Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p276
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 276/476)
Character Range: 2579047–2586377

potentially reversible cause while being treated with this drug.
                                                                                                                                            Patient must be of an age that is prior to their 19thbirthday at the time of this authority application; AND
                                                                                                                                            Patient must have SMA type III where the onset of signs/symptoms of SMA first occurred after their 3rdbirthday, but before their 19thbirthday (SMA type IIIB/IIIC).
                                                                                                                                            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
                                                                                                                                            Patient must be undergoing initial PBS‑subsidised treatment for untreated disease ‑ prescribe up to 3 repeat prescriptions to enable dosing occurring at days: 0 (original prescription), 14 (repeat 1), 28 (repeat 2), 63 (repeat 3) (i.e. the loading doses); OR
                                                                                                                                            Patient must be undergoing initial PBS‑subsidised treatment, but the patient has initiated treatment via non‑PBS supply (e.g. clinical trial, sponsor compassionate access) ‑ prescribe zero repeat prescriptions where loading doses are complete; AND
                                                                                                                                            Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS‑subsidised disease modifying treatment.
                                                                                                                                            The authority application must be made in writing and must include:
                                                                                                                                            (1) a completed authority prescription form; 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).
                                                                                                                                            Signs and symptoms of spinal muscular atrophy in the context of this PBS restriction are:
                                                                                                                                            (i) Failure to meet or regression in ability to perform age‑appropriate motor milestones,
                                                                                                                                            (ii) Proximal weakness,
                                                                                                                                            (iii) Hypotonia,
                                                                                                                                            (iv) Absence of deep tendon reflexes,
                                                                                                                                            (v) Any active denervation or chronic neurogenic changes found on electromyography,
                                                                                                                                            (vi) A compound muscle action potential below normative values for an age‑matched child.
                                                                                                                                            In this authority application, confirm:
                                                                                                                                            (1) the patient's medical history is consistent with a diagnosis of type IIIB/IIIC spinal muscular atrophy,
                                                                                                                                            (2) which of the above (i to vi) (at least 1) were present after their 3rdbirthday, but before their 19thbirthday,
                                                                                                                                            (3) the age of the patient (rounded to the nearest year) when the first sign/symptom was observed.
                                                                       C13270                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Initial PBS‑subsidised treatment in an adult who did not initiate PBS subsidy during childhood
                                                                                                                                            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
                                                                                                                                            Patient