Restricted mean survival time in advanced non-small cell lung cancer treated with immune checkpoint inhibitors.

OBJECTIVE: The purpose of this study was to review the effectiveness of immune checkpoint inhibitors (ICIs) in the first-line treatment of advanced non-small cell lung carcinoma with wild-type epidermal grow factor receptor (EGFR) or anaplastic lymphoma kinase. MATERIALS AND METHODS: After a standard literature search, we identified all randomized studies published on this issue. Our first inclusion criterion was the use of pembrolizumab, nivolumab, atezolizumab or durvalumab in the treatment arm versus chemotherapy in the control arm. The second criterion was the availability of information on overall survival at 2 years. The restricted mean survival time (RMST) was used to analyze the survival curves and rank the treatments. RESULTS: From the eligible studies, we selected 5 randomized trials that met our inclusion criteria. These trials studied a total of 11 cohorts of patients in whom the treatment arm received ICI as monotherapy (n=3) or in combination with either chemotherapy (n=2) or other monoclonal antibodies (n=1). All the control groups (n=5) received chemotherapy. Pembrolizumab (alone or in combination) showed improvement in overall survival compared with controls, but with borderline statistical significance. Nivolumab, atezolizumab and durvalumab failed to demonstrate any survival advantage. Overall, the RMSTs provided more conservative results than those previously reported using the hazard ratio. In comparing the values of RMST across treatments, pembrolizumab combined with chemotherapy ranked first. CONCLUSIONS: Our results summarized the efficacy of these treatments and showed that only pembrolizumab can have a role as the first-line treatment of NSCLC. These findings are at variance with those previously reported using the hazard ratio as the outcome measure.

Optimization of resources by drug management: A multicentred web-administered study on the use of ipilimumab in Italy.

OBJECTIVE: In a scenario of new expensive cancer therapies entering the market, strategies of optimisation and cost containment are crucial in oncology care. Better management of drug waste and centralization of drug preparation can be effective strategies to achieve these goals. The aim of this work is to describe the economic management of a high cost anticancer drug (ipilimumab) in some Italian reference centres. METHODS: This was an observational, multicentred study in which economical and clinical data of 21 cancer centres (418 patients) were collected during the enrollment period from February 2013 to August 2014. The follow-up period ended in July 2015. RESULTS: Participants purchased 10.7% more vials of ipilimumab than necessary for compounding. The results were variable among centres, and only five centres had a deviation lower than 5% between the drug purchased and the drug prescribed. Hospitals applying the drug day reached a statistically significant residual of drug effectively used compared to the amount prescribed (P = 0.018). Consequently, the price for treating a model patient was significantly lower in those hospitals (median spare of 7456 euro per patient). CONCLUSIONS: This study demonstrated that the careful management of drug waste and the application of drug-day, through a proper selection of vial and the ability to use the leftover drug, can generate economic savings. However, tailoring the drug stock to clinical need is still an open issue which deserves further analysis.

Burden of stroke in Italy: an economic model highlights savings arising from reduced disability following thrombolysis.

BACKGROUND: The consequences of stroke must be assessed not only in terms of incidence and mortality rates, but also in terms of disability, which may persist long after the acute phase. Thrombolysis, if timely administered, can effectively reduce post-stroke disability. AIMS: The economic model presented herein aims to evaluate, in eligible patients, the effects of alteplase on post-stroke disability and related costs over three-years. METHODS: The economic analysis was developed on the basis of four key components: clinical outcomes from international trials, economic consequences extracted from cost of illness studies, regulatory data from national and international agencies, and national epidemiological data. A population-level model estimated the difference in disability costs between patients treated with standard care versus those receiving thrombolytic therapy within 4×5 h of acute ischemic stroke. The analysis covered 36 months from discharge. RESULTS: Reduced costs related to post-stroke disability were observed in treated patients compared with those receiving standard care (control). The overall savings were €2330×15 per average patient: €1445×81 during the first 18 months, €362×25 between 18 and 24 months, and €522×09 in the 24-36 months period. The overall savings on 3174 Italian treated patients in 2013 were €7 395 907 over three-years. CONCLUSION: Our study reveals that performing thrombolytic therapy in eligible patients improves economic outcomes compared with patients receiving standard care. This model is useful for decision makers, both within and outside of the Italian national context, as a tool to assess the cost-effectiveness of thrombolysis in both short- and long-term period.