EMA Report Makes Recommendations for CAR-T Patient Registries

Regulatory NewsRegulatory News | 22 May 2018 |  By 

A new report from the European Medicines Agency (EMA) lays out recommendations for improving the use of patient registries to support the benefit-risk evaluation of chimeric antigen receptor (CAR) T-cell therapies.
The report comes as EMA considers whether to approve the first batch of CAR T-cell products submitted to it for marketing authorisation, including applications for Kite’s Yescarta (axicabtagene ciloleucel) and Novartis’ Kymriah (tisagenlecleucel), both of which have already been approved in the US.
The recommendations in the report are based on input EMA received at a workshop in February that included stakeholders from industry, patient representatives, national regulators and two large registry owners as part of the agency’s patient registry initiative.
“In the context of pre-submission discussions of CAR T-cell products included in the [priority medicines] PRIME scheme, it became clear that registries would play an essential role in risk-benefit evaluations, especially in post-authorisation data generation,” EMA writes.
According to EMA, one of the biggest priorities going forward will be to harmonize the data elements used across various registries, particularly those operated by the European Society for Blood and Marrow Transplantation (EBMT) and the US-based Center for International Blood and Marrow Transplant Research (CIBMTR).
Other priorities include establishing measures to ensure that data is collected systematically, enable data sharing and to improve communication across the various stakeholders, including registry owners, regulators and marketing authorization holders.
However, the workshop also identified a number of barriers to progress, including the need for more sustainable funding for registry owners and clear guidance from regulators and health technology assessment (HTA) bodies on quality assurance for registry data.


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