Satisfaction and practicality of a prefilled glatiramer acetate pen in relapsing-remitting multiple sclerosis patients.

Aim: Evaluation of practicality and patient satisfaction of a glatiramer acetate (GA) prefilled pen in patients with relapsing-remitting multiple sclerosis (RRMS). Patients & methods: A cross-sectional, multicenter, observational study evaluating patients' experiences with the GA-pen 3 months after its first use by means of self-reporting questionnaires. Primary end point was the proportion of patients who were satisfied with the pen. Results: 80 patients participated in the study. The majority (83.7%) was satisfied with the pen and 95% rated its application as easy or very easy. Conclusion: Most patients were satisfied with the GA-pen and rated its application as easy or very easy. Among the 12 device features, starting the injection without an injection button was considered the most appreciated feature. Improvements in pen functionality and design might allow patients to overcome many difficulties with self-injection, even those leading to nonadherence. But, this hypothesis awaits further validation by real-world follow-up studies.

When patients with relapsing­remitting multiple sclerosis are treated with an injectable multiple sclerosis (MS) medication like glatiramer acetate (GA), doctors and patients have to think about the different methods of administration such as syringe or pen. This study aimed to assess the practicality and patient satisfaction with a prefilled pen containing GA in individuals with relapsing­remitting multiple sclerosis. The study involved 80 patients and used self-reporting questionnaires to evaluate their experiences with the GA pen. The results showed that most of the patients were satisfied with the GA pen and found the application of the pen to be easy or very easy. Starting the injection without the need for an additional button press was particularly well received by patients. These findings suggest that improvements in the functionality and design of the pen may help patients overcome challenges associated with self-injection.

Implementation of a data control framework to ensure confidentiality, integrity, and availability of high-quality real-world data (RWD) in the NeuroTransData (NTD) registry.

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.

Development and Pilot Test of the Registry Evaluation and Quality Standards Tool: An Information Technology-Based Tool to Support and Review Registries.

OBJECTIVES: Health technology assessment (HTA) bodies are increasingly making use of real-world evidence and data. High-quality registries could be an asset for this; nevertheless, there is a lack of specified standards to assess the quality of data in the registry, or the registry itself. The European Network for Health Technology Assessment Joint Action 3 led the work to develop a tool for the evaluation of clinical registries: the "Registry Evaluation and Quality Standards Tool" (REQueST). METHODS: REQueST was developed in 4 steps: (1) A partnership between HTA bodies across Europe drafted the assessment criteria. (2) Multiple rounds of consultation across HTA bodies and the public domain developed an Excel version of REQueST. (3) This version was transformed into a web-based application. (4) An external pilot tested this REQueST tool with SMArtCARE and NeuroTransData registries. RESULTS: Haute Autorité de Santé, the National Institute for Health and Care Excellence, and the Croatian Institute of Public Health led the development of REQueST. Another 4 HTA bodies contributed regularly to development meetings, and all European Network for Health Technology Assessment partners were invited to contribute. Eight methodological, 12 essential, and 3 supplementary criteria were identified. Both pilot registries scored well, fulfilling the requirements for >70% of criteria, with none failed. Feedback by registry holders led to streamlining of the process and clarification of the criteria. CONCLUSIONS: The REQueST tool uses an iterative and collaborative methodology with registry holders. It has the potential to maximize the utility of registry data for decision making by regulatory and HTA bodies and provides a foundation for future research.

Development of Registry Data to Create Interactive Doctor-Patient Platforms for Personalized Patient Care, Taking the Example of the DESTINY System.

"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.

Impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany between 2010 and 2018: real-world data from the German NeuroTransData multiple sclerosis registry.

OBJECTIVE: To evaluate the impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany. DESIGN: This 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. RESULTS: Between 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. CONCLUSION: Sparked 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.

[Data on multiple sclerosis in Germany and their representation in the ambulatory registry NeuroTransData (NTD) network]. / Daten zur Multiplen Sklerose in Deutschland und ihre Abbildung im Register des ambulanten Netzwerkes NeuroTransData (NTD).

AIMS AND METHODOLOGY: Description of basic data, common symptoms and their medical, non-drug and combined symptomatic treatment in a large sample of MS patients undergoing outpatient treatment of the German NeuroTransData (NTD) physician network. RESULTS: Currently there are 21,407 patients in the registry. Average age is 49.0 ± 13.0 years, 72,3 % of them female, average disease duration is 14.3 ± 8,9 years. Relapsing-remitting MS (RRMS) was present in 77 %, secondary-progressive MS (SPMS) 15 %, PPMS 5 %. The mean EDSS score of the total sample was 2.8 (range 0,5-8). Fatigue was the most common symptom in all subtypes (96 %), followed by spasticity (all 31 %, SPMS: 47 %; PPMS 36 %). Regarding symptomatic drug treatment, non-drug treatment and combined treatment, there was a wide range of variation. While spasticity was treated in 81 %, only 21 % of patients with fatigue receiving any form of therapy, Also, fecal and urine incontinence often remained untreated in 69 % resp. 56 % of cases. CONCLUSIONS: Setup, development and maintenance of a registry for a complex and chronic disease like MS represents an instrument to assess and improve patient care in the outpatient setting. Our results are hard to compare with the DMSG-registry, another German, more hospital-based data collection. However, both registries identify fatigue as the most common symptom in MS.