Using registries to identify adverse events in rheumatic diseases.

Area of Science:

• Pediatric Rheumatology
• Pharmacovigilance
• Immunomodulatory Therapies

Background:

• Biologics and immunomodulatory drugs are effective for pediatric rheumatic diseases but carry risks like infection and malignancy.
• Current safety surveillance methods in children are limited by small sample sizes and inadequate comparators.
• Concerns exist regarding serial exposures to multiple immunomodulatory products in pediatric patients.

Purpose of the Study:

• To introduce a novel pharmacosurveillance model for pediatric rheumatic diseases.
• To address limitations in existing post-marketing surveillance for immunomodulatory products in children.
• To establish a robust system for monitoring safety risks associated with pediatric rheumatic disease treatments.

Main Methods:

• Development of the CARRA Consolidated Safety Registry (CoRe) by CARRA and DCRI in collaboration with FDA and stakeholders.
• Utilizing a multicenter, longitudinal pediatric rheumatic diseases registry with over 8000 participants.
• Implementing enrollment regardless of medication exposure and flexible data collection for safety outcomes and comparator groups.

Main Results:

• The CARRA CoRe registry provides access to a larger patient cohort than single-product registries.
• Enrollment flexibility allows for more accurate detection and evaluation of safety signals.
• The model is adaptable for various pediatric and adult diseases, meeting post-marketing surveillance requirements.

Conclusions:

• The CARRA CoRe model offers a transformative approach to pharmacosurveillance in pediatric rheumatic diseases.
• This registry enhances the ability to detect and evaluate safety risks of immunomodulatory products in children.
• The proposed model can be expanded to other disease areas, improving overall drug safety monitoring.