Biologic Treatment of Pediatric Rheumatic Diseases: Are we Spoilt for Choice?

Abstract

Approximately 15 years ago the first biological treatment for juvenile idiopathic arthritis (JIA) was introduced. Since the introduction of the TNF receptor fusion protein etanercept in 1998, now more than seven biologics are now registered, or under study for JIA. Notably, these biologics were initially not developed for JIA but derived from the experience in the treatment of adult rheumatic diseases. This simple fact still has major consequences on the prescription and reimbursement of biological treatments in pediatric rheumatic diseases (PRD). As an endorsement for clinical research in pediatric diseases, the EU launched a new act in 2006, the Pediatric Rule [101]. This rule states that drugs tested for a specific condition in adults are also required to be tested in related pediatric diseases [101]. Therefore, when pharmaceuticals submit a registration proposal to EMA, a Pediatric Investigational Plan must be included. This means increased efforts and costs for pharmaceutical companies. Despite the efforts of the EMA, many national and local institutional review boards are still reluctant to approve medication studies in children. In The Netherlands, therapeutic trials in children are only allowed under strict regulations. Children should benefit from the study, risks have to be negligible and the burden minimal. A proposal to amplify these strict regulations encountered a lot of resistance among members of the College of Human Rights, claiming to protect young children for medical trials. So the question arises: are pediatric rheumatologists convinced that therapeutic trials in children with rheumatic diseases are required in order to enlarge the spectrum of biological therapy, or do the current therapies provide s ufficient choices in the treatment of PRD?