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Patient eligibility
The Pharmaceutical Benefits Scheme (PBS) subsidises treatment with nusinersen, onasemnogene abeparvovec, and risdiplam under the National Health Act 1953, section 100 for patients with SMA.
Where the term ‘disease modifying therapy’ appears it refers to nusinersen and risdiplam. Where the term ‘gene therapy’ appears it refers to onasemnogene abeparvovec.
Nusinersen is PBS-subsidised for both symptomatic and pre-symptomatic SMA, whereas risdiplam is only PBS-subsidised for symptomatic SMA.
Onasemnogene abeparvovec is PBS-subsidised gene therapy for both symptomatic Type 1 SMA and pre-symptomatic SMA for patients no older than 9 months of age. Treatment with onasemnogene abeparvovec is once only in a lifetime.
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 nusinersen, onasemnogene abeparvovec, and risdiplam.
Section 100 arrangements
Nusinersen, onasemnogene abeparvovec, and risdiplam
These items are only PBS-subsidised for non-admitted patients, day admitted patients, or patients on discharge who are attending:
- an approved private hospital (nusinersen and risdiplam only)
- 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
Nusinersen for patients aged 18 years or younger
To be eligible for PBS-subsidised treatment with nusinersen for initial treatment, patients must be treated by or in consultation with a specialist medical practitioner who is both:
- experienced in diagnosing and managing SMA
- associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
For continuing treatment, patients must either be treated by, or have their treatment initiated, in consultation with a specialist medical practitioner who is experienced in diagnosing and managing SMA and associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
Hospitals recognised for managing SMA are:
- Queensland Children’s Hospital - Brisbane
- Royal Children’s Hospital - Melbourne
- Monash Children’s Hospital - Melbourne
- John Hunter Hospital - Newcastle
- Sydney Children’s Hospital - Randwick
- Children’s Hospital at Westmead
- Adelaide Women and Children’s Hospital
- Perth Children’s Hospital.
Nusinersen for adult patients with onset of SMA prior to 19 years of age
To be eligible for PBS-subsidised treatment with nusinersen for initial and continuing treatment, patients must be treated by:
- a specialist medical practitioner experienced in the diagnosis/management of SMA, or
- a medical practitioner directed to prescribe this benefit by a specialist medical practitioner experienced in the diagnosis/management of SMA.
Onasemnogene abeparvovec
To be eligible for PBS-subsidised treatment with onasemnogene abeparvovec for patients either untreated with disease modifying therapies or switching from disease modifying therapies to gene therapy, they must be treated by or in consultation with a specialist medical practitioner who is both:
- experienced in diagnosing and managing SMA
- associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
Hospitals recognised for managing SMA are:
- Queensland Children’s Hospital - Brisbane
- Royal Children’s Hospital - Melbourne
- Monash Children’s Hospital - Melbourne
- John Hunter Hospital - Newcastle
- Sydney Children’s Hospital - Randwick
- Children’s Hospital at Westmead
- Adelaide Women and Children’s Hospital
- Perth Children’s Hospital.
Patients must be treated in a treatment centre that is each of the following:
- recognised in managing SMA
- accredited in using this gene technology by the relevant authority
- has sourced or will source this product from an accredited supplier.
Accredited treatment centres and suppliers are those organisations accredited by the Gene Technology Regulator under section 92 of the Gene Technology Act 2000. The list of accredited organisations is available on the Department of Health and Aged Care website. These may be updated without notice.
Risdiplam
To be eligible for PBS-subsidised treatment with risdiplam for both initial and continuing treatment, patients must be treated by or in consultation with a specialist medical practitioner who is both:
- experienced in diagnosing and managing SMA
- associated with a neuromuscular clinic.
Authority applications
Disease modifying therapy
Patients 18 years of age or younger
Applying for initial treatment
Apply for initial authority approval to prescribe PBS-subsidised nusinersen or risdiplam for patients aged 18 years or younger to treat SMA 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
- spinal muscular atrophy - nusinersen or risdiplam - 18 years or younger - initial authority application form
- relevant attachments.
Applying for continuing treatment
Applications for continuing authority approval to prescribe PBS-subsidised nusinersen or risdiplam to treat SMA can be made either:
- in real time using the Online PBS Authorities system
- by calling the PBS Complex Drug Programs enquiry line.
Applying for change of treatment
Applications to change authority approval to prescribe PBS-subsidised nusinersen or risdiplam to treat symptomatic type I, II or IIIa SMA can be made either:
- in real time using the Online PBS Authorities system
- by calling the PBS Complex Drug Programs enquiry line.
Patients 19 years of age or older
Applying for initial treatment
Apply for initial authority approval to prescribe PBS-subsidised nusinersen for adult patients who did not initiate PBS subsidy during childhood to treat SMA 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
- spinal muscular atrophy adult - nusinersen - initial authority application form
- relevant attachments
Applying for continuing treatment
Applications to continue authority approval to prescribe PBS-subsidised nusinersen to treat SMA can be made either:
- in real time using the Online PBS Authorities system
- by calling the PBS Complex Drug Programs enquiry line.
Gene therapy
Apply for authority approval to prescribe PBS-subsidised onasemnogene abeparvovec to treat SMA 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
- spinal muscular atrophy - onasemnogene abeparvovec authority application form.
You must state the weight of the patient in kilograms and request the appropriate product pack presentation with respect to the mix of 5.5 mL and 8.3 mL vials.
More information
Call the PBS Complex Drugs Programs enquiry line for more information.