A new product, Hemlibra, called emicizumab during clinical trials and ACE-910 before that, is now licensed in Canada for patients with hemophilia A and inhibitors. With currently available therapies (FVIII, rFVIIa and aPCC), bleeding in individuals with hemophilia A and inhibitors is not easily controlled, and treatment is extremely burdensome. Most importantly, joint damage is serious and often occurs very young; the risk of death is increased. Hemlibra, shown to be effective at preventing bleeds, is administered subcutaneously, once a week or less.

The current Canadian Blood Services (CBS) process to add a new therapy to its formulary (and provide funding for it) includes more than 30 different steps and is designed to take between 12 and 24 months after approval by Health Canada. Under the current process, this potentially life-changing therapy would only be available to patients in late 2019 or early 2020. This is unacceptable.

On behalf of the approximately 100 individuals with hemophilia A and inhibitors, the CHS has encouraged CBS, the Canadian Agency for Drugs in Technology and Health (CADTH), the Institut national d’excellence en santé et en services sociaux (INESSS) in Quebec and the provincial and territorial ministers to rise to our challenge to have this therapy available by the end of 2018. CBS has committed to expedite the process as much as possible, but has to rely on other players in the system. The CHS will be watching and working to help find solutions to ensure this product is made available quickly for patients in need. Indeed, the CHS has already proposed potential solutions to health authorities.

Not all decisions need to be fast-tracked, but this one does. If health authorities cannot move quickly in a case like this where the evidence is clear, when can they? In the CHS’ view, expedited approval offers a rare win/win/win scenario (health benefit, quality-of-life improvement, budget savings). Every month that approval of reimbursement is delayed puts limbs and lives at risk and wastes hundreds of thousands of dollars.

The CHS has just learned of further delays in the review process that could mean this life-changing therapy would not be available to patients for many more months. The CHS contacted Provincial/Territorial Ministers of Health on November 22 to let them know this delay is unacceptable and to ask them to intervene to speed up the process so that a decision is made before the end of 2018.

The CHS is reaching out to our community to support the efforts in demanding a rapid review and early reimbursement decision by writing to their Minister of Health and local members of the legislative assembly (MLA) asking that Hemlibra be available by the end of 2018.  Please find more information below:

Key messages for members of the bleeding disorder community

CHS submission to CBS on emicizumab (June 2018) – Executive summary

Sample of letter sent to Provincial/Territorial Ministers of Health on November 22, 2018

Institute for clinical and economic review (ICER) – Emicizumab for Hemophilia A with Inhibitors: Effectiveness and Value