BackgroundAcquired thrombotic thrombocytopenic Purpura (aTTP) is a life-threatening ultra-orphan disease with a reported annual incidence between 1.5 and 6.0 cases per million in Europe and mainly affecting otherwise young and healthy adults aged 40 years on average. The goal of this study was to assess the incidence of aTTP in Germany.MethodsA systematic review was performed to determine the published evidence on the aTTP epidemiology in Germany. To obtain additional evidence on the proportion of aTTP cases within the national Thrombotic Microangiopathy (TMA) population a hospital-level study was performed, using a retrospective data collection approach. Diagnosis of aTTP was confirmed if ADAMTS13 level were < 10% and/or the medical records explicitly mentioned aTTP diagnosis. The aggregated hospital data were then projected to the national level using logistic regression techniques.ResultsThe systematic literature search did not provide incidence estimates of aTTP in Germany. Eight centers (≈27% of the top 30 TMA hospitals) delivered data according to a predefined data collection form. On average (year 2014–2016) a total number of 172 aTTP episodes per year was projected (95% confidence interval [95%CI]: 132–212). The majority were newly diagnosed aTTP cases (n = 121; 95%CI: 105–129), and 51 were recurrent aTTP cases (95%CI: 27–84). The average annual projected incidence (year 2014–2016) of aTTP episodes was 2.10 per million inhabitants in Germany (95%CI: 1.60–2.58).ConclusionsThe determined annual incidence of newly diagnosed aTTP cases and the overall annual incidence of aTTP episodes in Germany confirm the ultra-orphan character of aTTP. An external validation against international registries (France, UK and USA) shows that our findings are quite comparable with those international incidence rates.

New knowledge added by this study • The current project provided cost-effectiveness information for erlotinib and gefitinib based on four Asian phase-III clinical trials in non-small-cell lung cancer (NSCLC) patients using a threshold recommended by the World Health Organization.• The cost-effectiveness analysis indicates that erlotinib is cost-effective compared to gefitinib in first-line epidermal growth factor receptor (EGFR)-activating mutation-positive (MuT+) NSCLC patients in Hong Kong. Implications for clinical practice or policy• Erlotinib is efficacious and cost-effective, and hence should be considered a good option for treatment of EGFR MuT+ NSCLC patients.• Being cost-effective, erlotinib should be considered for reimbursement by health care payers in Hong Kong. A B S T R A C TObjective: To compare the effectiveness and costeffectiveness of erlotinib versus gefitinib as first-line treatment of epidermal growth factor receptoractivating mutation-positive non-small-cell lung cancer patients. Design: Indirect treatment comparison and a costeffectiveness assessment. Setting: Hong Kong. Patients: Those having epidermal growth factor receptor-activating mutation-positive non-smallcell lung cancer. Interventions: Erlotinib versus gefitinib use was compared on the basis of four relevant Asian phase-III randomised controlled trials: one for erlotinib (OPTIMAL) and three for gefitinib (IPASS; NEJGSG; WJTOG). The cost-effectiveness assessment model simulates the transition between the health states: progression-free survival, progression, and death over a lifetime horizon. The World Health Organization criterion (incremental cost-effectiveness ratio <3 times of gross domestic product/capita:

Given the increasing clinical and economic burden of obesity, it is of major importance to identify cost-effective approaches for obesity management. Areas covered: This study aims to systematically review and compile an overview of published decision models for health economic assessments (HEA) in obesity, in order to summarize and compare their key characteristics as well as to identify, inform and guide future research. Of the 4,293 abstracts identified, 87 papers met our inclusion criteria. A wide range of different methodological approaches have been identified. Of the 87 papers, 69 (79%) applied unique /distinctive modelling approaches. Expert commentary: This wide range of approaches suggests the need to develop recommendations /minimal requirements for model-based HEA of obesity. In order to reach this long-term goal, further research is required. Valuable future research steps would be to investigate the predictiveness, validity and quality of the identified modelling approaches.

Background Despite growth of robotic surgery, published literature lacks assessment of the cost of ownership (CoO) of a da Vinci robot by surgical service line and the associated benefit such data provides. Methods Based on real‐world data (RWD) from 14 US hospitals and ≈6000 da Vinci robotic cases, CoO was assessed using all relevant fixed and variable cost components, calculated by surgical service line. Results At a representative hospital with an efficient robotic program (n = 424 cases), the weighted average fixed cost per case was $984. Weighted average variable cost per case was $8025 (range: $3325 for Cholecystectomy—multiport, to $16 986 for Rectal Resection). Assessing weighted average by case, main variable cost drivers were non‐da Vinci supplies (49.5%), staff costs (28.6%), and da Vinci supplies (21.9%). Conclusions Case mix, annual robotic case volumes, and cut‐to‐close/patient‐in‐room time by surgical service line represent core variables influencing robotic program CoO, which help drive profitable program management.

This study aims to determine methodological variations in the event simulation approaches of published health economic decision models, in the field of obesity, and to investigate whether their predictiveness and validity were investigated via external event validation techniques, which investigate how well the model reproduces reality. Areas covered: A systematic review identified a total of 87 relevant papers, of which 72 that simulated obesity-associated events were included. Most frequently simulated events were coronary heart disease (≈ 83%), type 2 diabetes (≈ 74%), and stroke (≈ 66%). Only for ten published model-based health economic assessments in obesity an external event validation was performed (14%; 10 of 72), and only for one the predictiveness and validity of the event simulation was investigated in a cohort of obese subjects. Expert commentary: We identified a wide range of obesity related event simulation approaches. Published obesity models lack information on the predictive quality and validity of the applied event simulation approaches. Further work on comparing and validating these event simulation approaches is required to investigate their predictiveness and validity, which will offer guidance future modelling in the field of obesity.