EMA Qualifies CAR T-Cell Therapy Registry

Regulatory NewsRegulatory News | 01 March 2019 |  By 

The European Medicines Agency (EMA) this week qualified a registry from the European Society for Blood and Marrow Transplantation’s (EBMT), which means it is now suitable to perform pharmacoepidemiologic studies for regulatory purposes concerning Chimeric Antigen Receptor (CAR) T-cell therapies used to treat hematological malignancies.

The nonprofit EBMT, which also runs a registry of bone marrow transplants, explained how the qualification of the registry makes it a suitable platform for the collection of data for post-authorization safety surveillance and efficacy studies.

According to the qualification opinion from EMA, the agency was asked whether the EBMT Registry can be used as a source of data for CAR T-cell product comparative studies.

EMA’s Committee for Medicinal Products for Human Use (CHMP) said it “is of the opinion that randomised, controlled trials remain the standard for comparative evaluations. Registry based evaluations can extend and add to the findings from randomised trials or be useful in situations where randomised trials are not feasible, e.g. rare adverse reactions or long term safety evaluation. It is recognised that the EBMT collects data that might be suitable for comparative analyses. Whether or not these data can be used as source of comparative data for CAR T-cell studies will depend on the particular research question and/or the specific treatment studied.”

Other critical issues, according to EMA, are related to “completeness of data capture, the actual coverage (what proportion of patients overall is estimated to be included), data quality and consistency over time.”

On the same question of whether the registry can be used in comparative studies, EBMT explained how in recent years, the registry has been used for regulatory purposes for therapies involving plerixafor and defibrotide.

In addition, the registry was used in the evaluation of Zalmoxis, the first immunogene therapy for the treatment of adult patients with high-risk hematological malignancies, which was granted conditional authorization by EMA in 2016.

“The EBMT registry allowed for a matched pair analysis comparing the outcome of patients who received Zalmoxis versus those being treated as standard of care. This comparison was presented as an oral session during the American Society of Hematology annual meeting in 2016. Additionally, Zalmoxis was used as an example of using RWD in an EMA presentation at the Industry Stakeholder Platform on Research and Development Support on 25 April 2017,” the company’s response in the opinion says.

Qualification opinion on Cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry


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