New Study Examines Progress and Challenges in Biologic Treatments for Pediatric Rheumatic Diseases Over 25 Years

In a new study, researchers reviewed the advancements and ongoing challenges in the development of biologic therapies and disease prognosis of pediatric rheumatic diseases over the past 25 years, focusing on conditions including juvenile idiopathic arthritis (JIA), childhood-onset systemic lupus erythematosus (cSLE), and juvenile dermatomyositis (JDM). These diseases affect around 300,000 children in the U.S., and while biologic disease-modifying antirheumatic drugs (bDMARDs), better known as, biologics, have improved outcomes for JIA, the landscape is far less developed for cSLE and JDM. 

Belimumab is the only biologic approved for treatment of cSLE. It was initially approved for adults with SLE in 2011, cSLE in 2019, and childhood-onset lupus nephritis (LN) in 2022. Anifrolumab, another biologic therapy used in adults who have SLE and LN, was approved in 2021. However, pediatric studies for Anifrolumab began in 2023. While Anifrolumab shows promise, studies are still in early phases and not yet approved by the U.S. Food and Drug Administration (FDA) for children. 

The study also examines the regulatory landscape for pediatric rheumatic diseases. There are regulations in place aimed to ensure timely access to effective treatments while also encouraging pharmaceutical companies to conduct necessary pediatric studies. The Pediatric Research Equity Act (PREA) mandates that new drugs be studied if there is a similar disease in adults likely to affect children. Furthermore, the FDA Safety and Innovations Act requires earlier submission of pediatric study plans from manufacturers under PREA obligations. 

While there has been significant progress over the last 25 years with biologics for treatment of pediatric rheumatic diseases, the researchers note that there are still unmet needs and call for further drug development efforts and studies for the pediatric population. Learn more about our childhood lupus research program.

Read the study