Medication non-adherence is a major challenge in the real-life treatment of chronically ill patients. To meet this challenge, adherence interventions with a tailored approach towards patient-specific adherence barriers that are identified with a reliable and practicable questionnaire are needed. The aim of this investigation was to develop and validate such a questionnaire, the “Adherence Barriers Questionnaire (ABQ)”. The German ABQ was developed and tested in 432 patients with atrial fibrillation in a multicentre observational cohort study. Evaluation of the questionnaire included an assessment of internal consistency as well as factor analysis. Criterion-related external validity was assessed by comparing the ABQ score with (1) the degree of self-reported adherence and (2) the time in therapeutic range which describes the anticoagulation quality achieved by patients treated with oral anticoagulation. The final 14-item ABQ scale demonstrated high internal consistency (Cronbach’s alpha = 0.820). Factor analysis identified a three-factor solution, representing intentional adherence barriers with 5 items (31.9% of the variance), medication-/health care system-related adherence barriers with 5 items (13.3% of the variance) and unintentional adherence barriers with 4 items (7.7% of the variance). The ABQ correlated significantly with self-reported non-adherence (Spearman’s rho = 0.438, p < 0.001) as well as time in therapeutic range (Spearman’s rho = − 0.161, p < 0.010). Patients with above-average ABQ scores (increased number and/or strength of existing adherence barriers) were significantly (p < 0.005, Pearson Chi-Square) more likely to have a poor anticoagulation quality (TTR < 60%) than patients with a lower ABQ score (44.6% versus 27.3%). The ABQ is a practicable, reliable and valid instrument for identifying patient-specific barriers to medication-related adherence. Future research is required to examine the ability of the ABQ to identify patient perception/behaviour changes over time which may be important for the measurement of success of adherence interventions.

Objective: To develop and validate a questionnaire for the investigation of non-adherence (NA) barriers in patients receiving intravitreal injection (IVT). Design: Questionnaire development and cross-sectional patient survey combined with a retrospective medical chart review. Participants: German patients with neovascular age-related macular degeneration (nAMD) or diabetic macular edema (DME) receiving anti-vascular endothelial growth factor (anti-VEGF) treatment via IVT. Methods: The previously validated (indications: atrial fibrillation, human immunodeficiency virus, chronic inflammatory lung disease) Adherence Barriers Questionnaire (ABQ) was revised according to specifications of IVT, within the framework of an expert panel. The ABQ-IVT, which initially consisted of 24 items formulated as statements (4-point-Likert-scale ranging from “strongly agree” to “strongly disagree”), was applied in a cross-sectional survey. Evaluation of the questionnaire included an assessment of internal consistency and factor analysis. The occurrence of potential barriers in the patient sample was evaluated using descriptive statistics. To identify patient subpopulations, hierarchical cluster analysis was performed using ABQ-IVT answers as predictors. Due to difficulties in capturing NA as an external criterion, the evaluation of the questionnaire was limited to its internal validity and reliability. Main outcome measures: Patients’ answers to the ABQ-IVT questionnaire and interviews. Results: Of 253 patients, 234 (92%) were able to complete the ABQ-IVT questionnaire. Within the reliability analysis, the ABQ-IVT was reduced to 17 items. The condensed questionnaire demonstrated good internal consistency (Cronbach’s alpha = 0.78), and factor analysis showed no evidence for subscales of the questionnaire. Nearly half of the patients (49%) reported being affected by at least three different barriers. On average, a patient was affected by 3.1 barriers. The most frequently reported barriers were “Challenge due to time commitment of physician visits” (45% of the patients), “Depression” (29%) and “Travel and opportunity costs” (27%). Cluster analysis identified six patient subpopulations, each affected by different sets of barriers and differed regarding their patient characteristics. Conclusions: The ABQ-IVT is a practical and reliable instrument for identifying patient-specific barriers to IVT treatment adherence. In practice, the questionnaire may be useful in assessing whether individual patients are at higher risk of NA due to specific adherence barriers. Aside from better awareness, this allows earlier interventions, though these still need to be validated. Patient subpopulations face different barriers and may, therefore, need distinct preventative care.

The aim of this study was to analyse which factors predict the real-world macro-/microvascular event, hospitalisation and death risk in patients with type 2 diabetes mellitus. Furthermore, we aimed to investigate whether there exists both an under- and over-treatment risk of these patients. We used a German claims/clinical data set covering the years 2010–12. Diabetes-related events were defined as (1) macro-, (2) microvascular events leading to inpatient hospitalisation, (3) other hospitalisations with type 2 diabetes mellitus as main diagnosis, (4) all-cause death and (5) a composite outcome including all event categories 1–4. Factors associated with event risk were analysed by a Kaplan-Meier curve analysis and by multivariable Cox regression models. 229,042 patients with type 2 diabetes mellitus (mean age 70.2 years; mean CCI 6.03) were included. Among factors that increased the event risk were patients’ age, male gender, the adapted Charlson Comorbidity Index, the adapted Diabetes Complication Severity Index, previous events, and number of prescribed chronic medications. For systolic blood pressure/HbA1C, a double-J/U-curve pattern was detected: HbA1C of 6–6.5% (42-48 mmol/mol) and systolic blood pressure of 130-140 mmHg (17.3-18.7kPa) were associated with the lowest event risk, values below/above that range were associated with higher risk. However, this pattern was mainly driven by the death risk and was much less clearly observed for the macrovascular/microvascular/hospitalization risk and for young/less comorbid patients. Both blood pressure and HbA1C seem to be very important treatment targets, especially in comorbid old patients. It is of particular clinical importance that both over- and under-treatment pose a threat to patients with type 2 diabetes mellitus.

Background and objectives: This study describes preferences of German relapsed refractory multiple myeloma (RRMM) patients with novel proteasome inhibitor-based combination treatments. Methods: Patients with a minimum age of 18 years and a diagnosis of RRMM were included. Their preferences were assessed using a discrete choice experiment design, which was developed based on a literature review and two patient focus group discussions. The final discrete choice experiment design consisted of four attributes, namely “therapy application regimen,” “time without progression of disease,” “possibility of grade ≥3 adverse events (AEs) affecting the blood,” and “possibility of grade ≥3 AE heart failure.” Results: Analysis was based on 84 patients (36.9% females, mean age 62.7 years, mean multiple myeloma disease duration 5.5 years). Among the tested attributes, “therapy application regimen” was assigned the highest importance for treatment decisions (38.8%), the second important attribute was “time without progression of disease” (38.7%), followed by “possibility of AE heart failure” (13.9%) and “possibility of AEs affecting the blood” (8.6%). Patients preferred oral intake once a day and once a week over other application modes such as oral intake once a day and once a week plus twice-weekly infusions. Furthermore, they preferred longer disease progression-free time and lower risk of grade ≥3 AEs. The highest overall utility was derived for ixazomib + lenalidomide + dexamethasone (utility: 3.218), compared with lenalidomide + dexamethasone (2.769), and carfilzomib + lenalidomide + dexamethasone (1.928). Conclusion: RRMM patients prefer treatments with an all-oral application, a longer disease-progression-free time, and a lower probability of AEs. If patients face tradeoffs, they accept a lower progression-free time and/or higher AE rates to get an all-oral therapy.

Since persistence to first biological disease modifying anti-rheumatic drugs (bDMARDs) is far from ideal in rheumatoid arthritis (RA) patients, many do receive a second and/or third bDMARD treatment. However, little is known about treatment persistence of the second-line bDMARD and it is specifically unknown whether the mode of action of such a treatment is associated with different persistence rates. We aimed to assess discontinuation-, re-initiation- or continuation-rates of a 2nd bDMARD therapy as well as switching-rates to a third biological DMARD (3rd bDMARD) therapy in RA patients. Analysis was based on German claims data (2010–2013). Patients were included if they had received at least one prescription for an anti-TNF and at least one follow-up prescription of a 2nd bDMARD different from the first anti-TNF. Patient follow-up started on the date of the first prescription for the 2nd bDMARD and lasted for 12 months or until a patient’s death. 2667 RA patients received at least one anti-TNF prescription. Of these, 451 patients received a second bDMARD (340 anti-TNF, mean age 52.6 years; 111 non-anti-TNF, mean age 55.9 years). During the follow-up, 28.8% vs. 11.7% of the 2nd anti-TNF vs. non-anti-TNF patients (p < 0.001) switched to a 3rd bDMARD; 14.1% vs. 19.8% (p = 0.179) discontinued without re-start; 3.8% vs.1.8% (p = 0.387) re-started and 53.5 vs. 66.7% (p < 0.050) continued therapy. Patients in the non-anti-TNF group demonstrated longer drug survival (295 days) than patients in the anti-TNF group (264 days; p = 0.016). Independent variables associated with earlier discontinuation (including re-start) or switch were prescription of an anti-TNF as 2nd bDMARD (HR = 1.512) and a higher comorbidity level (CCI, HR = 1.112), whereas previous painkiller medication (HR = 0.629) was associated with later discontinuation or switch. Only 56.8% of RA patients continued 2nd bDMARD treatment after 12 months; 60% if re-start was included. Non-anti-TNF patients had a higher probability of continuing 2nd bDMARD therapy.