[Efficacy and effectiveness of biologic therapy in inflammatory bowel disease. EFIFECT study]

Abstract

Introduction: Randomized controlled trials provide the best scientific evidence for the efficacy of biological drugs in inflammatory bowel disease (IBD). However, findings obtained from these trials might not be reproducible in clinical practice. This study aimed to estimate the percentage of patients with IBD treated with biologics who would have been eligible for randomized controlled trials, and to compare the theoretical efficacy of biological drugs with their effectiveness in clinical practice.

Methods: We performed a retrospective multicenter study in 375 patients with IBD treated with anti-TNF agents and followed-up for 1 year. The eligibility criteria for the trial were taken from the ACCENT, SONIC, ACT, CLASSIC and CHARM trials. Eligible patients were included in a second analysis to compare results in clinical practice versus those hypothetically obtained if the patient had been included in a trial.

Results: Only 45.6% of 375 patients would have been eligible for pivotal trials. One-year clinical benefit (remission or response) was similar for eligible and non-eligible cohorts (68.4% vs. 68.6%, P=.608). The clinical benefit was greater for current clinical practice than for a hypothetical trial situation (68.4% vs. 44.4%, P<.001) in eligible patients.

Conclusion: More than half of patients with IBD treated with biologic drugs would not be represented in pivotal trials. The effectiveness of anti-TNF drugs in clinical practice exceeds their theoretical efficacy.

Keywords: Adalimumab; Efectividad; Effectiveness; Efficacy; Eficacia; Enfermedad inflamatoria intestinal; Inflammatory bowel disease; Infliximab.