Comparing Real-World Evidence With Clinical Trials in Juvenile Idiopathic Arthritis

To compare the clinical characteristics and outcomes of children with juvenile idiopathic arthritis (JIA) treated with biologic drugs in traditional clinical trials with the real-world data (RWD) in the CARRA Registry.

This was a retrospective observational study. We reviewed data from 3192 children in the CARRA Registry with JIA exposed to adalimumab or canakinumab, 2 historical trials of polyarticular JIA participants exposed to adalimumab (n = 171 and 32), and 1 trial for canakinumab in systemic JIA (n = 43). We made matched and unmatched comparisons of demographic information and the ACR-Pedi response rate based on trial eligibility criteria.

Very few children in the CARRA Registry would have been eligible for the historical clinical trials: < 10% for adalimumab and < 5% for canakinumab. There was a longer duration of follow-up and a wider range of body size in the CARRA Registry compared with the historical clinical trials. When matched on trial inclusion, the ACR-Pedi response rates broadly aligned with those observed in the trials for adalimumab, whereas the sample size for canakinumab limited comparisons for the response rate.

Children encountered in clinical practice had wider ranges of body size and less severe disease at drug initiation. These results underscore that registration trials may be enriched for patients with more severe disease and are not fully representative of the broader JIA population who use these drugs after marketing. Real-world evidence generated from observational data may play an increasingly important role in regulatory decision making, along with the continued need for controlled clinical trials.