AimTo evaluate the effectiveness of the Austrian Disease Management Program (DMP) ‘Therapie aktiv—Diabetes im Griff’ for patients with type 2 diabetes mellitus concerning patient-relevant outcomes (mortality, myocardial infarction and stroke) and costs.MethodsBased on routine health insurance data, we conducted a population-based retrospective cohort study using a propensity score (PS) matched control group design. The DMP-group consists of participants enrolled in the program during 2008 and 2009 (n = 7181). Out of 208.532 patients with no participation in the DMP up to 2013, PS-matched controls were selected with a matching ratio 1:3. In the PS-model, patient’s characteristics, form of antidiabetic drug therapy, several prescriptions, the number of hospital admissions and days, main discharge diagnoses and costs at baseline were included.ResultsOver a follow-up period of four years, we observed a significantly lower mortality rate in the DMP-group (9.4%) in comparison with the control group (15.9%, p<0.001). The cumulative number of hospital days and mean annual hospital costs were lower for DMP-participants resulting in significantly lower mean annual total costs, amounting to € 8226.80 per patient in the DMP-group and € 9231.10 in the control group respectively (p<0.001).ConclusionsThe evaluation shows a survival benefit and an average reduction of costs for participants in the DMP compared with the control-group. Despite we took great effort to ensure comparable groups, we cannot entirely rule out an influence by residual and unmeasured confounding due to the observational study design and the use of routine data. However, the results indicate that the disease management program implemented in Austria improves quality of care for patients with type 2 diabetes mellitus.

The introduction provisional approval strategies increases the approval of anticancer drugs with ambiguous benefit-risk profiles. Thus, in many instances, there is lacking evidence about overall survival (OS) at the time of marketing authorisation. Our objective was to monitor and characterise therapies with ambiguous benefit-risk profiles and identify any postapproval updates on median OS after at least 3 years of approval by the European Medicines Agency (EMA). Methods: We included all originator anticancer drugs with initially ambiguous benefit-risk profiles that received marketing authorisation by the EMA between January 1, 2009 and May 31, 2015. Our monitoring timeframe was at least 3 years after EMA approval. To identify study updates, the following three sources were included: clinicaltrials.gov, European Public Assessments Reports and PubMed. Results: In total, we identified 102 eligible approval studies. Out of these, a negative difference in median OS or no information was available in 43 (42.2%) instances. During monitoring, 14 updates with accessible positive information on OS could be identified. Including monitoring results, there are still 29 remaining therapies (28.4%) where no or negative information (n Z 24 [23.5%] and n Z 5 [4.9%], respectively) regarding OS is present at least 3 years after EMA approval. Conclusion: One-third of oncology drugs with ambiguous benefit-risk profiles at the time of approval fail to demonstrate a survival benefit even after several years of marketing

Health-related quality of life (HRQoL) is one of the most important patient-relevant study end-points for the direct measurement of the benefit of cancer drugs. Therefore, our aim is to detect cancer indications with no published information on HRQoL at the time of European Medicines Agency (EMA) approval and monitor any reported HRQoL evidence updates after at least three years of follow-up. Methods: We included all cancer indications that were approved by the EMA between January 2009 and October 2015. Our main sources of information were the EMA website, clinicaltrials.gov and a systematic literature search in PubMed. Information on HRQoL outcomes was extracted alongside evidence on median overall survival. Results: In total, we identified 110 indications, of which more than half (n Z 58, 53%) were lacking available information on HRQoL assessments at the time of EMA approval. After a monitoring period of at least three years, 24 updates were identified, resulting in 34 (31%) therapies where information on HRQoL was still not available. For the 76 therapies with reported information on HRQoL, cancer-specific instruments were mostly used (n Z 49/ 76). Regarding cumulative evidence on median overall survival and HRQoL, 33 (n Z 33/ 110, 30%) as well as 15 (n Z 15/110, 14%) cancer drugs were lacking information on both study end-points at the time of approval and after monitoring, respectively. Conclusion: Our results demonstrate that there is an urgent need of routine re-evaluation of reimbursed cancer drugs with initially missing information on major outcomes.

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The absence of a homogeneous, transparent and accessible coding system for diagnosis in Austrian primary care restrained our assessment. However, our study findings illustrate the potential utility and limitations of using claims-based measures to identify low-value care.