Document ID: chunk:federal_register_of_legislation:F2025C00158:clause:4_1:p150
Version: federal_register_of_legislation:F2025C00158
Segment Type: clause
Provision Reference: sch 4 cl 1 (pt 150/161)
Character Range: 14595206–14601830

drug is not PBS-subsidised if it is prescribed to an in-patient in a public hospital setting.
C13868              P13868         CN13868          Sapropterin                    Maternal hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                        Compliance with Authority Required procedures
                                                                                   Initial treatment - responsiveness testing
                                                                                   The treatment must be for the purpose of ascertaining the patient's response to treatment over a period of 7 days, with the intent to then use the drug to control phenylalanine levels under the treatment phase:
                                                                                    First continuing treatment, Indication: Hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU); AND
                                                                                   Patient must have a baseline blood phenylalanine level above 250 micromol/L prior to commencing treatment with this drug despite best efforts to rely on dietary modifications to control phenylalanine levels; AND
                                                                                   Must be treated by a metabolic physician; AND
                                                                                   Patient must be undergoing treatment with this drug for the first time; AND
                                                                                   Patient must not be undergoing treatment with this drug under this Treatment phase, more than once per lifetime following completion of this authority application; AND
                                                                                   Patient must not be undergoing simultaneous treatment with this drug under another PBS-listing (apply under either listing type, but not both simultaneously);
                                                                                   Patient must be one of:
                                                                                    (i) planning conception, (ii) pregnant.
C13876              P13876         CN13876          Ruxolitinib                    Grade II to IV acute graft versus host disease (aGVHD)                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                    Compliance with Authority Required procedures - Streamlined Authority Code 13876
                                                                                   Continuing treatment
                                                                                   Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND
                                                                                   Patient must have responding disease compared with baseline after 14 days of treatment demonstrated by either a:
                                                                                    (i) partial response (ii) complete response; AND
                                                                                   Must be treated by a haematologist.  or
                                                                                   Must be treated by an oncologist with allogeneic bone marrow transplantation experience.  or
                                                                                   Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types.
                                                                                   Response is defined as attaining a complete or partial response as assessed by Mount Sinai Acute GVHD International Consortium (MAGIC) criteria (Harris et al., 2016). Note that response is relative to the assessment of organ function affected by aGVHD prior to commencing initial treatment with ruxolitinib.
                                                                                   (a) complete response is defined as a score of 0 for the aGVHD grade in all evaluable organs, indicating a complete resolution of all signs and symptoms of aGVHD, without the administration of any additional systemic therapies for any earlier progression, mixed response or non-response of aGVHD.
                                                                                   (b) partial response is defined as an improvement of one stage, in at least one of the evaluable organs involved with aGVHD signs or symptoms, without disease progression in other organs or sites and without the administration of additional systemic therapies for any earlier progression, mixed response, or non-response of aGVHD.
                                                                                   The assessment of response must be documented in the