Document ID: chunk:federal_register_of_legislation:F2025C00124:clause:3_1:p279
Version: federal_register_of_legislation:F2025C00124
Segment Type: clause
Provision Reference: sch 3 cl 1 (pt 279/476)
Character Range: 2600405–2606938

be ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug.
                                                                                                                                            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 continuation of existing PBS‑subsidised treatment with this drug; AND
                                                                                                                                            Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS‑subsidised disease modifying treatment.
                                                                                                                                            Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day.
                                                                       C14459                                                               Spinal muscular atrophy (SMA)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                         Compliance with Authority Required procedures
                                                                                                                                            Continuing/maintenance treatment in an adult where treatment was initiated in adulthood
                                                                                                                                            The treatment must be each of: (i) occurring from week 104 onwards relative to the first administered dose, (ii) demonstrating a clinically meaningful response; OR
                                                                                                                                            The treatment must be occurring within the first 104 weeks from the first administered dose; AND
                                                                                                                                            Patient must not be receiving invasive permanent assisted ventilation in the absence of a potentially reversible cause while being treated with this drug.
                                                                                                                                            Must be treated by a specialist medical practitioner experienced in the diagnosis/management of SMA; OR
                                                                                                                                            Must be treated by a medical practitioner who has been directed to prescribe this benefit by a specialist medical practitioner experienced in the diagnosis/management of SMA; AND
                                                                                                                                            Patient must be undergoing continuation of existing PBS‑subsidised treatment with this drug; AND
                                                                                                                                            Patient must be undergoing concomitant treatment with best supportive care, but this benefit is the sole PBS‑subsidised disease modifying treatment.
                                                                                                                                            Where this authority application seeks to continue treatment beyond the first 104 weeks of treatment, comprehensive assessment must be undertaken periodically and documented, involving the patient and the treating physician to establish agreement that treatment is continuing to produce a clinically meaningful response.
                                                                                                                                            A clinically meaningful response is present where an improvement, stabilisation or minimal decline in symptoms has occurred as a result of this drug treatment and where there is agreement between the treating physician and patient over what constitutes improvement, stabilisation, or minimal decline.
                                                                                                                                            PBS subsidy must cease if there is no agreement on whether a clinically meaningful response is present.
                                                                                                                                            Undertake re‑assessments for a clinically meaningful response at least every six months. Document these re‑assessments in the patient's medical records.
                                                                                                                                            In undertaking comprehensive assessments, where practical, a clinically meaningful response assessment encompasses the patient's motor function as assessed using an instrument like the Revised Upper Limb Module (RULM), Hammersmith Functional Motor