Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Spinal muscular atrophy (SMA)

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; AND

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 Scale - Expanded (HFMSE) or 6-minute walk test (6MWT), and the patient's quality of life 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).