Cost comparison by treatment arm and center-level variations in cost and inpatient days on the phase III high-risk B acute lymphoblastic leukemia trial AALL0232.

The Children's Oncology Group (COG) develops and implements multi-institutional clinical trials with the primary goal of assessing the efficacy and safety profile of treatment regimens for various pediatric cancers. However, the monetary costs of treatment regimens are not measured. AALL0232 was a COG randomized phase III trial for children with acute lymphoblastic leukemia that found that dexamethasone (DEX) was a more effective glucocorticoid than prednisone (PRED) in patients younger than 10 years, but PRED was equally effective and less toxic in older patients. In addition, high-dose methotrexate (HD-MTX) led to better survival than escalating doses of methotrexate (C-MTX). Cost data from the Pediatric Health Information System database were merged with clinical data from the COG AALL0232 trial. Total and component costs were compared between treatment arms and across hospitals. Inpatient costs were higher in the HD-MTX and DEX arms when compared to the C-MTX and PRED arms at the end of therapy. There was no difference in cost between these arms at last follow-up. Considerable variation in total costs existed across centers to deliver the same therapy that was driven by differences in inpatient days and pharmacy costs. The more effective regimens were found to be more expensive during therapy but were ultimately cost-neutral in longer term follow-up. The variations in cost across centers suggest an opportunity to standardize resource utilization for patients receiving similar therapies, which could translate into reduced healthcare expenditures.

Cost effectiveness of pomalidomide in patients with relapsed and refractory multiple myeloma in Sweden.

BACKGROUND: Multiple myeloma (MM) patients who have progressed following treatment with both bortezomib and lenalidomide have a poor prognosis. In this late stage, other effective alternatives are limited, and patients in Sweden are often left with best supportive care. Pomalidomide is a new anti-angiogenic and immunomodulatory drug for the treatment of MM. Our objective was to evaluate the cost effectiveness of pomalidomide as an add-on to best supportive care in patients with relapsed and refractory MM in Sweden. MATERIAL AND METHODS: We developed a health-economic discrete event simulation model of a patient's course through stable disease and progressive disease, until death. It estimates life expectancy, quality-adjusted life years (QALYs) and costs from a societal perspective. Effectiveness data and utilities were taken from the MM-003 trial comparing pomalidomide plus low-dose dexamethasone with high-dose dexamethasone (HIDEX). Cost data were taken from official Swedish price lists, government sources and literature. RESULTS: The model estimates that, if a patient is treated with HIDEX, life expectancy is 1.12 years and the total cost is SEK 179 976 (€19 100), mainly indirect costs. With pomalidomide plus low-dose dexamethasone, life expectancy is 2.33 years, with a total cost of SEK 767 064 (€81 500), mainly in drug and indirect costs. Compared to HIDEX, pomalidomide treatment gives a QALY gain of 0.7351 and an incremental cost of SEK 587 088 (€62 400) consisting of increased drug costs (59%), incremental indirect costs (33%) and other healthcare costs (8%). The incremental cost-effectiveness ratio is SEK 798 613 (€84 900) per QALY gained. CONCLUSION: In a model of late-stage MM patients with a poor prognosis in the Swedish setting, pomalidomide is associated with a relatively high incremental cost per QALY gained. This model was accepted by the national Swedish reimbursement authority TLV, and pomalidomide was granted reimbursement in Sweden.

A cost threshold analysis of ciprofloxacin-dexamethasone versus ofloxacin for acute otitis media in pediatric patients with tympanostomy tubes.

OBJECTIVE: The aim of this study was to determine the cost-effectiveness hreshold of a ciprofloxacin 0.3% and dexamethasone 0.1% (CD) otic suspension relative to olfloxacin otic solution (OFX) for the treatment of acute otitis media in pediatric patients with tympanostomy tubes (AOMT). METHODS: This study used a decision-analytic model to simulate the costs and consequences of the ototopical treatment of AOMT. The AOMT model consisted of 3 tiers of antimicrobial therapy. Each successive tier represented the repeat treatment of clinical failures from the preceding tier. Patients were modeled for treatment until cured or until third-tier therapy was complete, at which time patients were considered cured. First-tier therapy modeled a comparison of CD and OFX using efficacy rates taken from a randomized clinical trial with a population of 599 patients. Second-tier therapy modeled the use of amoxicillin and clavulanic acid using an efficacy rate taken from the medical literature. Third-tier therapy was modeled as being pathogen specific and could follow 1 of 3 possible clinical pathways: (1) PO fluconazole, (2) IM ceftriaxone, or (3) IV antibiotics administered in a hospital setting. Third-tier therapeutic pathway probabilities were based on the microbiologic spectrum of the treatment failures from the clinical trial. Cost information (in year-2003 US dollars) was taken from accepted cost reference sources and presented from the perspective of a third-party payer. The economic outcome of interest was the cost-effectiveness threshold of CD relative to OFX. RESULTS: Given the model parameters, CD had a cost-effectiveness threshold value of 4.5 times the wholesale acquisition cost of OFX. Based on actual cost, first-tier CD therapy was more cost-effective than OFX up to a threshold price of US 152.64 dollars. CONCLUSION: In this decision-analytic model, CD was more cost-effective than OFX for AOMT therapy in pediatric patients up to a threshold price of 4.5 times the price of OFX.