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Background: Invasive fungal infections in neutropenic patients treated for haematological malignancies are associated with a high mortality rate and, therefore, require early treatment. As the diagnosis of invasive fungal infections is difficult, effective antifungal prophylaxis is desirable. So far, fluconazole has been the most commonly used. Objective: To assess the cost effectiveness of itraconazole compared with both fluconazole and no prophylaxis for the prevention of invasive fungal infections in haematological patients, mean age 51 years, in Germany and The Netherlands. Study design: We designed a probabilistic decision model to fully incorporate the uncertainty associated with the risk estimates of acquiring an invasive fungal infection. These risk estimates were extracted from two meta-analyses, evaluating the effectiveness of fluconazole and itraconazole and no prophylaxis. The perspective of the analysis was that of the healthcare sector; only medical costs were taken into account. All costs were reported in €, year 2004 values. Cost effectiveness was expressed as net costs per invasive fungal infection averted. No discounting was performed, as the model followed patients during their neutropenic period, which was assumed to be less than 1 year. Results: According to our probabilistic decision model, the monetary benefits of averted healthcare exceed the costs of itraconazole prophylaxis under baseline assumptions (95% CI: from cost-saving to €5000 per invasive fungal infection averted). Compared with fluconazole, itraconazole is estimated to be both more effective and more economically favourable, with a probability of almost 98%. Conclusions: In specific groups of neutropenic patients treated for haematological malignancies, itraconazole prophylaxis could potentially reduce overall healthcare expenditure, without harming effectiveness, in settings where fluconazole is common practice in the prophylaxis of invasive fungal infections. [ABSTRACT FROM AUTHOR]

Purpose: Targeted cancer therapies (TCTs) are drugs that specifically act on molecular targets within the cancer cell, causing its regression and/or destruction. Although TCTs offer clinically important gains in survival in one of the most challenging therapeutic areas, these gains are followed by considerable increases in health care expenditures. The aim of this study was to identify differences in the recommendations for TCTs in 3 European health care systems (Serbian, Scottish, and Dutch) and to examine the role of pharmacoeconomic (PE) assessments in such recommendations. Methods: A list of currently approved TCTs cited from the European Medicines Agency was crossreferenced with drug reimbursement reports issued by the National Health Insurance Fund for Serbia, the Scottish Medicines Consortium for Scotland, and the National Health Institute for the Netherlands. The following key variables were gathered from the reports: drug indication, registration status, reimbursement status, and outcome of the PE evaluation. Findings: There were 41 TCTs approved by the European Medicines Agency for 70 cancer indications. Of the total number of TCT indications, 20 were reimbursed in Serbia, and 25 are still without a decision from the national agency. The remaining TCT indications (n = 25) are not registered in Serbia. None of the submissions or the PE analyses were publicly available. The Scottish Medicines Consortium positively assessed 26 TCT indications and rejected 30. All appraisals were published, and the majority contained full PE assessments. Finally, the Dutch agency accepted 60 TCT indications and disapproved the use of 1. The majority of reimbursed drugs were exempted from PE evaluation in accordance with 2 recent policies regarding expensive hospital drugs. Implications: In the 3 examined health care systems, the reimbursement status of the TCTs differed significantly. Level of PE application within the TCT evaluation procedures seemed to largely affect the final reimbursement decisions. Although, there are special policies in the Netherlands that enabled fast access for 98% of the TCTs that applied for reimbursement, a clear definition of cost-effectiveness threshold and strict requirements for full cost utility assessments in Scotland led to acceptance of only 46% of the TCT submissions. More precise PE guidelines must still be designed for TCT reimbursement in Serbia. Guidelines must account for specific epidemic and economic conditions of the country and could build on the experiences of Scotland and the Netherlands. [ABSTRACT FROM AUTHOR]

Background: Pharmacoeconomic evaluations of new drug therapies are often required for reimbursement or guidance decisions. However, for orphan drugs, country-specific requirements exist. In the Netherlands, orphan drug developers can be exempted from providing a full pharmacoeconomic evaluation, whereas in Scotland, no such exceptions can be made, although additional modifying factors specific to orphan products may be considered., Objective: The aim of the present work was to identify differences in the outcomes of the recommendations for orphan drugs for rare diseases between 2 European countries: Scotland and the Netherlands., Methods: All orphan drug reports to the Dutch Committee for Pharmaceutical Assistance (Commissie Farma-ceutische Hulp [CFH]) and orphan drugs guidance issued by the Scottish Medicines Consortium (SMC) through May 2009 were reviewed from the respective organizations' Web sites. The following were gathered from the pharmacoeconomic analyses and recommendations for reimbursement in the Netherlands or guidance for drug use in Scotland: drug indication, outcome of pharmacoeconomic evaluation, recommendation, and exact date., Results: Dossiers for 38 orphan drugs were submitted to the CFH; 37 were submitted to the SMC. Only 1 submission to the CFH included a full pharmacoeconomic analysis; all other reports included only a cost analysis. Twenty-four submissions to the SMC were accompanied by a full pharmacoeconomic evaluation; no information could be obtained for 4 drugs. The remaining reports either did not include a cost-effectiveness analysis or were deemed insufficient by the SMC. Forty-three percent (10/23) of the cost-utility analyses submitted to the SMC reported an unfavorable outcome of more than £30,000/ quality-adjusted life-year (QALY) gained; of these, only 2 (20%) reporting incremental cost-utility ratios of £43.717 to £81.000 were granted a restricted positive recommendation. The CFH gave positive recommendations for reimbursement for 36 of 38 submissions (95%). Of the 37 orphan drugs submitted to the SMC, 19 (51%) received a positive recommendation for use. Seventy-three percent (8/11) of submissions to the SMC with an unfavorable cost-effectiveness estimate (ie, more than £30,000 [€34,000 or US $48,000] per QALY gained) received a negative recommendation for reimbursement., Conclusions: Ninety-five percent of orphan drug submissions were approved for reimbursement in the Netherlands, compared with 51% in Scotland, during the period of interest. Moreover, cost-effectiveness or cost-utility analyses were included in 24 of 37 submissions in Scotland, compared with only 1 of 38 in the Netherlands., (Copyright © 2010 Excerpta Medica Inc. All rights reserved.)