Original Title: Avis - Réévaluation de Rebinyn (nonacog bêta pégol) – Hémophilie de type B

Authors' objectives: At the request of the manufacturer Novo Nordisk Canada Inc., the Institut national d’excellence en santé et en services sociaux (INESSS) re-evaluated the blood system product RebinynTM (nonacog beta pegol), an intravenously injected recombinant factor IX (FIX) indicated in adults and children with hemophilia B (congenital factor IX deficiency or Christmas disease) for the control and prevention of bleeding episodes and in the perioperative setting as well as in patients 18 years and above with hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The indications assessed for this re-evaluation are identical to those accorded by Health Canada. INESSS previously evaluated nonacog beta pegol (evaluation of June 2020). During the first evaluation, INESSS issued an unfavourable recommendation for the addition of the product to the Liste des produits du système du sang du Québec because the therapeutic value had not been recognized by the Comité scientifique permanent de l'évaluation des médicaments aux fins d'inscriptions (CSEMI). This is the second evaluation of this product. The following five FIX currently listed on the Liste des produits du système du sang du Québec were used as the comparators of nonacog beta pegol: BeneFIXTM (nonacog alfa; standard half-life recombinant FIX), RixubisTM (nonacog gamma; standard half-life recombinant FIX), AlprolixTM (eftrénonacog alfa; extended half-life recombinant FIX), IdelvionTM (albutrepenonacog alfa; extended half-life recombinant FIX), ImmunineTM (plasma-derived FIX).

Authors' results and conclusions: RESULTS: Combined data from a real-world Canadian study, a post-market surveillance report on nonacog beta pegol, as well as previously evaluated clinical studies were considered for this evaluation. Efficacy • Nonacog beta pegol appears at least as effective as its comparators to prevent bleeding when used as a prophylactic. • Nonacog beta pegol appears at least as effective as its comparators to treat breakthrough bleeds. • In the studies listed, nonacog beta pegol demonstrates good or excellent hemostatic efficacy during surgery. Safety • The safety profile of nonacog beta pegol was considered acceptable. Quality of life • No new data regarding the impact of nonacog beta pegol on quality of life was presented. Considering the data presented as part of this reassessment, the expert consulted are of the opinion that the efficacy of nonacog beta pegol prophylaxis is comparable to the other FIX listed on the Liste des produits du système du sang du Québec. Experts also considered the safety profile of nonacog beta pegol to be similar to the other available therapeutic options for the targeted population. Furthermore, preoccupations regarding the potential accumulation of PEG in the choroid plexus remain theoretical in humans and would not prevent them from using nonacog beta pegol for the targeted population in Quebec.

Authors' recommendations: Members of the Comité scientifique permanent de l’évaluation des médicaments aux fins d’inscription (CSEMI) having exercised their right to vote are of the unanimous opinion that RebinynTM (nonagog beta pegol) should be added to the Liste des produits du système du sang du Québec for the treatment of hemophilia B (congenital factor IX deficiency or Christmas disease) in adults and children for the control and prevention of bleeding episodes and in the perioperative setting as well as in patients 18 years and above with hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes. In light of the available data, INESSS recommends that RebinynTM (nonacog beta pegol) be added to the Liste des produits du système du sang du Québec for the treatment of hemophilia B (congenital factor IX deficiency or Christmas disease) in adults and children for the control and prevention of bleeding episodes and in the perioperative setting as well as in patients 18 years and above with hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes. From a distributive justice perspective, the reimbursement of nonacog beta pegol for the requested indication would constitute a responsible, fair and equitable decision if the cost of using nonacog beta pegol does not exceed that of extended half-life FIX during the next call for tenders.

Authors' methods: Published trials and manufacturer data were reviewed to document the efficacy, safety and efficiency of nonacog beta pegol. Experiential and contextual data from expert consultations and patients are presented as well.

Project Status: Completed

URL for project: https://www.inesss.qc.ca/en/publications/publications/publication/reevaluation-de-rebinynmc-nonacog-beta-pegol-hemophilie-de-type-b.html

Year Published: 2021

URL for published report: https://www.inesss.qc.ca/en/publications/publications/publication/reevaluation-de-rebinynmc-nonacog-beta-pegol-hemophilie-de-type-b.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