Circular August 2024: Emicizumab clinical criteria for access
Access to be in line with clinical criteria described below, at authorised hospital sites, prescribed by a Pharmaceutical and Therapeutics Committee (PTC) authorised prescriber (preferably by clinical haemotologist). Indication Regimen Monitoring Indication Severe haemophilia A with factor VIII inhibitors, in patients where emicizumab therapy would be cost-neutral or cost-saving compared to the u
Access to be in line with clinical criteria described below, at authorised hospital sites, prescribed by a Pharmaceutical and Therapeutics Committee (PTC) authorised prescriber (preferably by clinical haemotologist).
Indication
Severe haemophilia A with factor VIII inhibitors, in patients where emicizumab therapy would be cost-neutral or cost-saving compared to the use of on-demand bypassing agents for the management of bleeds*; on a patient-by-patient basis.
This could include the following patients:
- Patients who have had an intracranial or other life-threatening bleed
- Patients who have an annualised bleeding rate (ABR) of ≥12 (any bleed**)
*Estimated at R2.5 million annual expenditure per patient (2024 prices). See costing report.
** Any bleeding event defined as:
Major bleeds:
- central nervous system (CNS) – intracranial
- gastrointestinal tract
- urogenital tract including gross haematuria
- severe injury
- neck/throat (airway)
- muscle compartment (e.g. forearm and calf)
- advanced joint and soft tissue
- hip and ilio-psoas muscle
Minor bleeds:
- early joint bleed (Pain/tingling in a joint of a patient with haemophilia suggests bleeding)
- muscle
- soft tissue
- epistaxis
- mild to moderate mouth and gum
- mild to moderate haematuria
| Motivation for use should include the following information: | |
| - Patient details and demographics - Diagnosis of severe haemophilia - Presence of factor VIII inhibitors |
- Clinical status and prognosis - Previous major bleeding episodes - Extent of bypassing agent usage |
Regimen
| Medicine | Emicizumab | |
| Route | Subcutaneous injection | |
| Dose | LOADING DOSE: | 3 mg/kg weekly for 4 weeks |
| MAINTENANCE DOSE: | THEN 1.5 mg/kg weekly OR 3 mg/kg every 2 weeks OR 6 mg/kg monthly |
|
- Current available strength of emicizumab: 30 mg, 60 mg, 105 mg, 150 mg.
- Adhere to the principle of rounding off dosage in mg, to appropriate vial(s), so as not to discard any product.
Monitoring
- Utilisation (of both emicizumab and bypassing agents to treat bleeds) and outcomes should be monitored by responsible Facility and Provincial Pharmaceutical and Therapeutic Committees (PTCs).
Clinical Criteria for Access to Emicizumab - August 2024
Click here for the pdf.