Original Title: Avis: Évaluation de AfstylaMC (rFVIII) – hémophilie de type A

Authors' objectives: At the request of the manufacturer, CSL Behring Canada Inc., INESSS evaluated the blood system product Afstyla™ (lonoctocog alfa), a recombinant human coagulation factor VIII (FVIII) that is administered intravenously. In Canada, lonoctocog alfa is indicated for for control and prevention of bleeding episodes, routine prophylaxis to prevent or reduce the frequency of bleeding episodes and for perioperative management of bleeding (surgical prophylaxis) in adults and children with hemophila A (congenital factor VIII deficiency). The indications for this re-evaluation are identical to those recognized by Health Canada. The following nine FVIII products are currently on the Liste des produits du système du sang du Québec and were used as comparators. They include six standard-acting products (Advate™, Helixate™, Kovaltry™, Nuwiq™, Xyntha™ (including Xyntha Solofuse™) and Zonovate™ (standard-acting), and three long-acting products (Adynovate™, Eloctate™ and Esperoct™)

Authors' results and conclusions: RESULTS: (1 Efficacy) - Lonoctocog alfa is considered a standard-acting FVIII. • Lonoctocog alfa appears to be at least as effective as its comparators in preventing bleeding when used prophylactically. • Lonoctocog alfa appears to be as effective as its comparators in treating breakthrough bleeding. • In the studies identified, lonoctocog alfa exhibited good or excellent hemostatic efficacy during surgery. (2 Safety) - The safety profile of lonoctocog alfa is considered acceptable. (3 Quality of life) - No data on the impact of lonoctocog alfa on quality of life were presented. Based on the available data, the expert consulted is of the opinion that the efficacy of prophylaxis with lonoctocog alfa is comparable to that provided by the comparators, that is, all the FVIII products on the Liste. In the expert’s opinion, the safety profile of lonoctocog alfa is comparable to that of the other options available for the target population.

Authors' recommendations: INESSS recommends including Afstyla™ (lonoctocog alfa) on the Liste des produits du système du sang du Québec for adults and children with hemophilia A (congenital FVIII deficiency) for:  control and prevention of bleeding episodes;  routine prophylaxis to prevent or reduce the frequency of bleeding episodes;  perioperative management of bleeding (surgical prophylaxis). From a distributive justice perspective, coverage of lonoctocog alfa for the requested indication would be a responsible, fair and equitable decision, provided the cost of using lonoctocog alfa does not exceed that of the least expensive standard-acting FVIII in the next call for tenders.

Authors' methods: Literature data and data provided by the manufacturer were reviewed to document the efficacy, safety, and cost-effectiveness of lonoctocog alfa. Contextual and experiential data from expert and patient consultations are presented as well. Lastly, INESSS performed a cost-effectiveness and budget impact analysis.

Project Status: Completed

URL for project: https://www.inesss.qc.ca/en/publications/publications/publication/evaluation-de-afstylamc-rfviii-hemophilie-de-type-a.html

Year Published: 2022

URL for published report: https://www.inesss.qc.ca/en/publications/publications/publication/evaluation-de-afstylamc-rfviii-hemophilie-de-type-a.html

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Canada

Province: Quebec

Organisation Name: Institut national d'excellence en sante et en services sociaux

Contact Address: L'Institut national d'excellence en sante et en services sociaux (INESSS) , 2021, avenue Union, bureau 10.083, Montreal, Quebec, Canada, H3A 2S9;Tel: 1+514-873-2563, Fax: 1+514-873-1369

Contact Name: demande@inesss.qc.ca

Contact Email: demande@inesss.qc.ca

Copyright: Gouvernement du Québec