Dapagliflozin in combination with insulin was estimated to be a cost-effective treatment option for patients with T2DM whose insulin treatment regimen does not provide adequate glycaemic control in a Dutch healthcare setting.

*On behalf of the German KIMS investigators; †On behalf of the Dutch KIMS investigators; ‡On behalf of the Swedish KIMS investigators.(Correspondence should be addressed to B Saller; EndoScience Endokrinologie Service GmbH, Thalkirchner Str. 1, 80337 Munich, Germany; Email: saller@endoscience.de) AbstractObjective: This study set out to determine the change in quality of life (QoL) and healthcare utilization during 2 years of growth hormone (GH) replacement therapy in adults with GH deficiency. Data were compared from three European countries. Design: Analysis was made from KIMS, the Pfizer International Metabolic Database on adult GH deficiency. Methods: QoL and healthcare utilization were measured at baseline and after 1 and 2 years of GH replacement in patient cohorts from Sweden (n Z302), The Netherlands (n Z103) and Germany (n Z98). QoL was assessed by the QoL-Assessment in Growth Hormone Deficient Adults (QoL-AGHDA) questionnaire, and the KIMS Patient Life Situation Form was used to evaluate healthcare utilization. Results: QoL improved significantly (P ! 0.0001) and comparably in all three cohorts. The improvement was seen during the first year of treatment and QoL remained improved during the second year. The number of days in hospital was reduced by 83% (P ! 0.0001) during GH replacement. There were no country-specific differences either at baseline or during follow-up. The same was true for the number of days of sick leave (reduction of 63%; P Z 0.0004). Significant reductions were recorded in the number of doctor visits in each of the three cohorts after 2 years of GH replacement (P ! 0.05). Conclusions: This study provides a detailed comparative analysis of GH replacement therapy in GHD patients in three European countries. Despite some differences in treatment strategies, the beneficial effects on QoL, patient-reported outcomes and healthcare utilization are essentially similar in the healthcare environment of Western European countries.European Journal of Endocrinology 154 843-850

Transferability of a model-based UK-study in three steps proved to be an efficient method to provide an early indication of the cost-effectiveness of trastuzumab and has led to the provisional reimbursement of the treatment.

No abstract

Although differences in real-world and trial population were found, using real-world data as well as results from long-term trial follow-up showed favourable ICERs for rituximab maintenance. Nevertheless, results showed that caution is required with data synthesis, interpretation and generalisability of results. As different scenarios provide answers to different questions, we recommend healthcare decision-makers to recognise the importance of calculating several cost-effectiveness scenarios.

BackgroundStroke prevention is the main goal of treating patients with atrial fibrillation (AF). Vitamin-K antagonists (VKAs) present an effective treatment in stroke prevention, however, the risk of bleeding and the requirement for regular coagulation monitoring are limiting their use. Apixaban is a novel oral anticoagulant associated with significantly lower hazard rates for stroke, major bleedings and treatment discontinuations, compared to VKAs.ObjectiveTo estimate the cost-effectiveness of apixaban compared to VKAs in non-valvular AF patients in the Netherlands.MethodsPreviously published lifetime Markov model using efficacy data from the ARISTOTLE and the AVERROES trial was modified to reflect the use of oral anticoagulants in the Netherlands. Dutch specific costs, baseline population stroke risk and coagulation monitoring levels were incorporated. Univariate, probabilistic sensitivity and scenario analyses on the impact of different coagulation monitoring levels were performed on the incremental cost-effectiveness ratio (ICER).ResultsTreatment with apixaban compared to VKAs resulted in an ICER of €10,576 per quality adjusted life year (QALY). Those findings correspond with lower number of strokes and bleedings associated with the use of apixaban compared to VKAs. Univariate sensitivity analyses revealed model sensitivity to the absolute stroke risk with apixaban and treatment discontinuations risks with apixaban and VKAs. The probability that apixaban is cost-effective at a willingness-to-pay threshold of €20,000/QALY was 68%. Results of the scenario analyses on the impact of different coagulation monitoring levels were quite robust.ConclusionsIn patients with non-valvular AF, apixaban is likely to be a cost-effective alternative to VKAs in the Netherlands.

The data from these focus groups should be viewed in light that the patients participating were all well enough to do so. Needs of people with more advanced disease (and their carers) may be different. Conclusions: This study contributes to increased understandings of the patient experience, and that of informal carers, to assist in the provision of information for decision making and in supporting patients in coping with NSCLC. These findings will also contribute to understandings of the experiences of NSCLC patients and carers, compared to those with other cancers. Care should be taken in ensuring that people with NSCLC do not "fall through the cracks" of treatment services and are able to find the support they require.

4277 OBJECTIVES: Effectiveness of rituximab maintenance treatment among patients with relapsed follicular lymphoma (FL) has been established in a randomised setting (EORTC20981 trial). Although the golden standard, results might not be generalisable to unselected real-world patient populations. Outcomes research was conducted to calculate real-world cost-effectiveness of second line rituximab maintenance compared to observation in FL patients in the Netherlands. METHODS: Two Dutch population-based registries, PHAROS and HemoBase respectively, collected information on patient characteristics, treatment, and resource utilisation on patients diagnosed with FL in the past 20 years. Patients responding to second line chemotherapy were included in the observation or maintenance group. A Markov model was applied to calculate real-world cost-effectiveness. RESULTS: Although 735 FL patients entered the registries, only 57 and 49 patients were eligible and included in the observation and maintenance group, respectively. Median age was 61 years and similar in both groups. Differences between the groups were found for second line treatment with rituximab (77% and 92% in the observation and maintenance group p<0.05) and years since diagnosis (p<0.01). Besides, the proportion of people with a partial response was higher in the observation group (60%) compared to the maintenance group (33%). The maintenance group showed higher 3-year overall survival (75% versus 63%) and longer time till next treatment (p<0.01). Compared to the trial, our real-world population was older and fewer patients were diagnosed with stage IV FL. Real-world prescription of rituximab was comparable to the trial although cycles were prescribed more frequent. Daily practice data combined with trial efficacy data resulted in cost-effectiveness ratios between € 3,614 and € 5,246 per life year gained and €3,555 and € 5,156 per quality-adjusted life year. CONCLUSIONS: Real-world FL patients were not identical to trial patients emphasising the importance of studying real-world data. Nevertheless, these data also suggest that rituximab is an effective treatment in daily life. However, analysing non-randomised groups is challenging. Careful interpretation is required since observed dissimilarities between the groups suggested the representation of different types of patients. Nevertheless, real-world cost-effectiveness for second line rituximab maintenance was favourable compared to other haematological treatments. Disclosures: Pompen: Roche Netherlands: Employment. Révil:Roche Netherlands: Employment.