Europe – Draft qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation – Overview of comments received

Welcoming the qualification process for the EBMT CAR-T initiatives ARM welcomes and thanks the EMA for the qualification of the EBMT CAR-T registry initiative allowing a public consultation on CHMP responses and EBMT briefing. Many advanced therapies, including
CAR-T products, seek to provide a transformative and long-lasting, potentially curative, effect with a single or few administrations, potentially enabling a shift from a focus on chronic treatment to possible cures. Real-life data generation and patient long-term
follow-up will therefore be critically important for substantiating the medium- to long-term safety and efficacy profiles of these medicinal products.
The EBMT registry is one of the first registries to be used in the context of real evidence data collection for a specific class of Advanced Therapy Medicinal Products (ATMPs). The answers provided in the consultation document therefore may set a precedent for the
future use of other registries capturing data on ATMPs…