No abstract

“Real-world evidence (RWE)” is becoming increasingly important in order to integrate the results of randomized studies into everyday clinical practice. The data collection of RWE is usually derived from large-scale national and international registries, often driven by academic centers. We have developed a digitalized doctor–patient platform called DESTINY (DatabasE-assiSted Therapy decIsioN support sYstem) that is utilized by NeuroTransData (NTD), a network of neurologists and psychiatrists throughout Germany. This platform can be integrated into everyday practice and, as well as being used for scientific evaluations in healthcare research, can also serve as an individual, personalized treatment application. Its various modules allow for a timely identification of side-effects or interactions of treatments, can involve patients via the “My NTC Health Guide” portal, and can collect data of individual disease histories that are integrated into innovative algorithms, e.g., for the prediction of treatment response [currently available for multiple sclerosis (MS), with other indications in the pipeline]. Here, we describe the doctor–patient platform DESTINY for outpatient neurological practices and its contribution to improved treatment success as well as reduction of healthcare costs. Platforms like DESTINY may facilitate the goal of personalized healthcare.

Objective To implement a dynamic data management and control framework that meets the multiple demands of high data quality, rigorous information technology security, and flexibility to continuously incorporate new methodology for a large disease registry. Materials and Methods Guided by relevant sections of the COBIT framework and ISO 27001 standard, we created a data control framework supporting high-quality real-world data (RWD) studies in multiple disease areas. We first mapped and described the entire data journey and identified potential risks for data loss or inconsistencies. Based on this map, we implemented a control framework adhering to best practices and tested its effectiveness through an analysis of random data samples. An internal strategy board was set up to regularly identify and implement potential improvements. Results We herein describe the implementation of a data management and control framework for multiple sclerosis, one disease area in the NeuroTransData (NTD) registry that exemplifies the dynamic needs for high-quality RWD analysis. Regular manual and automated analysis of random data samples at multiple checkpoints guided the development and implementation of the framework and continue to ensure timely identification of potential threats to data accuracy. Discussion and conclusions High-quality RWD, especially those derived from long-term disease registries, are of increasing importance from regulatory and reimbursement perspectives, requiring owners to provide data of comparable quality to clinical trials. The framework presented herein responds to the call for transparency in real-world analyses and allows doctors and patients to experience an immediate benefit of the collected data for individualized optimal care.

ObjectiveTo evaluate the impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany.DesignThis study employs real-world data captured in-time during clinical visits in 67 German neurology outpatient offices of the NeuroTransData (NTD) multiple sclerosis (MS) registry between 1 January 2010 and 30 June 2019, including 237 976 visits of 17 553 patients with RRMS. Adherence and clinical effectiveness parameters were analysed by descriptive statistics, time-to-event analysis overall and by disease-modifying therapies (DMTs) stratified by administration modes (injectable, oral and infusion). Three time periods were compared: 2010–2012, 2013–2015 and 2016–2018.ResultsBetween 2010 and 2018, an increasing proportion of patients with RRMS were treated with DMTs and treatment was initiated sooner after diagnosis of MS. Introduction of oral DMT temporarily induced higher readiness to switch. Comparing the three index periods, there was a continuous decrease of annualised relapse rates, less frequent Expanded Disability Status Scale (EDSS) progression and increasing periods without relapse, EDSS worsening and with stability of no-evidence-of-disease-activity 2 and 3 criteria, lower conversion rates to secondary progressive MS on oral and on injectable DMTs.ConclusionSparked by the availability of new mainly oral DMTs, RRMS treatment effectiveness improved clinically meaningful between 2010 and 2018. As similar effects were seen for injectable and oral DMTs more than for infusions, a better personalised treatment allocation in many patients is likely. These results indicate that there is an overall beneficial effect for the whole patient with MS population as a result of the greater selection of available DMTs, a benefit beyond the head-to-head comparative efficacy, resulting from an increased probability and readiness to individualise MS therapy.

Zusammenfassung Anliegen und Methodik Beschreibung der Basisdaten, der häufigsten Symptome und deren medikamentöser, nichtmedikamentöser und kombinierter symptomatischer Behandlung in einer großen Stichprobe von MS-Patienten, die sich in ambulanter Behandlung der Mitgliedspraxen des NeuroTransData (NTD)-Arztnetzwerkes in Behandlung befinden. Ergebnisse Aktuell sind 21407 Patienten im Register. Das Durchschnittsalter betrug 49,0 ± 13,0 Jahre, der Frauenanteil lag bei 72,3 %, die Krankheitsdauer bei 14,3 ± 8,9 Jahren. Eine schubförmig-remittierende MS (RRMS) lag in 77 % vor, 15 % sekundär-progrediente MS, primär-progrediente MS (PPMS) 5 %. Der durchschnittliche EDSS-Score der Gesamtstichprobe lag bei 2,8 (Range 0,5–8). Fatigue war in allen Subtypen das häufigste Symptom (96 %), gefolgt von der Spastik (31 %), v.a. bei der SPMS (47 %) und PPMS (36 %). Hinsichtlich der medikamentösen, nichtmedikamentösen und kombinierten symptomatischen Behandlung zeigte sich eine große Variationsbreite. Während in der Gesamtpopulation die Spastik in 81 % behandelt wurde, erhalten nur 21 % der Patienten eine Behandlung der Fatigue. Auch Defäkations- und Miktionsstörungen waren in unserer Stichprobe häufig noch unbehandelt (69 % bzw. 56 %). Schlussfolgerungen Aufbau, kontinuierliche Weiterentwicklung und Pflege eines Registers stellen bei komplexen und chronischen Erkrankungen wie der MS ein wichtiges Instrument zur Beurteilung und damit auch zur Verbesserung und zum Vergleich der Behandlungsart und ihrer Defizite dar.Unsere Ergebnisse sind methodisch schwer vergleichbar mit der Stichprobe des MS-Registers der DMSG. Die Fatigue war aber in beiden Registern das häufigste Symptom.