Global Labeling Conference

There has been an increase in Cell and Gene Therapies (CGT) being developed and approved as novel treatments for rare diseases. Unique situations, challenges, and requirements face CGTs which require the labeling team to engage with a broad stake-holder network during development. Considerations such as safety concerns associated with CGTs (including benefit:risk considerations for specific populations), study expectations and design elements, alternative endpoints/data-sets informing the label, traceability, and planning of long-term follow-up requirements and supply chain. CGT examples and labeling themes and considerations to support the development of CGTs labels for rare diseases will be provided. The session will finish with a patient advocacy speaker for Friedreich ataxia (FA), a progressive neurodegenerative disorder that affects the nervous system. An opportunity to hear considerations, questions, support, communication and education requirements, that face the treatment journey for an individual with a rare disease which has the potential for treatment with a cell and gene therapy.