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EMA releases their draft qualification opinion on the cellular therapy module of the EBMT registry for public consultation

by Melanie Chaboissier
Legal & Regulatory Affairs Committee

We are happy to announce that EMA has released its draft qualification opinion on using the EBMT registry to support Chimeric antigen receptor (CAR) T-cell therapy post-authorisation follow-up and risk-benefit evaluations. The Committee for Medicinal Products for Human Use (CHMP) considers that the cellular therapy module of the EBMT registry may be used as a data source for regulatory purposes in the context of CAR-T cell therapies authorised for haematological malignancies. The draft opinion goes on to stipulate in detail the scope of the studies that may be performed based on the registry.

In late 2016 the EBMT responded to EMA’s Patient Registry Initiative. Subsequent discussions ultimately led  in October 2017 to EBMT formally requesting a qualification opinion by EMA of its registry as a suitable data source for regulatory purposes within the context of CAR-T product regulation and post-authorisation drug utilisation, efficacy/effectiveness and safety studies.

Jürgen Kuball, EBMT treasurer and main liaison with the EMA, says: “Based on this draft opinion, the EBMT is very optimistic that EMA will formally recognize the value of our registry. Such recognition would lead to greatly improved communication among the various stakeholders, including registry owners, regulators and marketing authorization holders, giving confidence to users on the data collected and ultimately helping to bring safe and effective therapies to our patients”. He adds “regardless of the final outcome, the interaction with EMA has been tremendously positive for EBMT whereby external scrutiny has highlighted both our strengths and areas for improvement. We are very grateful to EMA for this opportunity”. He furthermore acknowledges the active contributions of EBMT Staff and volunteers to this achievement: Eoin McGrath, Anja van Biezen, Carmen Ruiz de Elvira, Christian Chabannon and Chiara Bonini.

The public consultation process will last eight weeks leading to the adoption of the final qualification opinion by the CHMP subject to the nature of the submissions received, wider discussions and scientific developments.

For more information, please click on the link below to read the full draft qualification opinion document.