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Do disparities between populations in randomized controlled trials and the real world lead to differences in

Emma Gray1, Suzanne Norris1,2, Susanne Schmitz3

  • 1School of Medicine, Trinity College Dublin, Dublin, Ireland.

Journal of Comparative Effectiveness Research
|November 18, 2016
PubMed
Abstract

Insights

Direct-acting antiviral treatment for hepatitis C virus (HCV) shows different results in clinical trials versus real-world studies. Better patient representation in trials is needed for generalizable outcomes.

Area of Science:

  • Hepatology
  • Infectious Diseases
  • Clinical Research Methodology

Background:

  • Direct-acting antiviral (DAA) treatments have revolutionized hepatitis C virus (HCV) management.
  • Discrepancies exist between efficacy observed in randomized controlled trials (RCTs) and effectiveness in real-world observational studies.

Purpose of the Study:

  • To systematically review and identify reasons for the gap between efficacy and effectiveness of DAA regimens for genotype 1 HCV.
  • To compare outcomes reported in RCTs versus observational studies.

Main Methods:

  • Systematic review conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines.
  • Analysis of baseline demographics and sustained virological response (SVR) rates.

Main Results:

  • Statistically significant differences (p < 0.05) were found in baseline demographics between RCTs and observational studies.
  • Significant differences in sustained virological response rates were also observed between study types.
  • These disparities suggest varying patient populations and treatment contexts.

Conclusions:

  • RCT findings for DAA treatment efficacy may not fully generalize to real-world clinical settings.
  • Increased inclusion of diverse patient populations, representative of those receiving treatment, is crucial for RCTs.
  • Future research should focus on bridging the gap between trial efficacy and real-world effectiveness for HCV treatment.
Keywords:
direct-acting antiviral agentseffectivenessefficacyhepatitis Cobservational studiesrandomized controlled trialssystematic review

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