in the management of SCLC. METHODS: Using data from a UK Medical Research Council trial (LU19) to assess chemotherapy dose intensification in patients with previously untreated SCLC of any disease extent, a retrospective cost-effectiveness analysis was undertaken. Resource use data, including hospitalisations and non-protocol cancer treatments, were collected during the first 6-month treatment phase of the trial. Mean costs ( pound, 2003 values) of managing patients in the two arms of the trial were calculated. Mean survival duration was calculated for the two groups using patient-specific follow-up data collected in the trial. Incremental cost-effectiveness analysis was undertaken, and uncertainty in cost effectiveness was expressed using cost-effectiveness acceptability curves. RESULTS: The use of G-CSF in addition to ACE chemotherapy is more costly ( 4647 pounds) but results in longer mean survival duration (0.20 years; 0.18 years when discounted). This generates an incremental cost per additional life-year of 25,816 pounds for ACE + G-CSF therapy. The probability of the addition of G-CSF being cost effective, if decision makers are willing to pay 30,000 pounds for an additional life-year, is 0.57. Secondary analysis suggests that cost effectiveness is likely to be sensitive to assumptions about the health-related quality of life (HR-QOL) experienced by patients. CONCLUSION: Based on data collected in the LU19 trial, chemotherapy dose intensification using G-CSF in SCLC adds to health service costs but increases survival duration. Its overall cost effectiveness is likely to be finely balanced.     

A model of the long-term cost effectiveness of scheduled maintenance treatment with infliximab for moderate-to-severe ulcerative colitis

Background Infliximab (IFX) has been shown to be efficacious in moderate‐severe ulcerative colitis (UC). Aim To evaluate the cost‐effectiveness of a scheduled maintenance treatment (SMT) with IFX in moderate‐severe UC patients. Methods A Markov model was constructed to simulate the progression of a cohort of moderate‐severe UC patients treated with IFX (5 mg/kg) SMT. Transitions were estimated from two phase III trials of IFX (ACT I and ACT II). Standard care, comprising immunomodulators and/or corticosteroids was used as a comparator. Two separate treatment strategies were evaluated – continued treatment in IFX responders and continued treatment in IFX patients achieving remission. The dose of IFX was estimated for a 73 kg typical UC patient in the UK. The results were calculated over 10 years using a discount rate of 3.5% for costs and outcomes. The outcome measure was quality‐adjusted life years (QALYs) estimated using EQ‐5D. Sensitivity analyses explored the uncertainty around the results. Results The incremental cost effectiveness ratio (ICER) for IFX was £27 424 in the responder strategy and £19 696 in the remission strategy at 10 years. In sensitivity analysis, the ICER for IFX in the responder strategy ranged from £21 066 to £86 322 and in the remission strategy ranged from £14 728 to £46 765. The model time horizon and patient body weight were important factors affecting results. Conclusion Eight‐week SMT with IFX appears to be a cost‐effective treatment option for adult patients suffering from moderate to severe UC.     

Incremental cost analysis of ambulatory clinic and home-based intravenous therapy for patients with multiple myeloma

BACKGROUND: Patients with multiple myeloma and other forms of cancer receiving pamidronate via intravenous (IV) infusion at the Hamilton Regional Cancer Centre in Hamilton, Ontario, Canada face 2 treatment options: they can have their entire treatment completed at the clinic using traditional IV therapy (e.g. IV bag and pole) or they can have the treatment initiated at the clinic and then return home to complete the treatment utilising a portable and disposable IV therapy device. OBJECTIVE: To perform a cost analysis of these 2 treatment options. PERSPECTIVE: Societal. METHODS AND PATIENTS: Data on all patients with multiple myeloma who attended the Hamilton Regional Cancer Centre for pamidronate therapy from November 1, 1997 to October 31, 1998 were collected from clinic records. As almost all of these patients with multiple myeloma completed their IV therapy at home, comparison to clinic-based therapy was based on derived cost estimates. Cost data, where possible, were acquired from the Hamilton Regional Cancer Centre's records. A sensitivity analysis was also conducted. RESULTS: In the base-case scenario for the study period, the incremental cost of the infusion device and training in Canadian dollars ($Can; 1998 values) for the 48 patients (299 cycles) who had their infusion initiated at the clinic but completed at home was $Can 5.50/cycle ($Can 4,636 for the 299 cycles). If these 48 patients had had their entire infusion at the clinic, the incremental costs of overtime treatment, parking, clinic overheads and lost work or leisure time would have been $Can 68.49/cycle ($Can 20,477 for the 299 cycles). Therefore, shifting treatment from the clinic to the home resulted in net cost savings to society of $Can 52.98/cycle ($Can 15,841 for the 299 cycles). Sensitivity analysis of best- and worst-cost scenarios did not alter the substantive findings although the relative difference between treatment options varied. In the best-case scenario, home treatment was $Can 95.97/cycle ($Can 28,696 for the 299 cycles) less costly than clinic treatment, while in the worst-case scenario, home treatment was $Can 17.19/cycle ($Can 5,141 for the 299 cycles) less costly than clinic treatment. The results also demonstrated that clinic overheads, the cost of a portable and disposable infusion device and the cost of lost work and leisure time had the greatest impact on incremental costs for each treatment option. CONCLUSION: Subject to study limitations, a significant cost advantage was demonstrated through the home-based treatment option for patients with multiple myeloma. Key issues that must be addressed in future evaluations include the precise determination of clinic overheads, the valuation of lost work and/or leisure time and the direct cost of portable and disposable infusion devices.     

Cost effectiveness of esomeprazole compared with omeprazole in the acute treatment of patients with reflux oesophagitis in the UK

BACKGROUND: Clinical studies have demonstrated that esomeprazole is superior to omeprazole for the acute treatment of reflux oesophagitis. OBJECTIVE: To compare the cost effectiveness of esomeprazole 40mg once daily with omeprazole 20mg once daily in patients with reflux oesophagitis. METHODS: Pooled data were used from three 8-week clinical trials comparing the efficacy and safety of esomeprazole 40mg once daily and omeprazole 20mg once daily for the acute treatment of reflux oesophagitis. A simple decision analysis model, using UK direct medical costs, compared the cost effectiveness of the two treatments. Healing probabilities derived from the clinical studies using the Life Table method were used to estimate the effectiveness and cost of treating 100 patients with reflux oesophagitis. Patient management assumptions were based on a clinical management survey involving 25 UK physicians. PERSPECTIVE: UK National Health Service. RESULTS: After 4 weeks' therapy, the Life Table estimated the oesophageal healing rate to be 77.7% in esomeprazole 40mg once-daily recipients (n = 2446), compared with 67.6% in omeprazole 20mg once-daily recipients (n = 2431; p < 0.001). The corresponding values after 8 weeks' treatment were 93.4% and 86.2%, respectively (p < 0.001). The model predicted that when considering healing probabilities over 8 weeks, esomeprazole 40mg once daily produced total direct cost savings of pound1290 (14%) when compared with omeprazole 20mg once daily. When considering the cost of treating patients who had failed treatment (defined as patient not healed as assessed by endoscopy) after 8 weeks, the cost advantage for esomeprazole was even greater. CONCLUSION: Esomeprazole 40mg once daily is cost effective compared with omeprazole 20mg once daily in the acute treatment of patients with reflux oesophagitis; esomeprazole provides greater effectiveness at a lower cost.     

The contribution of observational studies to the knowledge of drug effectiveness in heart failure

AIMS: Randomized controlled trials (RCTs) are the golden standard for the assessment of drug efficacy. Little is known about the add-on value of observational studies in heart failure (HF). We aimed to assess the contribution of observational studies to actual knowledge regarding the effectiveness of angiotensin-converting enzyme inhibitors (ACEI), and beta-blockers (BB) in HF. METHODS: Observational studies that assessed the effectiveness of ACEI and BB in HF were identified by searching Medline, Embase, Cochrane Database (1990-2005) and the bibliographies of published articles. Cohort, case-control and time-series analysis studies were considered for inclusion. Studies with <100 patients and those who did not perform a multivariate analysis were excluded. RESULTS: A total of 23 cohort studies met the inclusion criteria. Studies of ACEI and BB showed a decrease in mortality with drug use in elderly patients with a broad range of ejection fraction (EF), and in those with depressed EF. Additionally, they showed a decrease in mortality in patients with renal insufficiency. The effect of ACEI and BB in HF with preserved EF was not clear, although last evidence suggests a potential benefit. Low-dose ACEI and BB may have beneficial effects. Target doses of ACEI seemed superior to low doses, but there was no clear dose-response relationship. CONCLUSIONS: Observational studies in HF validate the effectiveness of ACEI and BB in populations underrepresented or excluded from RCTs. Observational studies of drug effectiveness provide relevant additional information for clinical practice.     

Cost effectiveness of ibandronate for the prevention of fractures in inflammatory bowel disease-related osteoporosis: cost-utility analysis using a Markov model

BACKGROUND: Osteoporosis is a frequent complication in patients with inflammatory bowel disease. Recent studies have shown bisphosphonates to considerably reduce fracture risk in patients with osteoporosis, and preventing fractures with bisphosphonates has been reported to be cost effective in older populations. However, no studies of the cost effectiveness of these agents in preventing fractures in patients with inflammatory bowel disease are available. OBJECTIVE: To investigate the cost effectiveness of the bisphosphonate ibandronate combined with calcium/colecalciferol ('ibandronate') in patients with osteopenia or osteoporosis due to inflammatory bowel disease in Germany. Treatment strategies used for comparison were sodium fluoride combined with calcium/colecalciferol ('fluoride') and calcium/colecalciferol ('calcium') alone. STUDY DESIGN AND METHODS: A cost-utility analysis was conducted using data from a randomized controlled trial (RCT). Changes in bone mineral density (BMD) were adjusted and predicted for a standardized population receiving each respective treatment. A Markov model was developed, with probabilities of transition to fracture states consisting of BMD-dependent and -independent components. The BMD-dependent component was assessed using predicted change in BMD from the RCT. The independent component captured differences in bone quality and micro-architecture resulting from prevalent fractures or treatment with anti-resorptive drugs.The analysis was conducted for a population with a mean age of the RCT patients (women aged 36 years, men aged 38 years) with osteopenia (T-score about -2.0 at baseline), a population of the same age with osteoporosis (T-score of -3.0 at baseline) and for an older population (both sexes aged 65 years) with osteoporosis (T-score of -3.0). Outcomes were measured as costs per QALY gained from a societal perspective. The treatment duration in the RCT was 42 months. A 5-year period was assumed to follow, during which the treatment effects linearly declined to 0. The simulation time was 10 years.Prices for medication and treatment were presented as year 2004 values; costs and effects were discounted at 5%. To test the robustness of the results, univariate and probabilistic sensitivity analyses (Monte Carlo simulation) were conducted. RESULTS: The calcium strategy dominated the fluoride strategy. When the ibandronate strategy was compared with the calcium strategy, the base-case cost-effectiveness ratios (costs per QALY gained) were between euro 407 375 for an older female population with osteoporosis and euro 6 516 345 for a younger female population with osteopenia. Univariate sensitivity analyses resulted in variations between 4% of base-case results and dominance of calcium. In Monte Carlo simulations, conducted for the various populations, the probability of an ICER of ibandronate below euro 50 000 per QALY was never greater than 20.2%. CONCLUSION: The ibandronate strategy is unlikely to be considered cost effective by decision makers in men or women with characteristics of those in the target population of the RCT, or in older populations with osteoporosis.