Single-arm trials (SAT) using external comparator (EC) data are increasingly submitted for Health Technology Assessment (HTA). We compared the submission rate, acceptance rate and success factors for real world data (RWD) compared to other evidence as ECs. Furthermore, we evaluated whether the assessment was positive.

To perform a 1, 3, and 5-year, multiple line, cost analysis of four triplet therapies used in the treatment of relapsed or refractory multiple myeloma (rrMM) patients from the perspective of the German statutory health insurance (GKV). The analysis compared costs of therapies in the second and three subsequent treatment lines over a 1, 3, and 5-year period. The analysis considered triplet therapies including ixazomib plus lenalidomide plus dexamethasone (IRd), elotuzumab plus lenalidomide plus dexamethasone (ERd), daratumumab plus lenalidomide plus dexamethasone (DRd), carfilzomib plus lenalidomide plus dexamethasone (KRd). The treatment duration for each regimen was estimated from modeled parametric progression-free survival curves (digitized and derived from clinical trials) where censored patients were removed. The probability of progression after second line treatment initiation was used to inform the average post-progression therapy cost for each regimen. In the third line, three drugs were considered including daratumumab plus bortezomib plus dexamethasone (DVd), panobinostat plus bortezomib plus dexamethasone (VFd), and pomalidomide plus dexamethasone (Pd). All seven drugs were assessed in the fourth and fifth line. Although the total cost across all four triplet therapies was substantial in second line, it made up about two-thirds of total costs when subsequent treatment lines were included. This cost analysis can be used to help inform decision makers and payers regarding costs of triplet regimens for the treatment of rrMM. The study shows that post-progression costs for specific regimens are substantial and should not be ignored.

Background: Patients with non-valvular atrial fibrillation (NVAF) have a five times higher stroke risk. For more than 50years, vitamin K antagonists (VKAs) have been the primary medication for stroke prevention. Apixaban, a non-vitamin K oral anticoagulant (NOAC), has demonstrated better efficacy and safety characteristics than the VKA warfarin in the ARISTOTLE trial. This study aims to quantify the potential societal effects of using apixaban instead of VKA in the German NVAF population from 2017 to 2030. Methods: Using an existing Markov model and a dynamic population approach, we modelled the health benefits of apixaban in patients with NVAF compared to VKA therapy in the German population from 2017 to 2030. Results: The results represent the extrapolated direct long-term health benefits of apixaban over VKA therapy for the German NVAF population. From 2017 until 2030, the use of apixaban instead of a VKA could avoid 52,185 major clinical events. This includes 15,383 non-fatal strokes or SEs, 22,483 non-fatal major bleeds, and 14,319 all-cause deaths, which correspond to 109,887 life years gained. Conclusion: This study demonstrated that using apixaban instead of VKA for stroke prevention can lead to considerable reduction in cardiovascular events.

In the recent years, the European Medicines Agency has approved several non-vitamin K oral anticoagulants (NOACs) for the treatment of venous thromboembolism (VTE). We aimed to assess the demographic and clinical characteristics as well as the current anticoagulation treatment patterns of German patients with VTE. We conducted a retrospective database analysis of German claims data from the InGef research database. The study population included patients with a new diagnosis of VTE who initiated anticoagulant treatment including parenteral anticoagulants (PACs), phenprocoumon and all NOACs between January 2013 and September 2015. All patients were characterized regarding the type of anticoagulation treatment, VTE type and the setting of care in which VTE was diagnosed. In addition, we assessed demographic and clinical characteristics for each type of anticoagulation treatment. The study population comprised 19,406 VTE patients with a mean age of 60 years and a female proportion of 51%. Overall, 74% were diagnosed with deep vein thrombosis (DVT) and 26% with pulmonary embolism (PE). About 60% of VTE cases developed and were treated in the outpatient setting, 31% developed in the outpatient and were treated in the inpatient setting and 9% developed and were treated in the inpatient setting. The majority DVT cases developed and were treated in outpatient setting (77.4%) while PE mostly occurred in the outpatient setting and was treated in hospital (71%). DVT cases were most frequently treated with PACs only (43%) while PE patients most often received rivaroxaban (39%). On average, patients treated with apixaban and phenprocoumon were 5 years older and suffered from more comorbidities than initiators of other types of anticoagulation treatment. Demographic and clinical characteristics of initiators of individual types of anticoagulation treatments with VTE differed and the choice of anticoagulation treatment varied by type of VTE and setting of care.