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Nervous system conditions and treatments

Session: Poster Session C

(190) Do efficacy results obtained from randomised controlled trials translate to effectiveness data from observational studies for relapsing-remitting multiple sclerosis?

Wednesday, August 28, 2024
8:00 AM – 1:30 PM CEST
Location: Convention Hall II

    Presenting Author(s)

  • Stefan Verweij, MSc

    PhD candidate RWE
    PharmacoTherapy, -Epidemiology and -Economics, Groningen Research Institute of Pharmacy, University of Groningen, 9713 AV Groningen, The Netherlands
    Groningen, Netherlands

    • Co-Author(s)

  • WA

    Wouter Ahmed

    PharmacoTherapy, -Epidemiology and -Economics, Groningen Research Institute of Pharmacy, University of Groningen, 9713 AV Groningen, The Netherlands, Netherlands

  • Guiling Zhou, MSc (she/her/hers)

    PhD student
    PharmacoTherapy, -Epidemiology and -Economics, Groningen Research Institute of Pharmacy, University of Groningen, 9713 AV Groningen, The Netherlands
    Groningen, Netherlands

  • DM

    Dimitris Mavridis

    Department of Primary Education, University of Ioannina, Ioannina, Greece, Greece

  • SN

    Stavros Nikolakopoulos

    Dutch Medicines Evaluation Board, Utrecht, The Netherlands, Greece

  • AE

    Andre Elferink

    Dutch Medicines Evaluation Board, Utrecht, The Netherlands, Netherlands

  • KO

    Katrien Oude Rengerink, PhD

    Dutch Medicines Evaluation Board, Utrecht, The Netherlands, Netherlands

  • MB

    Maarten J. Bijlsma, PhD

    PharmacoTherapy, -Epidemiology and -Economics, Groningen Research Institute of Pharmacy, University of Groningen, 9713 AV Groningen, The Netherlands, Netherlands

  • PM

    Peter G.M. Mol, PhD

    Dutch Medicines Evaluation Board, Utrecht, The Netherlands, Netherlands

  • EH

    Eelko Hak, PhD

    PharmacoTherapy, -Epidemiology and -Economics, Groningen Research Institute of Pharmacy, University of Groningen, 9713 AV Groningen, The Netherlands, Netherlands

Background: Randomised 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, such as those were a randomised controlled trial is deemed unfeasible or unethical, although their effect sizes might differ.

Objectives: This review investigates how the effect estimates obtained from randomised controlled trials compare to those obtained from observational studies, using relapsing-remitting multiple sclerosis as an example.

Methods: A systematic review of randomised controlled trials and observational studies was conducted on relapsing-remitting multiple sclerosis studies. All disease-modifying therapies approved for the European market were included. 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 randomised controlled trials were compared with those from the network of observational studies.

Results: Effect estimates from 26 observational studies showed greater magnitudes and were less precise compared to estimates obtained from 21 randomised 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.

Conclusions: In 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 randomised controlled trials in relapsing-remitting multiple sclerosis, for instance facilitating the extrapolation of trial results to the broader patient population.