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.

Keywords:
Multiple sclerosis, Real-world evidence, Real-world data, Randomised controlled trials, Registries, Pharmacoeconomics


Autoři: Tjalf Ziemssen 1*;  Jan Hillert 2;  Helmut Butzkueven 3
Působiště autorů: Center of Clinical Neuroscience, Department of Neurology, MS Center Dresden, Center of Clinical Neuroscience, University Hospital Carl Gustav Carus, Dresden University of Technology, Fetscherstr. 74, 01 07 Dresden, Germany. 1;  Department of Clinical Neuroscience and Center for Molecular Medicine, Karolinska Institute, Stockholm, Sweden. 2;  Department of Neurology, Royal Melbourne Hospital, Victoria, Australia. 3
Vyšlo v časopise: BMC Medicine 2016, 14:81
Kategorie: Opinion
prolekare.web.journal.doi_sk: 10.1186/s12916-016-0627-1

© 2016 The Author(s). Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
The electronic version of this article is the complete one and can be found online at: https://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-016-0627-1

Souhrn

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.

Keywords:
Multiple sclerosis, Real-world evidence, Real-world data, Randomised controlled trials, Registries, Pharmacoeconomics


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