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      Real-world persistence and benefit–risk profile of fingolimod over 36 months in Germany

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          Abstract

          Objective

          To assess the long-term real-world benefit–risk profile of fingolimod in patients with relapsing MS in Germany.

          Methods

          This analysis used data from the noninterventional real-world study, Post-Authorization Non-interventional German sAfety study of GilEnyA (PANGAEA), to assess prospectively the persistence, effectiveness, and safety of fingolimod over 36 months (±90 days) in Germany. For inclusion in the effectiveness analysis (n = 2,537), patients were required to have received fingolimod for the first time in PANGAEA, to have at least 12 months of data, and to have completed each 12-month follow-up period. For the safety analysis (n = 3,266), patients were additionally allowed to have received fingolimod before enrollment.

          Results

          At baseline, 94.7% of patients in the effectiveness analysis had received a previous disease-modifying therapy. After 36 months, 70.4% of patients were still receiving fingolimod. Over this period, annualized relapse rates decreased to 0.265 (95% CI: 0.244–0.286) from 1.79 (95% CI: 1.75–1.83), and mean Expanded Disability Status Scale scores remained stable (mean change from baseline: +0.049 [95% CI: −0.015 to +0.114]). In total, 16% of patients had 6-month confirmed disability improvement, 12.5% had 6-month confirmed disability worsening, and 52.4% were free from relapses and 6-month confirmed disability worsening. Adverse events (AEs) and serious AEs were experienced by up to 23.4% and 3.9% of patients, respectively, during any of the 12-month follow-up periods. The frequency and nature of AEs were in line with previous findings.

          Conclusions

          Using systematically collected data from PANGAEA, this analysis demonstrates the sustained effectiveness, high persistence, and manageable safety profile of fingolimod over 36 months.

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          Most cited references25

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          Optimizing treatment success in multiple sclerosis

          Despite important advances in the treatment of multiple sclerosis (MS) over recent years, the introduction of several disease-modifying therapies (DMTs), the burden of progressive disability and premature mortality associated with the condition remains substantial. This burden, together with the high healthcare and societal costs associated with MS, creates a compelling case for early treatment optimization with highly efficacious therapies. Often, patients receive several first-line therapies, while more recent and in part more effective treatments are still being introduced only after these have failed. However, with the availability of highly efficacious therapies, a novel treatment strategy has emerged, where the aim is to achieve no evidence of disease activity (NEDA). Achieving NEDA necessitates regular monitoring of relapses, disability and functionality. However, there is only a poor correlation between conventional magnetic resonance imaging measures like T2 hyperintense lesion burden and the level of clinical disability. Hence, MRI-based measures of brain atrophy have emerged in recent years potentially reflecting the magnitude of MS-related neuroaxonal damage. Currently available DMTs differ markedly in their effects on brain atrophy: some, such as fingolimod, have been shown to significantly slow brain volume loss, compared to placebo, whereas others have shown either no, inconsistent, or delayed effects. In addition to regular monitoring, treatment optimization also requires early intervention with efficacious therapies, because accumulating evidence shows that effective intervention during a limited period early in the course of MS is critical for maintaining neurological function and preventing subsequent disability. Together, the advent of new MS therapies and evolving management strategies offer exciting new opportunities to optimize treatment outcomes.
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            Treatment decisions in multiple sclerosis — insights from real-world observational studies

            Real-world observational studies have the potential to answer questions about multiple sclerosis (MS) treatment that randomized controlled trials cannot. Trojano and colleagues discuss the pitfalls and necessary safeguards in observational studies, and the insights that such studies have provided into treatment decisions for patients with MS.
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              The importance of collecting structured clinical information on multiple sclerosis

              Background Randomized controlled trials (RCTs) are the ‘gold standard’ in the generation of drug efficacy and safety evidence. However, enrolment criteria, timelines and atypical comparators of RCTs limit their relevance to standard clinical practice. Discussion Real-world data (RWD) provide longitudinal information on the comparative effectiveness and tolerability of drugs, as well as their impact on resource use, medical costs, and pharmacoeconomic and patient-reported outcomes. This is particularly important in multiple sclerosis (MS), where economic treatment benefits of long-term disability reduction are a cornerstone of payer drug approvals – these are typically not examined in the RCT itself but modelled using real-world datasets. Importantly, surrogate markers used in RCTs to predict the prevention of long-term disability progression can only truly be assessed through RWD methodologies. Summary We discuss the differences between RCTs and RWD studies, describe how RWD complements the evidence base from RCTs in MS, summarize the different methods of RWD collection, and explain the importance of structuring data analysis to avoid bias. Guidance on performing and identifying high-quality real-world evidence studies is also provided.
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                Author and article information

                Contributors
                Journal
                Neurol Neuroimmunol Neuroinflamm
                Neurol Neuroimmunol Neuroinflamm
                nnn
                NEURIMMINFL
                Neurology® Neuroimmunology & Neuroinflammation
                Lippincott Williams & Wilkins (Hagerstown, MD )
                2332-7812
                07 March 2019
                May 2019
                07 March 2019
                : 6
                : 3
                : e548
                Affiliations
                From the Center of Clinical Neuroscience (T.Z.), Neurological University Clinic Carl Gustav Carus, University of Technology, Dresden; NeuroPoint Patient Academy and Neurological Practice (M.L.), Ulm; Department of Neurology (B.T.), Center of Neuroimmunology, Philipps-University, Marburg; Bonn Neurological Practice (S.S.); Neurological Practice (H.A.), Munich; Department of Neurology (L.K.), University Hospital Münster, Münster; Centre for Multiple Sclerosis (J.H.), Jewish Hospital Berlin; Kassel and Vellmar Neurology Practice (C.L.), Vellmar, Germany; Oxford PharmaGenesis (C.A-M.C.); Oxford PharmaGenesis (J.A.F.), United Kingdom; and Novartis Pharma GmbH (C.C.), Nuremberg, Germany.
                Author notes
                Correspondence Prof. Ziemssen Tjalf.Ziemssen@ 123456uniklinikum-dresden.de

                Funding information and disclosures are provided at the end of the article. Full disclosure form information provided by the authors is available with the full text of this article at Neurology.org/NN.

                The Article Processing Charge was funded by the authors.

                Article
                NEURIMMINFL2018017921
                10.1212/NXI.0000000000000548
                6410931
                30882022
                5eceb8ac-7e80-4f79-8f4e-a76295999219
                Copyright © 2019 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.

                This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

                History
                : 27 July 2018
                : 08 January 2019
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