ATMPs in Germany - Ebvallo®

👉 Ebvallo is the first authorized allogeneic T-cell immunotherapy.

Ebvallo (active substance: tabelecleucel) was authorized in December 2022 for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD). The gene therapy Ebvallo targets and eliminates EBV-positive cells. The pivotal study is the single-arm trial ALLELE including 43 patients with EBV+ PTLD following solid organ transplant or hematopoietic cell transplantation. In this trial, patients were assigned to pre-specified cohorts based on transplant type and prior therapy treatment failure. Ebvallo is available in Germany.

🏆German HTA rating:

No direct comparative data are available for Ebvallo. Accordingly, the German HTA rating in October 2023 resulted in a non-quantifiable additional benefit, only due to the orphan drug status.

For HTA, data from the single-arm study ALLELE, the expanded access study EBV-CTL-201 as well as indirect comparisons between the ALLELE and the retrospective, non-interventional observational study ATA129-RS002 were submitted.

As previously observed, the indirect comparisons based on single-arm trials were not sufficient for German HTA as given the uncertainties regarding the selection of confounders, the structural equality of the study populations and the small effect sizes.

💵 Launch price: € 75,000 € per vial. Treatment is recommended for 2–8 cycles using 3 vials per cycle, resulting in 6–24 vials of treatment. Reimbursement price: € 55,444.45. Rebate: 26 %

📘 Special features of reimbursement:

Ebvallo is reimbursed and subject to the ATMP quality assurance guideline.

💡Evidence of almost 200 patients with EBV+ PTLD was presented for HTA. Remarkably, only 7–30 patients in Germany per year are assumed to exist in the indication of Ebvallo. Looking at the HTA resolution, the size of the patient population had no impact on the evidence rating. Yet, an impact on the price negotiations might be possible.