Neuroblastoma in children and adolescents – G-BA has quadruple combination evaluated for off-label use

26.09.2023 13:37

Around 150 children and young people in Germany develop neuroblastoma every year. The malignant disease of the nervous system is the second most common cancer in children. In the high-risk variant of neuroblastoma, the overall survival rate is below 30 %. Does a quadruple combination of the active ingredients temozolomide, irinotecan, sirolimus and dasatinib, which is not approved for this area of application, improve the chances of survival for children and adolescents? The combination of active ingredients can currently only be used off-label in individual cases at the expense of statutory health insurance (GKV).

The Federal Joint Committee (G-BA) commissioned the Off-Label expert group to assess the state of scientific knowledge. The current reason is the positive results of the now completed RIST-rNB-2011 study. She examined the effect of the quadruple combination in children and adolescents with relapsed or refractory neuroblastoma. The expert group should view and classify these results. On this basis, the G-BA will decide whether off-label use of the quadruple combination becomes a regular GKV service.

Study compared quadruple with double combination

The RIST-rNB-2011 study is a randomized and controlled comparative study. She examined the treatment results of 124 children and adolescents with recurrent neuroblastoma or neuroblastoma that had progressed during initial treatment. The treatment results of the unapproved quadruple combination (irinotecan, temozolomide, dasatanib and sirolimus) were compared with those of a dual combination (irinotecan and temozolomide).

Under certain conditions, a regulation of the off-label use of drugs in non-commercial clinical studies is permitted at the expense of the GKV. This is possible if it is expected that off-label use can significantly improve the treatment of a serious disease compared to available therapies. The G-BA had approved the application to cover the costs of the off-label use of the four active ingredients as part of the RIST-rNB-2011 study. The sponsor of the study was the Regensburg University Hospital.