G-BA starts consultations on data collection accompanying the use of Exagamglogen Autotemcel in sickle cell disease
05.06.2023 17:49
The Federal Joint Committee (G-BA) is examining whether in-use data collection (AbD) is possible for the active ingredient exagamglogen autotemcel for the treatment of sickle cell disease. He began discussions on this on June 1st.
The focus is on the use of the gene therapy drug in patients aged 12 and over with recurrent vaso-occlusive crises who have a specific gene change (genotype βS/βS, βS/β0 or βS/β+) and for whom there is no compatible stem cell donation from a relative is available. Since there are no directly comparative data from the approval study compared to existing treatment alternatives, the G-BA is now examining to what extent data from care could improve the study situation for the additional benefit assessment.
The active ingredient Exagamglogen Autotemcel is the first genetic cell therapy based on the CRISPR/Cas9 technology. It is a one-time infusion. Here, the hematopoietic stem cells are gene-edited so that they produce high amounts of fetal hemoglobin (hemoglobin F) in the red blood cells, which is intended to counteract the disease-causing form of hemoglobin (hemoglobin S). The active ingredient is currently in the European approval process for two blood diseases: sickle cell disease and transfusion-dependent beta-thalassemia.
With the decision of June 1st, the G-BA commissioned the Institute for Quality and Efficiency in Health Care to develop a concept with details for a possible AbD by September 1st, 2023. The G-BA will then carry out a professional participation process for this concept, involving, among others, the Federal Institute for Drugs and Medical Devices, the Paul Ehrlich Institute, the scientific and medical specialist societies, the Drug Commission of the German medical profession, the pharmaceutical company concerned itself and, if necessary Involve other experts, such as registry operators.
The G-BA is expected to decide at the end of 2023/beginning of 2024 whether the pharmaceutical company will be required to collect data from routine clinical care and evaluate it for a benefit assessment.
Resolution on this report:
Medicines Directive/Appendix