Paroxysmal nocturnal haemoglobinuria

Patient eligibility

The Pharmaceutical Benefits Scheme (PBS) subsidises treatment with eculizumab, pegcetacoplan or ravulizumab under the National Health Act 1953, section 100 for patients with paroxysmal nocturnal haemoglobinuria (PNH). Patients must be eligible for the PBS and meet the relevant restriction criteria.

The Schedule of Pharmaceutical Benefits on the PBS website outlines the restrictions for prescribing eculizumab, pegcetacoplan or ravulizumab.

Section 100 arrangements

eculizumab, pegcetacoplan or ravulizumab

These items are only PBS-subsidised for non-admitted patients, day admitted patients, or patients on discharge who are attending:

• an approved private hospital
• a public participating hospital
• a public hospital.

These items aren’t PBS-subsidised for public hospital in-patients. You must include the hospital name and provider number on the authority application form.

Treatment specifics

To be eligible for PBS-subsidised treatment with these medications, patients must be treated by a haematologist or by a non-specialist medical practitioner in consultation with a haematologist.

Complement 5 (C5) inhibitors are defined as eculizumab or ravulizumab

Patients switching from a C5 inhibitor to pegcetacoplan are required to take pegcetacoplan in addition to their current dose of C5 inhibitor for the first 4 weeks of treatment. This minimises the risk of haemolysis occurring when treatment stops abruptly. After 4 weeks, the patient should discontinue treatment with the C5 inhibitor before continuing on monotherapy with pegcetacoplan.

Authority applications

C5 inhibitors - eculizumab and ravulizumab

Applying for initial, switching, returning or balance of supply treatment

Apply for initial, switching, returning or balance of supply authority approval to prescribe PBS-subsidised eculizumab or ravulizumab to treat paroxysmal nocturnal haemoglobinuria in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All written applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab - initial, switching, returning or balance of supply authority application form
• relevant attachments.

Applying for initial grandfather treatment

For patients who received non-PBS-subsidised eculizumab or ravulizumab treatment before 1 March 2022 for paroxysmal nocturnal haemoglobinuria, apply for initial grandfather authority approval in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All written applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab - initial grandfather authority application form
• relevant attachments.

Applying for continuing treatment

Apply for continuing authority approval to prescribe PBS-subsidised eculizumab or ravulizumab to treat paroxysmal nocturnal haemoglobinuria in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab continuing authority application form
• relevant attachments.

Pegcetacoplan

Applying for initial treatment

Apply for initial authority approval to prescribe PBS-subsidised pegcetacoplan to treat paroxysmal nocturnal haemoglobinuria in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All written applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - pegcetacoplan - initial authority application form
• relevant attachments.

relevant attachments.

Applying for initial grandfather treatment

For patients who received non-PBS-subsidised pegcetacoplan treatment before 1 December 2022 for paroxysmal nocturnal haemoglobinuria, apply for initial grandfather authority approval in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All written applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - pegcetacoplan - initial grandfather authority application form
• relevant attachments.

Applying for continuing or returning treatment

Apply for continuing or returning authority approval to prescribe PBS-subsidised pegcetacoplan to treat paroxysmal nocturnal haemoglobinuria in writing and either:

• use HPOS Form upload
• mail to PBS Complex Drugs Programs.

All applications must include the completed:

• authority prescription form or forms
• paroxysmal nocturnal haemoglobinuria - pegcetacoplan - continuing or returning authority application form
• relevant attachments

More information

Call the PBS Complex Drugs Programs enquiry line for more information.