Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p273
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 273/476)
Character Range: 2557122–2566272

Patient must be untreated with gene therapy.
                                                                                                                                            Patient must be 18 years of age or under.
                                                                                                                                            Defined signs and symptoms of type I SMA are:
                                                                                                                                            i) Onset before 6 months of age; and
                                                                                                                                            ii) Failure to meet or regression in ability to perform age‑appropriate motor milestones; or
                                                                                                                                            iii) Proximal weakness; or
                                                                                                                                            iv) Hypotonia; or
                                                                                                                                            v) Absence of deep tendon reflexes; or
                                                                                                                                            vi) Failure to gain weight appropriate for age; or
                                                                                                                                            vii) Any active chronic neurogenic changes; or
                                                                                                                                            viii) A compound muscle action potential below normative values for an age‑matched child.
                                                                                                                                            Defined signs and symptoms of type II SMA are:
                                                                                                                                            i) Onset between 6 and 18 months; and
                                                                                                                                            ii) Failure to meet or regression in ability to perform age‑appropriate motor milestones; or
                                                                                                                                            iii) Proximal weakness; or
                                                                                                                                            iv) Weakness in trunk righting/derotation; or
                                                                                                                                            v) Hypotonia; or
                                                                                                                                            vi) Absence of deep tendon reflexes; or
                                                                                                                                            vii) Failure to gain weight appropriate for age; or
                                                                                                                                            viii) Any active chronic neurogenic changes; or
                                                                                                                                            ix) A compound muscle action potential below normative values for an age‑matched child.
                                                                                                                                            Defined signs and symptoms of type IIIa SMA are:
                                                                                                                                            i) Onset between 18 months and 3 years of age; and
                                                                                                                                            ii) Failure to meet or regression in ability to perform age‑appropriate motor milestones; or
                                                                                                                                            iii) Proximal weakness; or
                                                                                                                                            iv) Hypotonia; or
                                                                                                                                            v) Absence of deep tendon reflexes; or
                                                                                                                                            vi) Failure to gain weight appropriate for age; or
                                                                                                                                            vii) Any active chronic neurogenic changes; or
                                                                                                                                            viii) A compound muscle action potential below normative values for an age‑matched child.
                                                                                                                                            Application for authorisation of initial treatment must be in writing and must include:
                                                                                                                                            (a) a completed authority prescription form; and
                                                                                                                                            (b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
                                                                                                                                            i) specification of SMA type (I, II or IIIa); and
                                                                                                                                            (ii) sign(s) and symptom(s) that the patient has experienced; and
                                                                                                                                            (iii) patient's age at the onset of sign(s) and symptom(s).
                                                                       C12676                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Initial treatment occurring after onasemnogene abeparvovec therapy in a patient with one of: (i) Type 1 SMA, or, (ii) pre‑symptomatic SMA
                                                                                                                                            Patient must have experienced a regression in a developmental state listed below (see 'Definition') despite treatment with gene therapy ‑ confirm that this: (i) not due to an acute concomitant illness; (ii) not due to non‑compliance to best‑supportive care, (iii) apparent for at least 3 months, (iv) verified by another clinician in the treatment team ‑ state the full name of this clinician plus their profession (e.g. medical practitioner, nurse, physiotherapist; this is not an exhaustive list of examples); 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