Module232025

ATMPs for Pediatrics: Key Points

ATMP Types : Gene therapy (GTMPs), somatic cell therapy (sCTMPs), tissue-engineered products (TEPs), and device-combined ATMPs. Regulatory Framework : EMA oversees ATMPs in the EU with tailored authorization paths, including pediatric incentives like PUMA. Development Guidelines : Requires GMP, GCP, GLP practices and pharmacovigilance for post-approval monitoring. Pediatric ATMP Challenges : Few approved ATMPs for pediatrics; barriers include trial access, finances, and suitable models. Access Initiatives : PRIME accelerates approval, and the EU Clinical Trials Register promotes transparency.

Quality (CMC) Development Considerations

The Organisation for Professionals in Regulatory Affairs