ATMP Webinar: A Practical Primer for Clinical Coordinators, Nurses & Pharmacists

This webinar is the first in a series organized under Key Initiative 1.3 of the RareKids-CAN strategic priority, ‘Strengthening Pediatric Rare Disease Clinical Trial Capacity and Advanced Therapy Medicinal Product Readiness.’ It provides a practical introduction to Advanced Therapy Medicinal Products (ATMPs) for clinical coordinators, nurses, pharmacists, and research staff working in pediatric rare disease trials. Participants will gain a foundational understanding of gene therapies, cell therapies, and antisense oligonucleotides (ASOs) and how these trials differ from non-ATMP products or trials.

By the end of the session, participants will feel more confident explaining ATMPs to patients and families in clear, accessible language. By the end of this session, participants will be able to:

• Define ATMPs and distinguish between gene therapy, cell therapy, and ASOs

• Describe how ATMPs differ from traditional therapies (small molecules, biologics, etc)

• Explain, at a high level, how ATMPs work and are made

• Recognize key operational considerations

• Confidently explain ATMPs to patients and families in plain language