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Spinal muscular atrophy (SMA) Program in Pharmaceutical Benefits Scheme (PBS) 012-18053100



SMA restriction and item codes

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PBS Complex Drugs Programs

Zolgensma® checklist

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FAQs from Service Officers

Table 1: see Table 2 in Processing Complex Authority Required Listings for FAQs about Complex programs. In the following table 'you' refers to the Service Officer.

Item

Description

1

You have received a delayed assessment initial SMA application for nusinersen or risdiplam, but there is no form attached. Do you reject the application?

No. As of 1 November 2024, all initial requests for SMA medicines can be made using Online PBS Authorities (OPA) system as a delayed assessment. An application form is not required when prescribers submit as a delayed assessment.

2

Does the prescribing doctor for SMA treatment have to be a specialist?

No. Treatment can be initiated by or in consultation with the required specialist.

3

You receive a telephone call from a prescriber requesting a continuing approval for nusinersen. You remember that this is for SMA, and the medications are high cost items. Do you escalate this call?

No. Process the application as normal, asking the required questions in OPA.

4

What is the initial/loading dosing schedule for nusinersen?

Quantity of 1 vial and 3 repeats - 12mg dosed at days 0, 14, 28 & 63.

5

What is the standard maintenance dose of nusinersen and what to enter in OPA system?

Quantity of 1 vial and 0 repeat. Dosing to enter in OPA - 1 every 4 months.

6

What’s the quantity and repeats that can be approved for a pre-symptomatic SMA patient changing from risdiplam to nusinersen?

  • Approve quantity of 1 vial and 0 repeat if loading is not intended
  • Override the OPA rejection and approve quantity of 1 vial and 3 repeats if loading doses are requested

7

A prescriber enquires about a continuing application for nusinersen, and how can he prescribe this item. What do you advise?

The application can be done via self-serve or over the telephone.

8

Can an adult >18 years be treated under the initial criteria for risdiplam?

Yes, if the patient had the onset of SMA during childhood but never had PBS-subsidised treatment during childhood.

9

You have received a written applications for risdiplam for a patient weighing 20kg. The prescriber requests a quantity of 4, How do you proceed if all criteria met?

Approve with a change in quantity from 4 to 3. Dosing of risdiplam is weight based and no increase in the maximum quantity of 3 is allowed.

10

You have received a written application for onasemnogene abeparvovec (Zolgensma®). The patient’s weight is 3.7kg. What would be the total number of 8.3ml vials you could approve?

All applications for Zolgensma® must be escalated to Pharmaceutical Advisor (PA) for approval. The answer is 2 to the question if being asked.