the UK (42%). Of these, the most frequently utilized endpoints were clinical in the US (62.5%) and both clinical and humanistic (20%, respectively) in the UK. Humanistic outcomes were not mentioned in US-based contracts. 25% of US-based contracts also stipulated components of patient compliance, compared to 1 UK-based contract. Conclusions: In an environment of rising prices, VBCs are becoming a cost containment strategy. However to date, only 61 VBCs were noted in the US and UK over 2 decades, which could be driven by lack of contracts and/or transparency in reporting. The lack of contracts could be driven by the challenges of data collection and analysis, which will likely be mitigated by the development of RWE methods for VBCs. More research and transparency is needed on the structure and successes of VBCs and how RWE can improve VBCs.

No abstract

Objectives: Real world sick funds data in Germany were used to reveal the treatment patterns for Diffuse Large B-cell Lymphoma (DLBCL) and to identify unmet medical needs. Methods: In two independent cohorts of German claims data 819 and 189 DLBCL patients were identified and diagnoses, hospitalization, medication and health costs were analyzed for at least two consecutive years between 2013 and 2016. Results: In the first [second] cohort, mean age of DLBCL patients was 60.3 [66.5] years, with two peaks at 50-54 and 70-74 years. Total annual health care costs for DLBCL patients ranged from 25,000 to 60,000 EUR versus 1,259 EUR in healthy insured. Per year, mean 3.2 [3.5] hospitalizations with an average of 31.5 [34.5] hospital days were observed. During the observation period less than half of the DLBCL patients received oncological treatment and only few patients received stem cell transplantation or radiation. Most frequent pharmacological first line treatments were Rituximab (RTX) + CHOP (57%, [56%]) and RTX in combination with Bendamustine (8%, [7%]). In both analyses consistently 84% of patients did not receive further therapies after a first line DLBCL treatment during the limited observation period. Conclusions: Despite limitations in sick fund claims analyses, these databases are a reasonable source for real world evidence (RWE) of rare diseases. Both cohorts were well in agreement and revealed standard treatment pathways consistent with the German treatment guidelines. All DLBCL patients frequently required hospitalization and generated significant costs. A high unmet medical need exists for treatments other than palliative care, especially for a tolerable and effective outpatient therapy in elderly relapsed / refractory DLBCL patients.