FDA Pediatric Lupus Workshop

We are excited to share that CARRA and the Pediatric Rheumatology Collaborative Study Group (PRCSG) have collaborated to initiate the convening and planning of an FDA Workshop: Accelerating Product Development for Pediatric Systemic Lupus Erythematosus (SLE). This forum provides an opportunity for the broader community to discuss ways to streamline drug development and ensure that our community’s voices are included in regulatory decision-making.

This important event will bring together physicians, researchers, regulators, pharmaceutical industry leaders, patient advocates and families to work towards more ways to get SLE treatments approved for pediatric use more quickly. In the past it has taken as long as eight years after regulatory approval for adults to get pediatric approval for a lupus treatment.

“Everyone wins if we can reduce the time it takes to determine that a treatment is safe and effective for children,” said Stacy Ardoin, MD, MSc, who is board-certified in adult and pediatric rheumatology and is serving as CARRA President. “We are poised to make sure our pediatric patients shouldn’t have to wait years and years for a treatment.”

Many CARRA leaders are speaking at this event to share their expertise on topics such as the current treatment landscape for pediatric lupus, unmet medical need, the relationship between pediatric and adult SLE, and extrapolation of efficacy from adult data for pediatrics. The agenda also includes insights from pharmacology experts on issues such as the similarities and differences between adults and pediatric SLE in drug pharmacology and statistical approaches to optimize dose selection.

CARRA and PRCSG are poised to support this effort through our extensive networks, and are proud to collaborate on this important initiative for our shared community.

“This workshop brings together rigorous science, strong partnerships and a clear regulatory pathway,” said Hermine Brunner, MD, MSc, MBA, and Scientific Director of PRCSG. “By combining the strengths of PRCSG, an organization dedicated to conducting high-quality clinical trials for children with rheumatic disease, CARRA, industry, and the FDA, we are laying the groundwork for better outcomes for children living with SLE.”

The CARRA Registry, which is the largest ongoing, observational registry in North America collects clinical and patient-reported information in pediatric rheumatic diseases, such as lupus. The CARRA Registry serves as an important real-world data source which can be used to inform study design, extrapolate efficacy data, and facilitate safety and efficacy monitoring once a drug is on the market and being prescribed.

PRCSG, which is a consortium of over 80 academic clinical pediatric rheumatology centers in the United States and Canada, has completed, or is currently in the process of conducting, more than 45 trials in children with rheumatic disease, including SLE. PRCSG has performed the vast majority of trials of medications for JIA in North America since 1977.

The workshop, which is co-hosted by the FDA and the Center of Excellence in Regulatory Science and Innovation, will be at the FDA in Silver Spring, Maryland on July 30-31. Registration is free and this event is also available virtually.

Register here: https://www.mcersi.umd.edu/workshops/accelerating-product-development-for-pediatric-systemic-lupus-erythematosus-sle