Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p348
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 348/476)
Character Range: 3089763–3096507

including, but not limited to, level of independence. Quality of life may be informed by use of the SMA Health Index (SMA‑HI) or SMA Functional Rating Scale (SMAFRS).
                                                                       C14435                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Written Authority Required procedures
                                                                                                                                            Initial treatment occurring after onasemnogene abeparvovec therapy in a patient with Type 1 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 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; AND
                                                                                                                                            The treatment must not be in combination with PBS‑subsidised treatment with nusinersen for this condition.
                                                                                                                                            Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic; AND
                                                                                                                                            Patient must be undergoing treatment under this Treatment phase listing once only ‑ for continuing treatment beyond this authority application, refer to the drug's relevant 'Continuing treatment' listing for the patient's SMA type.
                                                                                                                                            Patient must have a prior authority approval for any drug PBS‑listed for symptomatic Type 1 SMA, with at least one approval having been for gene therapy.
                                                                                                                                            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).
                                                                                                                                            Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.
                                                                                                                                            Confirm that a previous PBS authority application has been approved for symptomatic Type 1 SMA.
                                                                                                                                            Definition:
                                                                                                                                            Various childhood developmental states (1 to 9) are listed below, some followed by further observations (a up to d). Where at least one developmental state/observation is no longer present, that developmental state has regressed.
                                                                                                                                            0. Absence of developmental states (1 to 9) listed below:
                                                                                                                                            1. Rolls from side to side on back;
                                                                                                                                            2. Child holds head erect for at least 3 seconds unsupported;
                                                                                                                                            3. Sitting, but with assistance;
                                                                                                                                            4. Sitting