Document ID: chunk:federal_register_of_legislation:F2024L01395:schedule:1:p17
Version: federal_register_of_legislation:F2024L01395
Segment Type: schedule
Provision Reference: sch 1 (pt 17/20)
Character Range: 56904–60432

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 be undergoing treatment with this pharmaceutical benefit with the intent that treatment with the replaced disease modifying agent is/has ceased.
              Patient must be no older than 9 months of age; AND
              Patient must have pre-symptomatic SMA with 3 copies of the SMN2 gene.
              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).
              Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.
              Confirm that a previous PBS authority application has been approved for pre-symptomatic SMA with 3 copies of SMN2 gene.
              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.
              Adhere to any Product Information or local treatment guidelines with respect to treatment-free ('wash out') periods prior to 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