Do efficacy results obtained from randomized controlled trials translate to effectiveness data from observational studies for relapsing–remitting multiple sclerosis?

Author:

Verweij Stefan12ORCID,Ahmed Wouter3,Zhou Guiling1,Mavridis Dimitris4,Nikolakopoulos Stavros256,Elferink Andre J.2,Rengerink Katrien Oude2,Bijlsma Maarten J.17,Mol Peter G. M.23,Hak Eelko1

Affiliation:

1. Unit of PharmacoTherapy, Epidemiology and Economics Groningen Research Institute of Pharmacy Groningen The Netherlands

2. Dutch Medicines Evaluation Board Utrecht The Netherlands

3. Department of Clinical Pharmacy and Pharmacology University Medical Center Groningen, University of Groningen Groningen The Netherlands

4. Department of Primary Education University of Ioannina Ioannina Greece

5. Department of Psychology University of Ioannina Ioannina Greece

6. Data Science and Biostatistics, Julius Center for Health Sciences and Primary Care University Medical Center Utrecht Utrecht The Netherlands

7. Laboratory of Population Health Max Planck Institute for Demographic Research Rostock Germany

Abstract

AbstractBackgroundRandomized controlled trials are considered the gold standard in regulatory decision making, as observational studies are known to have important methodological limitations. However, real‐world evidence may be helpful in specific situations. This review investigates how the effect estimates obtained from randomized controlled trials compare to those obtained from observational studies, using drug therapy for relapsing–remitting multiple sclerosis as an example.Study Design and SettingA systematic review of randomized controlled trials and observational studies was conducted. The primary outcome was the annualized relapse rate. Using (network) meta‐analysis together with posterior predictive distributions, the drug‐specific rate ratios from the network of randomized controlled trials were compared with those from the network of observational studies.ResultsEffect estimates from 26 observational studies showed greater magnitudes and were less precise compared to estimates obtained from 21 randomized controlled trials. Twenty of the 28 treatment comparisons between designs had similar rate ratios. Seven inconsistencies in observed rate ratios could be attributed to two specific disease‐modifying therapies.ConclusionIn this case study, estimates from observational studies predominantly agreed with estimates from randomized controlled trials given their posterior predictive distributions. Multiple observational studies together may therefore supplement additional pivotal randomized controlled trials in relapsing–remitting multiple sclerosis, for instance facilitating the extrapolation of trial results to the broader patient population.

Publisher

Wiley

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