Workshop Findings Summarize Innovative Approaches to Pediatric Juvenile Idiopathic Arthritis Treatment Development

Use of Extrapolation to Support Regulatory Decision-Making Discussed

(Washington, DC – August 4, 2023) – Findings from a recent stakeholder workshop on the development of therapeutics for polyarticular JIA (pJIA) were published online in the April 27, 2023 issue of Arthritis & Rheumatology. The report, “Therapeutic Development in Polyarticular Course Juvenile Idiopathic Arthritis (pcJIA): Extrapolation, Dose Selection, and Clinical Trial Design” sheds light on the persistent challenges that delay drug development and access to newer, safe, and effective treatments for children and adolescents living with pJIA.

Historically, pharmaceutical companies receiving regulatory approval for drug treatments in adults were not required to pursue a pediatric indication. According to the FDA website, before the agency initiated a pediatric program, only about 20 percent of drugs approved by the FDA were labeled for pediatric use, leaving clinicians to make drug-related decisions for ill children and adolescents based on limited dosing, safety, and efficacy information. While there has been a marked increase in recent years in FDA-approved treatments for children and adolescents, due in part to federal regulations such as the Pediatric Research Equity Act (PREA), many children and adolescents with pJIA continue to have active disease and the treatment burden remains untenable for patients and providers.

“We need efficient ways to get safe and effective drugs to children more quickly,” states the report’s lead author Laura Schanberg, MD, co-chair of the CARRA Research and Registry Oversight Committee and Professor of Pediatric Rheumatology at Duke University School of Medicine. “This has become more complicated as the number of effective treatments grows, leaving fewer children available for placebo-controlled trials and fewer parents willing to participate in such trials. Extrapolation of results from adult trials and other novel approaches discussed at the workshop, will increase clinical trial feasibility and expedite drug development in pJIA and pediatrics more generally.”

In 2019, the FDA and University of Maryland CERSI (Center of Excellence in Regulatory Science and Innovation), in cooperation with Childhood Arthritis and Rheumatology Research Alliance (CARRA), gathered leaders from pediatric rheumatology, federal agencies, the European Medicine Agency (EMA), the National Institutes of Health (NIH) academia, patients, and industry stakeholders to facilitate a broad public discussion of the current state of the science, treatment development, accessibility, and outcomes in pJIA. Workshop attendees discussed the highest areas of need and outlined approaches for expediting treatment development that include:

• Appropriate extrapolation of efficacy from adult rheumatoid arthritis (RA) trials as part of the pediatric development plan. This requires pre-trial planning to make sure RA trial design and outcomes facilitate extrapolation to pediatrics.
• The need for companies to plan earlier, work with experts in the field, and develop development programs for RA that facilitate pJIA development programs.
• Increased focus on consistent long-term follow up to evaluate the safety of new therapeutics in pJIA patients via existing disease registries.

“The accumulated experience amassed by pediatric rheumatology treating providers, research communities, and drug developers in the past ten to fifteen years have allowed for discussions about new and innovative study designs in children with rare diseases such as pJIA,” said Nikolay Nikolov, MD, director of the Division of Rheumatology and Transplant Medicine in the FDA’s Center for Drug Evaluation and Research. “The utilization of pediatric extrapolation to support regulatory decisions has been of great interest to the FDA. Following the discussions at the workshop, the Agency has employed pediatric extrapolation to support regulatory decisions and provide access to new safe and effective therapies to pediatric patients with pcJIA and jPsA. We look forward to continuing the discussion with this community.”

A direct link to the workshop findings can be found at: https://doi.org/10.1002/art.42534.

About Childhood Arthritis and Rheumatology Research Alliance (CARRA)

Formed in 2002, CARRA’s mission is to conduct collaborative research to prevent, treat, and cure pediatric rheumatic diseases. CARRA is a 501(c)3 registered non-profit organization. For more information, please visit https://carragroup.org/.

For more information contact:
Kelly Mieszkalski, Communications Director, CARRA
[email protected]