Pharmacoeconomic report: Emicizumab (Hemlibra) Hoffman-La Roche Ltd. : indication: bleeding prevention, hemophilia A.
The Health Canada indication for emicizumab includes patients with hemophilia A regardless of their disease severity, while the sponsor's submitted reimbursement request includes only severe patients and mild and moderate patients who meet specific eligibility criteria. The modelled population...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
Canadian Agency for Drugs and Technologies in Health
February 2021, 2021
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| Edition: | Final |
| Series: | CADTH drug reimbursement review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | The Health Canada indication for emicizumab includes patients with hemophilia A regardless of their disease severity, while the sponsor's submitted reimbursement request includes only severe patients and mild and moderate patients who meet specific eligibility criteria. The modelled population only investigated patients with severe hemophilia A, as per HAVEN 3's eligibility criteria. Given that the sponsor's reimbursement request does not align with the modelled population, uncertainty remains regarding the cost-effectiveness of emicizumab in both the reimbursement-requested population and the full Health Canada indication CADTH undertook reanalysis to address limitations in the sponsor's submission, including adjusting dispensing to the nearest vial size, removing treatment-specific utility estimates, and setting patients' starting ages and weights to better reflect the HAVEN 3 patient population. CADTH's findings remained aligned with the sponsor's: emicizumab is not a cost-effective option at a willingness-to-pay threshold of $50,000 per quality-adjusted life-year (QALY) in patients with severe hemophilia A without inhibitors. In the CADTH base-case reanalysis, emicizumab was associated with an incremental cost-effectiveness ratio (ICER) of $5.53 million per QALY gained compared to factor VIII (FVIII) prophylaxis. The model was highly sensitive to the price of emicizumab and its comparators. To achieve an ICER of $50,000 per QALY, the price of emicizumab would need to be reduced by at least 89%. However, a greater price reduction may be required if the prices of FVIII products are lower than those published by the Patented Medicine Prices Review Board (PMPRB). While the evidence for bleeding efficacy is robust for the use of emicizumab in Canadian patients not receiving treatment with prophylactic FVIII, the evidence directly comparing emicizumab to prophylactic FVIII is limited. In patients with severe hemophilia A without inhibitors, emicizumab demonstrated statistically and clinically significant improvements in bleeding outcomes (i.e., annualized bleeding ratio for treated bleeds, all bleeds, treated joint bleeds, and treated spontaneous bleeds) compared to on-demand FVIII treatment. Limited comparative evidence exists to establish the comparative effectiveness and safety of emicizumab compared to FVIII prophylaxis, although current evidence suggests that emicizumab showed a reduction in bleeding outcomes compared to no prophylaxis (non-randomized comparison). |
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| Physical Description: | 1 PDF file (39 pages) illustrations |