Original Title: Avis: Esperoct (turoctocog alfa pegol) – Hemophilia A, avis d’introduction à la Liste des produits du système du sang du Québec

Authors' objectives: The Institut national d’excellence en santé et en services sociaux (INESSS) evaluated the stable blood product Esperoct (turoctocog alfa pegol, N8-GP), a recombinant factor VIII (FVIII) conjugated with a polyethylene glycol (PEG) molecule and administered by intravenous injection. It is indicated for routine prophylaxis, on-demand treatment and the management of perioperative bleeding in patients with hemophilia A (congenital FVIII deficiency). The recombinant FVIII Advate, Adynovate, Eloctate, Kovaltry, Nuwiq, Xyntha (including Xyntha Solofuse) and Zonovate are currently listed on the Liste des produits du système du sang du Québec. Nuwiq and Zonovate are distributed by Héma-Québec, and Eloctate is available under an MSSS directive.

Authors' results and conclusions: EFFICACY: • For individuals over 12 years of age treated prophylactically with Esperoct, the estimated median annualized bleeding rate was 1.18, a 96% reduction compared to patients treated on-demand (Pathfinder 2 study). Over the mean study duration of 299 days, 40% of the individuals treated prophylactically reported no bleeding. • For children under 12 years of age treated prophylactically, the median annualized bleeding rate was 1.95 (Pathfinder 5). During the mean observation period of 182 days, 42% of the children reported no bleeding. SAFETY: • The most frequently observed adverse events were skin problems (rash, erythema and pruritus) and local reactions at the injection site. The severity of all these reactions ranged from low to moderate. • Severe adverse events associated with Esperoct administration (development of inhibitors, intervertebral disc infection, severe allergic reaction and increased symptoms of moderate bleeding) have been reported in four (4/270, or 1.5%) patients. • Only one individual exposed to Esperoct for more than 50 days developed inhibitors out of a total of 235 (0.4%) patients previously treated prophylactically with at least one FVIII. • One child (1/68, or 1.5%) developed antibodies to PEG following exposure to Esperoct in the Pathfinder 5 study with no clinical consequences. • Nine individuals (9/270, or 3.3%) reported antibodies against a CHO cell antigen with no clinical consequences. EFFICIENCY: • Esperoct, at the submitted price, is a least efficient treatment option compared to the other recombinant FVIIIs currently distributed in Quebec, namely Nuwiq, Zonovate and Eloctate. BUDGET IMPACT:• Replacing Eloctate, the only long-acting FVIII currently distributed in Quebec, with Esperoct, at the submitted price, could lead to additional expenses estimated at $1.4 million over the next 3 years.

Authors' recommendations: The members of the Comité scientifique permanent de l’évaluation des médicaments aux fins d’inscription unanimously recognized the therapeutic value of turoctocog alfa pegol (Esperoct) for routine prophylaxis, the on-demand treatment of bleeding episodes, and perioperative management in children and adults with hemophilia A. Consequently, the members unanimously share the opinion that turoctocog alfa pegol (Esperoct) should be added to the Liste des produits du système du sang du Québec. However, they stress that, in the interest of equitable and reasonable access to treatments, this addition should be accompanied by measures aimed at reducing the economic burden so as not to create an incremental cost in relation to the currently distributed therapies.

Project Status: Completed

URL for project: https://www.inesss.qc.ca/publications/repertoire-des-publications/publication/esperoctmc-mise-a-jour-de-la-liste-des-produits-du-systeme-du-sang-du-quebec-octobre-2019.html

Year Published: 2019

URL for published report: https://www.inesss.qc.ca/publications/repertoire-des-publications/publication/esperoctmc-mise-a-jour-de-la-liste-des-produits-du-systeme-du-sang-du-quebec-octobre-2019.html

Requestor: Minister of Health

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