Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p304
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 304/476)
Character Range: 2767772–2774264

administering this benefit.
                                                                       C16045                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Use in a patient untreated with disease modifying therapies for this condition
                                                                                                                                            The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
                                                                                                                                            The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
                                                                                                                                            The condition must be pre-symptomatic SMA, with genetic confirmation that there are 3 copies of the survival motor neuron 2 (SMN2) gene; AND
                                                                                                                                            The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes; AND
                                                                                                                                            The treatment must be given concomitantly with best supportive care for this condition.
                                                                                                                                            Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; AND
                                                                                                                                            Must be treated in a treatment centre that is each of: (i) recognised in the management of SMA, (ii) accredited in the use of this gene technology by the relevant authority, (iii) will(has) source(d) this product from an accredited supplier, as specified in the administrative notes to this listing; AND
                                                                                                                                            Patient must be undergoing treatment with this pharmaceutical benefit once only in a lifetime; AND
                                                                                                                                            Patient must not be undergoing treatment with this pharmaceutical benefit through this listing where prior treatment has occurred with any of: (i) nusinersen, (ii) risdiplam.
                                                                                                                                            Patient must be no older than 9 months of age.
                                                                                                                                            The authority application must be made in writing and must include:
                                                                                                                                            (1) details of the proposed prescription; and
                                                                                                                                            (2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).
                                                                                                                                            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.
                                                                                                                                            Confirm that genetic testing has been completed to demonstrate the following in support of an SMA diagnosis:
                                                                                                                                            (i) 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; or
                                                                                                                                            (ii) deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variance in the remaining single copy of the SMN1 gene.
                                                                                                                                            Confirm that there is a genetic test