EMA's new workshop report describes the opportunities and challenges in using patient registries for the benefit-risk evaluations of chimeric antigen receptor (CAR) T-cell therapies.
The recommendations in the report are based on the Chimeric antigen receptor (CAR) T-cell therapy registries workshop organised by the EMA on February 9th 2018 to which the EBMT was invited along with CIBMTR as part of the European agency's Patient Registry Initiative and stakeholders from industry, professional societies, patient representatives, health technology assessment agencies and national regulators.
The EMA states that one of the most important priorities will be to harmonise the data used in the registries, particularly the EBMT and CIBMTR registries. Also, the objective is to establish 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, giving confidence to users on the data collected.
Christian Chabannon, chairperson of the EBMT Cellular Therapy and immunobiology Working Party highlights: "CAR-T cells and other related somatic cell therapy medicinal products or gene therapy medicinal products that are made of living cells raise specific issues in terms of persistence and clinical effects. The manufacturing and delivery of these highly personalised medicines happens in the context of complex and costly organizations where academic and industry partners collaborate. Post-marketing evaluation of long-term effects in “real life” medical practices is thus essential to accurately measure their risks and efficacy, and appreciate their sustainability for our healthcare systems." Christian Chabannon adds: "This is where continental registries such as EBMT become essential actors with a proven track record of long-term surveys of large and representative patients populations undergoing complex therapeutic procedures such as hematopoietic cell transplantation, and the ability to rapidly detect practice-changing events that are the consequences of the introduction of biomedical innovations.”
EBMT has recently implemented a new Cellular Therapy form that will allow to more accurately capture the nature, sequence and effects of modern cellular therapies including CAR-T Cells.
In this context, EBMT's decision to upgrade the registry (Read “MACRO platform to support patient registry”) at significant cost has been more than justified. EBMT is leading a unique effort to standardise registration of cellular therapies such as CAR-T in Europe and across the globe and provide guidance on the minimal datasets needed for long term surveillance of patients receiving CAR-T Cells. EBMT has requested a qualification opinion of the registry from EMA and hopes for a positive decision in the near future.