Study Design
Use emerging data sources and advances in evidence methods to demonstrate product value and safety.


This paper outlines a modern, patient‑centered approach to meeting complex regulatory and operational demands of long‑term follow‑up, demonstrating how real‑world data, digital and decentralized tools, and adaptable study design can improve retention, data quality, and scalability over 5–15 years.
It presents a forward‑looking vision for sustainable, efficient LTFU that protects patients, advances science, and supports the full lifecycle of cell and gene therapies.
Use emerging data sources and advances in evidence methods to demonstrate product value and safety.