- Date 15 December 2021
- Documents : 7
- Economic analysis
- History
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Reason for request
Key points
Favourable opinion for reimbursement as monotherapy for the treatment of children or adolescents aged 1 year or older with high-risk first relapsed Philadelphia chromosome negative CD19 positive B-precursor acute lymphoblastic leukaemia (ALL) as part of the consolidation therapy.
What therapeutic improvement?
Therapeutic improvement compared to conventional chemotherapy.
Role in the care pathway?
In patients with first relapsed Philadelphia chromosome negative CD19 positive B-precursor acute lymphoblastic leukaemia (ALL), second-line therapy is not standardised but is generally based on re-induction polychemotherapy followed by a further consolidation phase. The objective is to achieve a second remission and to enable the patient to have access to allogeneic haematopoietic stem cell transplantation in the best possible haematological conditions and general health with, insofar as possible, negative minimum residual disease (MRD) before allogeneic transplantation. It is now established, in fact, that the efficacy of allogeneic transplantation (measured by the absence of post-transplant relapse) depends on the level of pre-transplant disease.
In the event of a high-risk relapse (definition based on the time to relapse and its location, see Annex 1), it is usually recommended to administer polychemotherapy protocols at higher doses than in standard-risk situations, inducing short and/or long-term toxicities before having access to allogeneic transplantation.
Role of the medicinal product in the care pathway
BLINCYTO (blinatumomab) represents a treatment to be favoured over conventional chemotherapy as a third block of consolidation therapy for the treatment of children or adolescents aged 1 year or older with Philadelphia chromosome negative CD19 positive B-precursor ALL, having achieved complete remission after a first high-risk relapse.
The Committee highlights the fact that BLINCYTO (blinatumomab) can mainly be administered to patients at home, unlike chemotherapy treatments, which require full hospitalisation throughout treatment, particularly due to the risk of infectious complications associated with the haematological toxicity of chemotherapies.
Clinical Benefit
| Substantial |
The Committee deems that the clinical benefit of BLINCYTO (blinatumomab) is substantial as monotherapy for the treatment of paediatric patients aged 1 year or older with high-risk first relapsed Philadelphia chromosome negative CD19 positive B-precursor acute lymphoblastic leukaemia (ALL) as part of the consolidation therapy. |
Clinical Added Value
| moderate |
Considering:
but in view of:
the Committee considers that BLINCYTO (blinatumomab) provides a moderate clinical added value (CAV III) compared to conventional chemotherapy in the treatment of children and adolescents aged 1 year or older with high-risk first relapsed Philadelphia chromosome-negative CD19 positive B-precursor ALL as part of the third block of consolidation therapy.
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Avis économique
Ce produit a fait l'objet d'un avis économique rendu par la Commission d'évaluation économique et de santé publique le 14 décembre 2021.
L’évaluation porte sur le traitement des patients pédiatriques à partir de l’âge de 1 an, présentant une leucémie aigüe lymphoblastique (LAL) à précurseurs B exprimant le CD19 avec chromosome Philadelphie négatif en première rechute à haut risque dans le cadre du traitement de consolidation (après l’induction et 2 blocs de chimiothérapie de consolidation). Cette indication est superposable à l’indication de l’AMM.
Sous les hypothèses retenues par l’industriel, le produit est associé à un RDCR de 7 392 € par QALY gagné et 6 300 € par année de vie gagnée versus HC3 sur un horizon temporel vie entière.
L’incertitude générée par les choix méthodologiques retenus dans la modélisation et par la variabilité statistique des paramètres est limitée.
Le produit n’a pas fait l’objet d’une analyse d’impact budgétaire.
> BLINCYTO - Avis économique (pdf)
