Ontario has approved access to HEMGENIX, the first gene therapy for people with hemophilia B. British Columbia has also opened access, while other provinces continue their reviews. Because HEMGENIX is not distributed through Canadian Blood Services, access is handled through provincial drug programs rather than the usual factor‑replacement channels.
For people in Ontario, the process involves several coordinated steps between the individual, their hematologist, and the provincial Exceptional Access Program. BC follows a similar overall structure, but the details below focus specifically on Ontario’s requirements.
Ontario’s Access Pathway
1. Shared decision‑making
- Individuals meet with their hematologist to discuss whether gene therapy is appropriate.
- This includes reviewing potential benefits, risks, long‑term monitoring, and personal treatment goals.
2. Eligibility assessment
- Hematologists confirm whether the person meets clinical criteria, including:
- Age 18 or older
- Currently using prophylaxis
- AAV5 antibody levels below the required threshold
- Additional medical considerations may apply.
3. Enrollment in CSL PLUS™
- This support program helps coordinate reimbursement steps, travel assistance (when applicable), and follow‑up monitoring.
- It works alongside the hemophilia treatment centre team.
4. Registration with Ontario’s drug program
- Individuals must be registered with the Trillium Drug Program, which is required for coverage of HEMGENIX.
5. Physician application through Ontario’s Exceptional Access Program
- The hematologist submits a case‑by‑case request to the Exceptional Access Program (EAP)
Hemophilia treatment centres across Canada have been briefed on these processes and can guide individuals through each step. The Canadian Hemophilia Society continues to monitor provincial decisions and will share updates as they become available.
