[PHARAO study: drug treatment of inflammatory rheumatic diseases : Guideline-conform treatment with biologics follows availability of biosimilars]. / PHARAO-Studie: Arzneimittelversorgung entzündlich rheumatischer Erkrankungen : Leitliniengerechte Versorgung mit Biologika folgt Verfügbarkeit von Biosimilars.

BACKGROUND: With the introduction of tumor necrosis factor (TNF) alpha inhibitors, the treatment of inflammatory rheumatic diseases (IRD) has undergone a fundamental change. Several of the originally high-priced biologics are now accessible as lower cost biosimilars, removing a significant impediment to prescription. OBJECTIVE: The present study investigated whether the availability of biosimilars is associated with an improvement in the care of IRD. Moreover, the subjective acceptance of biosimilars by physicians and patients was investigated and compared with objectifiable parameters. MATERIAL AND METHODS: Pseudonymized claims data of the Bavarian Association of Statutory Health Insurance Physicians from 2014 to 2019 as well as a paper and pencil survey of patients and rheumatologists formed the data basis of the study. RESULTS: During the observation period, the proportion of diagnosed patients receiving drug therapy increased from 38.5% to 43.2%. Also, the care changed in terms of the prescribed agents. Conventional drug therapy declined overall and, in particular, glucocorticoid prescriptions decreased from 39.3% in 2014 to 34.3% in 2019. At the same time, the proportion of targeted treatments increased from 12.3% to 20.4%. The median duration of basic treatment before first-time bDMARD use dropped from 3.15 years in 2014 to 2.17 years in 2019. CONCLUSION: Over the observation period, in which three biosimilars entered the market, the care of patients with IRD improved both quantitatively and qualitatively. The market share of biosimilars increased in parallel with this development. With an overall high acceptance of biosimilars, the assessment of the disease course by physicians and patients indicates a slight subjectively perceived advantage of therapy with originals compared to biosimilars, which, however, is not confirmed when standardized scores are applied. A possible explanation for this might be a nocebo effect, which could be minimized by suitable communication strategies.

Use of prognostic gene expression profiling tests in primary breast cancer treatment: a German real-world patient survey.

Aims: In primary breast cancer, gene expression profiling tests can support adjuvant chemotherapy treatment decisions. Real-world test use in Germany was investigated in an online survey of female breast cancer patients (n = 475). Materials & methods: Relationships between three groups were examined for clinical and statistical relevance: no test indication (n = 353), test indication and tested (n = 65), and test indication but not tested (n = 57). Results: A total of 47% of participants with a test indication were not tested. Test rates increased by 23% from 2012-2018 (49%) to 2019-2021 (60%). A total of 65% of patients without testing received chemotherapy, whereas only 38% of tested patients received chemotherapy. Conclusion: The use of gene expression profiling tests correlates with a real-world chemotherapy reduction. Gene expression profiling testing may improve patient confidence in the decision for or against chemotherapy.

In many cases, breast cancer can be removed by surgery. In addition to surgery, breast cancer patients may also receive chemotherapy; however, chemotherapy is not always useful. A gene expression profiling test can help physicians and patients decide if chemotherapy should be used. In a survey, 475 breast cancer patients in Germany were asked if they received such a test and chemotherapy. A total of 65% of patients who were not tested received chemotherapy compared with 38% of patients who were tested. Patients who received a test also felt more certain about their treatment decision. However, four of ten patients who were diagnosed between 2019 and 2021 and for whom a test would have helped in the treatment decision did not receive a test. Therefore, there is still room to increase the use of gene expression profiling tests for the benefit of breast cancer patients in Germany.

[Estimating the incidence of venous thromboembolism (VTE) using various types of routine data of the German healthcare system]. / Schätzung der Inzidenz venöser Thromboembolien (VTE) anhand verschiedener Routinedaten des Gesundheitswesens in Deutschland.

BACKGROUND: Venous thromboembolism (VTE) mainly manifests as deep vein thrombosis (TVT) or pulmonary embolism (LE), and is the third most common cardiovascular disease worldwide. However, robust evidence on the incidence of VTE in Germany is lacking. OBJECTIVE: Estimation and comparison of the incidence of VTE based on different routine data sources of the German healthcare system. METHODS: Estimates and comparisons of the incidence of VTE, TVT and LE were made using two databases that both covered the inpatient and the outpatient setting; the DaTraV database comprising information of all persons subject to compulsory health insurance, and the Health Risk Institute (HRI) database derived from approximately 70 statutory health insurance funds. In addition, IMS Disease Analyzer, a medical record database comprising information from the outpatient setting, was used as a data source. RESULTS: Patterns of age- and sex-specific VTE incidence estimates were comparable between all databases used. However, estimates based on the medical record database were comparatively high. Analyses of DaTraV data led to a VTE incidence of 0.14%. Use of HRI data yielded comparable results (0.17-0.20%). VTE incidence based on data of the IMS Disease Analyzer was comparatively high (0.32%). DISCUSSION: Results on the VTE incidence based on DaTraV or HRI date are comparable to international evidence, whereas the use of the IMS Disease Analyzer data presumably led to an overestimation due to double-counting of VTE cases. Different types of routine healthcare data sources can therefore lead to very heterogeneous results. Thus, the selection of adequate data sources strongly depends on the study question and the quality of the dataset.

[Involvement of scientific societies in early benefit assessment: Simulated participation or valuable additional input?] / Einbindung wissenschaftlicher Fachgesellschaften in die frühe Nutzenbewertung von Arzneimitteln: simulierte Teilhabe oder wertvolle zusätzliche Information?

BACKGROUND: The German framework of early benefit assessment (EBA) of drugs also provides for the participation of scientific medical societies. The aim of their inclusion is to assure that care providers can critically assess all aspects of the EBA and provide insights into relevant aspects regarding the provision of care. This study systematically reviews the frequency of participation of the scientific medical societies (FGs) and the Drug Commission of the German Medical Association (AkdÄ) within the scope of the EBA. In addition, the positioning of AkdÄ/FG is compared to the Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (G-BA) with a focus on antidiabetic drugs and cancer drugs. METHODS: A literature analysis was performed based on the comprehensive documentation of benefit assessments published by G-BA. All proceedings of antidiabetic drugs and cancer drugs were included, for which a decision was published by August 6, 2015. In addition, statements of FGs or AkdÄ were identified by an exploratory literature review and included in the analysis. The statements considered were assessed with regard to three categories: (1) additional benefit, (2) appropriate comparator (ZVT) and (3) suitability of the endpoints. For each procedure and category, it was assessed whether there was agreement or disagreement between IGWiG/G-BA and AkdÄ/FGs statements. Regarding the additional benefit, a deviating position was further differentiated according to the level of additional benefit (higher/lower). Afterwards, the proportion of favorable and unfavorable positions was calculated, stratified by FGs and AkdÄ and, separately, for proceedings of antidiabetics and cancer drugs. RESULTS: The literature review revealed 41 proceedings of cancer drugs and 21 proceedings of antidiabetic drugs which were included in the analyses. Statements by AkdÄ/FGs were identified in 90 % of the proceedings for antidiabetic drugs and in 98 % of the proceedings for cancer drugs. In general, the AkdÄ was more often in agreement with the IQWiG than with the FGs' positions. In addition, a different position was more frequent in the proceedings concerning antidiabetic drugs than in the proceedings concerning cancer drugs. Furthermore, the G-BA decision was more frequently in line with the AkdÄ position than with the FGs' position, and this applies to both indications. CONCLUSION: There was a high willingness to participate in the commenting procedure of the EBA. At the same time, the analyses revealed partially heterogeneous positions, both between FGs/AkdÄ and IQWiG/G-BA, as well as between FGs and AkdÄ. The results thus emphasize the need for such discussions within the framework of the EBA.

Effects of continuous and intermittent renal replacement therapies among adult patients with acute kidney injury.

Background: Dialysis-dependent acute kidney injury (AKI) can be treated using continuous (CRRT) or intermittent renal replacement therapies (IRRT). Although some studies suggest that CRRT may have advantages over IRRT, study findings are inconsistent. This study assessed differences between CRRT and IRRT regarding important clinical outcomes (such as mortality and renal recovery) and cost-effectiveness. Additionally, ethical aspects that are linked to renal replacement therapies in the intensive care setting are considered. Methods: Systematic searches in MEDLINE, EMBASE, and Cochrane Library including RCTs, observational studies, and cost-effectiveness studies were performed. Results were pooled using a random effects-model. Results: Forty-nine studies were included. Findings show a higher rate of renal recovery among survivors who initially received CRRT as compared with IRRT. This advantage applies to the analysis of all studies with different observation periods (Relative Risk (RR) 1.10; 95% Confidence Interval (CI) [1.05, 1.16]) and to a selection of studies with observation periods of 90 days (RR 1.07; 95% CI [1.04, 1.09]). Regarding observation periods beyond there are no differences when only two identified studies were analyzed. Patients initially receiving CRRT have higher mortality as compared to IRRT (RR 1.17; 95% CI [1.06, 1.28]). This difference is attributable to observational studies and may have been caused by allocation bias since seriously ill patients more often initially receive CRRT instead of IRRT. CRRT do not significantly differ from IRRT with respect to change of mean arterial pressure, hypotensive episodes, hemodynamic instability, and length of stay. Data on cost-effectiveness is inconsistent. Recent analyzes indicate that initial CRRT is cost-effective compared to initial IRRT due to a reduction of the rate of long-term dialysis dependence. As regards a short time horizon, this cost benefit has not been shown. Conclusion: Findings of the conducted assessment show that initial CRRT is associated with higher rates of renal recovery. Potential long-term effects on clinical outcomes for more than three months could not be analyzed and should be investigated in further studies. Economical analyzes indicate that initial CRRT is cost-effective when costs of long-term dialysis dependence are considered. However, transferability of the economic analyzes to the German health care system is limited and the conduction of economical analyzes using national cost data should be considered.

[Rapid Report Hemophilia - Evidence in treatment of hemophilia from the IQWiG's perspective]. / Rapid Report Hämophilie.

The German Institute for Quality and Efficiency in Health Care (IQWiG) conducted a rapid report to assess the therapy of hemophilia patients. Based on a systematic literature search the IQWiG identified 16 studies which show that there is now sufficient information regarding questions that previously lacked data. A benefit assessment of prophylactic and on-demand-treatment concerning different treatment outcomes shows the superiority of the prophylactic therapy regarding major bleedings for all age groups. For the group of youths and adults the analysis shows additional benefits concerning health status and pain within the preceding four weeks. The alignment of guidelines with the identified evidence shows minor correspondence between the guidelines' references and included studies. However, the guidelines' statements primarily correspond with the results of the conducted benefit assessment. CONCLUSION: The IQWiG's rapid report shows prophylactic therapy to be superior to on-demand therapy. These findings represent a clear indication for attending physicians and payers for prophylaxis in patients with severe hemophilia A.

[Dialysis care in Germany - demand, outpatient care and challenges]. / Dialyseversorgung in Deutschland : Bedarf, Angebot und Herausforderungen.

BACKGROUND: Dialysis is the mostly used renal replacement therapy in patients with end-stage renal disease. The aim of the study was to analyze the present dialysis care system and to scrutinize future changes regarding the need of dialysis as well as the system of care. METHODS: The study is based on a structured literature search in Pubmed; selecting relevant studies by predefined criteria. Prevalence of ESRD and the share of nephrologists in outpatient care were modeled until 2020. Guideline-based interviews with experts including a two-round Delphi survey were conducted to identify options for action. RESULTS: The number of dialysis-dependent patients will increase by one fifth from 83,000 in 2013 to 100,000 in 2020 while the share of nephrologists in outpatient care will decrease by 8% simultaneously. Therefore, young nephrologists and team-building in dialysis care need to be promoted. Home therapy should be used more often to cover the increasing need of dialysis in the future. Besides changes in the structures of care, shared-decision-making needs to be strengthened. CONCLUSIONS: The study offers concrete options to strengthen outpatient care by nephrologists (a) and to use home therapy more often (b) in order to provide adequate and appropriate dialysis care until 2020.

Multiple sclerosis in Germany: data analysis of administrative prevalence and healthcare delivery in the statutory health system.

BACKGROUND: Healthcare-utilization data for multiple sclerosis (MS) are scarce in Germany. The Purpose of the study was to analyse administrative prevalence of MS, medication use and type of specialists involved in MS treatment in the outpatient setting in Bavaria. METHODS: Pseudonymized claims data from Bavarian Statutory Health Insurance (SHI)-accredited physicians were used. Administrative prevalence of MS was defined as having ≥1 MS diagnosis (International Classification of Diseases, 10th edition, code G35) documented by a neurologist or psychiatrist, or ≥1 prescription for disease-modifying drugs (DMDs)). The administrative prevalence calculated for Bavaria was projected to Germany. DMD prescription and involvement of different specialities in health care service for MS patients was analysed. RESULTS: Administrative prevalence of MS in Bavaria increased from 0.123% to 0.175% of insured persons between 2005 and 2009; when projected, this yielded ~102,000-143,000 patients with MS in the German population. The percentage of patients receiving ≥1 DMD prescription increased from 45.5% to 50.5%. Patients with MS were mainly treated by neurologists in the ambulatory care setting. CONCLUSIONS: These results provide important information on the administrative prevalence of MS in Bavaria and on healthcare provision for patients, which is relevant for resource planning in the healthcare sector.