C o p y r i g h t I n f o r m a U K L i m i t e d 2 0 0 8 N o t f o r S a l e o r C o m e r c i a l D i s t r i b u t i o n U n a u t h o r i z e d u s e p r o h i b i t e d . A u t h o r i s e d u s e r s c a n d o w n l o a d ,Objectives: Iron chelation treatment (ICT) in -thalassemia major (-TM) patients undergoing blood transfusions can cause low satisfaction, low compliance, with possible negative consequences on treatment success, patients' wellbeing, and costs. The purpose was to estimate the societal burden attributable to -TM in terms of direct and indirect costs, health-related quality-of-life (HRQoL), satisfaction and compliance with ICT in patients undergoing transfusions and ICT.Research design and methods: The naturalistic, multicenter, longitudinal Italian-THAlassemia-Cost-&-Outcomes-Assessment (ITHACA) cost-of-illness study was conducted involving patients of any age, on ICT for at least 3 years, who were enrolled at 8 Italian Thalassemia Care Centers. Costs were estimated from the societal perspective, quantified with tariffs, prices, or net earnings valid in 2006.Results: One-hundred and thirty-seven patients were enrolled (median age ¼ 28.3, 3-48 years, 49.6% male) and retrospectively observed for a median of 11.6 months. Mean direct costs were E1242/patient/month, 55.5% attributable to ICT, 33.2% attributable to transfusions. Relevant quantity and quality of productivity was lost. Both physical and mental components of HRQoL were compromised. Little difficulties remembering to take ICT and positive satisfaction with the perceived effectiveness of therapy were declared, but not good levels of satisfaction with acceptance, perception of side effects and burden of ICT.Conclusions: The management of -TM patients undergoing transfusions and ICT is efficacious, although costly, but overall benefits were not always perceived as optimal by patients. Efforts must be focused to improve patients' acceptance and satisfaction with their therapy; this would contribute to a better compliance and hence an increase in treatment effectiveness and patients' overall wellbeing, with expected improved allocation of human and economic resources.

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Background: Patients with beta Thalassemia Major (TM) require life-long blood transfusions and, to avoid iron overload, Iron Chelation Treatment (ICT), based on 8–12 hour infusions of Deferoxamine (DFO) for 5–7 days/week, and/or Deferiprone (L1) orally administered. ICT regimen often causes low satisfaction and low compliance, with potentially negative consequences on patients’ health, wellbeing and costs. Aims: to investigate direct and indirect costs for TM patients on ICT. Methods: The Italian-THAlassemia-Cost-&-Outcomes-Assessment (ITHACA) was a naturalistic multicentre study conducted to evaluate costs, quality of life, compliance and treatment satisfaction in TM patients undergoing ICT. Patients of any age, on ICT for at least 3 years, were sequentially enrolled at 8 Italian Thalassemia Care Centers. Direct and indirect costs were estimated from retrospective data: those on direct costs were drawn from medical records, referred to a median of 11.6 months before enrolment. As regards as indirect costs, patients completed modified versions of the Health and Labour Questionnaire (HLQ). Questions on indirect costs were referred to a 2 weeks (adults) or a 12 weeks (children) time horizon. Costs were estimated from a societal perspective, using tariffs, prices, average net earning applied in 2006, as appropriate. Results: 137 patients were enrolled and aged a median of 28.3 years (2.7–48.5), 49.6% were male. Overall, mean direct cost was 1,245.33euro/patient/month, with ICT representing 55.4% of costs, followed by transfusions (33.1%), hospitalizations and surgery (3.3%), laboratory and instrumental tests and medical visits (3.1%), concomitant medications (1.6%), non-medical costs (transportation, 3.4%). Patients treated with DFO were 51.5%, 31.6% were treated with L1, 16.9% with DFO+L1. Treatment in patients taking DFO cost on average 552.88euro/patient/month plus 211.20 euro for pump and consumables; patients taking L1 cost 383.25 euro/patient/month, patients taking DFO+L1 cost 918.41 euro. Concerning indirect costs, 116 adults (> 16 years old) completed the HLQ. Sixty-one patients (52.7%) were in paid employment. Twenty-one patients (34.4%) had experienced absenteeism from work in the previous 2 weeks due to ill health. The average absenteeism among patients in paid employment was 0.7 days/week. Indirect costs related to absenteeism were 27.6 euro/patient/week. Fifteen patients (26.6% of patients in paid employment) were hindered by health problems at work. Indirect costs per patient based on hours needed to catch up on tasks neglected due to health problems were 9.4 euro/week. Patients received 1.24 hours of household help/week. Twenty-one children (<15 years old) or their caregivers completed the HLQ children-part. Eighteen children (85.7%) missed schooldays in previous twelve weeks (mean=0.66 days/week) and 6 missed regular activities. Discussion: Transfusions and ICT account for 90% of total costs in TM patients, corresponding to approximately 1,000 euro/patient/month. The disease and the current treatment adopted have a high socio-economic impact on both TM patients and the healthcare system.

Background:Lung cancer is the leading cause of cancer deaths worldwide (1.38 million cancer deaths, 18.2% of the total) and of cancer morbidity (1.61 million new cases, 12.7% of all new cancers). Currently only three second-line non-small-cell lung cancer (NSCLC) pharmacotherapies are licensed in the European Union: the chemotherapies pemetrexed and docetaxel and the epidermal growth factor receptor tyrosine kinase inhibitor erlotinib. These therapy alternatives have shown a comparable efficacy (survival benefit). In the past, cost comparisons showed that erlotinib was less costly compared to docetaxel, which in turn is cheaper than pemetrexed. Nowadays erlotinib (and docetaxel) are still less expensive than pemetrexed; but docetaxel lost patent protection (basic compound patent) at the end of 2010, so docetaxel drug costs have decreased rapidly and the question remains whether erlotinib is still the least costly therapy alternative in second-line NSCLC.Material and methods:Italy was selected for base case analysis to compare the total therapy costs, estimated by combining country-specific drug costs, administration costs, and adverse event costs of erlotinib and generic docetaxel in second-line NSCLC therapy. Sensitivity analyses on central input parameters have been performed.Results:The total costs of treating one patient with erlotinib therapy of €5121 are lower than the docetaxel costs of €6699 for the Italian health care setting. Although the drug costs of erlotinib are higher than generic docetaxel (incremental €3770): the costs of intravenous chemotherapy administration (incremental −€4510), and the costs of adverse event therapy (incremental −€837) lead to higher total therapy costs for docetaxel compared to the epidermal growth factor receptor tyrosine kinase inhibitor therapy erlotinib.Conclusion:The cost comparison findings for Italy show that erlotinib is still the less costly therapy alternative in second-line NSCLC. These results were robust to changes of central input parameters and robust to further potential price decreases for docetaxel.

The evolving field of microbiome research offers an excellent opportunity for biomarker identification, understanding drug metabolization disparities, and improving personalized medicine. However, the complexities of host–microbe ecological interactions hinder clinical transferability. Among other factors, the microbiome is deeply influenced by age and social determinants of health, including environmental factors such as diet and lifestyle conditions. In this article, the bidirectionality of social and host–microorganism interactions in health will be discussed. While the field of microbiome-related personalized medicine evolves, it is clear that social determinants of health should be mitigated. Furthermore, microbiome research exemplifies the need for specific pediatric investigation plans to improve children’s health.