TRECONDI (treosulfan) - Haematopoietic stem cell transplantation (alloHSCT)

Opinions on drugs - Posted on Sep 05 2024

Reason for request

Indication extension

Summary of opinion

Favourable opinion for reimbursement in the following indication: “treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in paediatric patients older than one month with non-malignant diseases”.

Clinical Benefit

Low	The clinical benefit of TRECONDI (treosulfan) in combination with fludarabine is low in paediatric patients (older than one month) with non-malignant diseases as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT).
Insufficient

Clinical Added Value

no clinical added value

Considering:

the not very robust quality of the evidence of the efficacy of TRECONDI (treosulfan) in combination with fludarabine based on data from a descriptive phase 2 study;
the absence of data enabling a conclusion to be reached with respect to an improvement in overall survival, quality of life or a better safety of the treosulfan-fludarabine combination compared to the busulfan-fludarabine combination;
the absence of comparative data versus the conditioning regimens used in paediatric patients;
the partially met substantial medical need in this indication;

the Committee considers that TRECONDI (treosulfan) provides no clinical added value (CAV V) in the current care pathway, which includes clinically relevant comparators.