Decision to widen access and change the funded enzyme replacement therapy for Gaucher disease

Special Authority for subsidy

Special Authority approved by the Gaucher Treatment Panel

Application details may be obtained from PHARMAC’s website or:

The Co-ordinator, Gaucher Treatment Panel
PHARMAC, PO Box 10 254
Wellington

Phone:      (04) 460 4990

Facsimile: (04) 916 7571

Email:        gaucherpanel@pharmac.govt.nz

Completed application forms must be sent to the coordinator for the Gaucher Treatment Panel and will be considered by Gaucher Treatment Panel at the next practicable opportunity.

Notification of Gaucher Treatment Panel’s decision will be sent to the patient, the applying clinician and the patient's GP (if specified).

Access Criteria

Initial application from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

1. The patient has a diagnosis of symptomatic type 1 or type 3* Gaucher disease confirmed by the demonstration of specific deficiency of glucocerebrosidase in leukocytes or cultured skin fibroblasts, and genotypic analysis; and
2. Patient does not have another life-threatening or severe disease where the prognosis is unlikely to be influenced by taliglucerase alfa or might be reasonably expected to compromise a response to therapy with taliglucerase alfa; and
3. Taliglucerase alfa is to be administered at a dose no greater than 30 unit/kg every other week rounded to the nearest whole vial (200 units), unless otherwise agreed by PHARMAC; and
4. Supporting clinical information including test reports, MRI whole body STIR, serum glucosylsphingosine, haematological data, and other relevant investigations, are submitted to the Gaucher Panel for assessment; and
5. Any of the following:
   1. Patient has haematological complications such as haemoglobin less than 95 g/l, symptomatic anaemia, thrombocytopenia; at least two episodes of severely symptomatic splenic infarcts confirmed with imagery; or massive symptomatic splenomegaly; or
   2. Patient has skeletal complications such as acute bone crisis requiring hospitalisation or major pain management strategies; radiological MRI Evidence of incipient destruction of any major joint (e.g. hips or shoulder); spontaneous fractures or vertebral collapse; chronic bone pain not controlled by other pharmaceuticals; or
   3. Patient has significant liver dysfunction or hepatomegaly attributable to Gaucher disease; or
   4. Patient has reduced vital capacity from clinically significant or progressive pulmonary disease due to Gaucher disease; or
   5. Patient is a child and has experienced growth failure with significant decrease in percentile linear growth over a 6-12 month period.

*Unapproved indication

Renewal from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

1. Patient has demonstrated a symptomatic improvement or no deterioration in the main symptom for which therapy was initiated; and
2. Patient has demonstrated a clinically objective improvement or no deterioration in haemoglobin levels, platelet counts and liver and spleen size; and
3. Radiological (MRI) signs of bone activity performed at one year and two years since initiation of treatment begins, and two to three yearly thereafter, demonstrate no deterioration shown by the MRI, compared with MRI taken immediately prior to commencement of therapy or adjusted dose; and
4. Serum glucosylsphingosine levels taken at least 6 to 12 monthly show a decrease compared with baseline; and
5. Patient has not had severe infusion-related adverse reactions which were not preventable by appropriate pre-medication and/or adjustment of infusion rates; and
6. Patient has not developed another medical condition that might reasonably be expected to compromise a response to ERT; and
7. Patient is compliant with regular treatment and taliglucerase alfa is to be administered at a dose no greater than 30 unit/kg every other week rounded to the nearest whole vial (200 units), unless otherwise agreed by PHARMAC; and
8. Supporting clinical information including test reports, MRI whole body STIR, serum glucosylsphingosine, haematological data, and other relevant investigations are submitted to the Gaucher Panel for assessment as required.