Von Hippel-Lindau (VHL) disease

Treatment Phase: Initiating or recommencing treatment

Clinical criteria:

• The condition must have been diagnosed by at least one of: (i) a germline VHL alteration; (ii) at least two manifestations highly characteristic of VHL disease; (iii) at least one manifestation highly characteristic of VHL disease with a documented family history of VHL,

AND

• The condition must be at least one of: (i) VHL-associated non-metastatic renal cell carcinoma (RCC); (ii) VHL-associated central nervous system (CNS) haemangioblastoma; (iii) VHL-associated non-metastatic pancreatic neuroendocrine tumour (pNET),

AND

• Patient must not have tumour(s) that require immediate surgery as assessed by the treating clinician,

AND

• Patient must be untreated with this drug for this condition; OR

• Patient must have previously received PBS-subsidised treatment with this drug for this condition for a different tumour type; OR

• Patient must have previously received PBS-subsidised treatment with this drug for this condition and ceased previous treatment for family planning purposes,

AND

• Patient must have WHO performance status no higher than 1; OR

• The condition must be VHL-associated brainstem tumour(s), or brain herniation, which temporarily affected the patient's WHO performance status to be higher than 1,

AND

• The treatment must be the sole PBS-subsidised therapy for VHL disease associated tumours.

Treatment criteria:

• Must be treated by a physician with expertise in the management of VHL disease associated tumours.

Patients who cease therapy for reasons other than, clinical disease progression or metastasis, may re-initiate PBS-subsidised treatment through the initiating or recommencing treatment phase.

For the purpose of administering this restriction, the highly characteristic manifestations of VHL disease include but not limited to:

(i) retinal, spinal, or cerebellar haemangioblastoma;

(ii) adrenal or extra-adrenal phaeochromocytoma;

(iii) renal cell carcinoma;

(iv) multiple renal and pancreatic cysts;

(v) endolymphatic sac tumours, papillary cystadenomas of the epididymis or broad ligament, or pancreatic neuroendocrine tumours.

Von Hippel-Lindau (VHL) disease

Treatment Phase: Continuing treatment

Clinical criteria:

• Patient must have previously received PBS-subsidised treatment with this drug for this condition for the same tumour type,

AND

• Patient must not have developed VHL-associated metastatic disease,

AND

• Patient must have demonstrated clinical improvement or stabilisation of the condition while being treated with this drug, the details of which must be kept with the patient's record,

AND

• The treatment must be the sole PBS-subsidised therapy for VHL disease associated tumours.

Treatment criteria:

• Must be treated by a physician with expertise in the management of VHL disease associated tumours.

Patients who cease therapy for reasons other than, clinical disease progression or metastasis, may re-initiate PBS-subsidised treatment through the initiating or recommencing treatment phase.

For the purpose of administering this restriction, clinical improvement or stabilisation of the patient's condition includes but is not limited to:

(i) avoidance of surgery;

(ii) avoidance of renal replacement therapy such as dialysis or renal transplantation in patients with VHL- associated renal cell carcinoma (RCC);

(iii) experiencing clinical benefit in at least one of the VHL associated conditions, as determined by the treating clinician(s).