How can trial designs better serve the needs of children and young people with juvenile idiopathic arthritis?

In juvenile idiopathic arthritis we have seen remarkable progress in the number of available licensed biological and small molecule treatments in the past two decades, leading to improved outcomes for patients. Designing clinical trials for these therapeutics is fraught with ethical, legislative, and practical challenges. However, many aspects of current clinical trial design in juvenile idiopathic arthritis do not meet the needs of patients and clinicians. Commonly used withdrawal trial designs raise substantial ethical concerns for patients and families who believe that they do not enable evidence-based and patient-centred decisions around medication choices. In this Viewpoint, we present the personal views of a patient and parent network that is of the opinion that current trial design in juvenile idiopathic arthritis is failing children and young people with juvenile idiopathic arthritis and set out the need for change informed by lived experience.
Competing Interests: Declaration of interests AVR has received consulting fees from Eli Lilly, UCB, AbbVie, Novartis, and Alimera Biosciences. AVR has also received payment or honoraria from Eli Lilly, AbbVie, Pfizer, Novartis, Roche, and Sobi. AVR has participated on a data safety monitoring board or advisory board for Eli Lilly. EE is a trustee for Children's Chronic Arthritis Association (charity number 1185378). All other authors declare no competing interests. Funding for the CLUSTER Project has been provided by generous grants from the Medical Research Council (grant number MR/R013926/1), Versus Arthritis (grant number 22084), Great Ormond Street Hospital Children's Charity (grant number VS0518), and Olivia's Vision. This work is supported by the National Institute for Health and Care Research (NIHR) Great Ormond Street Hospital Biomedical Research Centre, the NIHR Manchester Biomedical Research Centre, the British Society for Rheumatology, and the UK's Experimental Arthritis Treatment Centre for Children, supported by Versus Arthritis (grant number 20621). The CLUSTER consortium has been provided with generous grants from AbbVie and Sobi, and in-kind contributions from GSK, Pfizer, and UCB. The views expressed are those of the authors and not necessarily those of the National Health Service, the NIHR, or the Department of Health.
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