Rare diseases

Authority to supply imatinib mesylate (Glivec®) for rare diseases through the Pharmaceutical Benefits Scheme (PBS) under the Section 85 arrangements of the National Health Act 1953.

Qualifying rare diseases

Imatinib mesylate is available as a PBS subsidised therapy for the treatment of patients with the following rare diseases:

• dermatofibrosarcoma protuberans
• hypereosinophilic syndrome or chronic eosinophilic leukaemia
• Platelet-Derived Growth Factor Receptor (PDGFR) B fusion gene-positive myelodysplastic or myeloproliferative disorder
• aggressive systemic mastocytosis with eosinophilia.

Restriction details

Either of the following two restrictions must apply before approval will be granted to prescribe imatinib mesylate under the PBS:

• initial treatment
  or
• continuing treatment for patients who have previously received treatment with PBS subsidised imatinib mesylate.

The Schedule of Pharmaceutical Benefits outlines the restrictions for prescribing imatinib mesylate for rare diseases.

Item details

Dose

The maximum dose that will be approved for:

• dermatofibrosarcoma protuberans is 800 mg/day
• hypereosinophilic syndrome or chronic eosinophilic leukaemia is 400 mg/day
• PDGFRB fusion gene-positive myelodysplastic or myeloproliferative disorder is 400 mg/day
• aggressive systemic mastocytosis with eosinophilia is 400 mg/day.

^ŦImportant: it is essential that a separate authority prescription is written for each strength of imatinib mesylate tablet.

Continuing therapy is dependent on patients demonstrating a response to imatinib mesylate therapy, irrespective of the daily imatinib mesylate dose received.

Patient eligibility

Patients must meet the relevant restrictions as per the criteria and be eligible to receive pharmaceutical benefits.

Who is eligible for the PBS?

Initial application requirements

 Dermatofibrosarcoma protuberans patients:

• written evidence, if relevant.

Hypereosinophilic syndrome or chronic eosinophilic leukaemia patients, a copy of:

• the pathology report confirming FIP1L1-PDGFRA fusion gene
• the full blood examination report confirming the presence of hypereosinophilic syndrome or chronic eosinophilic leukaemia
• the radiology, nuclear medicine, respiratory function or anatomical pathology report(s) providing details of organs involved.

 PDGFRB fusion gene-positive myelodysplastic or myeloproliferative disorder, a copy of:

• the pathology report confirming the PDGFR gene re-arrangement
• the bone marrow biopsy report which demonstrates the presence of a myelodysplastic or myeloproliferative disorder.

 Aggressive systemic mastocytosis with eosinophilia patients, a copy of:

• the pathology report confirming FIP1L1-PDGFRA fusion gene
• the bone marrow biopsy report and/or other tissue biopsy report confirming the diagnosis of aggressive systemic mastocytosis
• the full blood examination report demonstrating eosinophilia
• the radiology, nuclear medicine, respiratory function or anatomical pathology report(s) providing details of symptomatic organ involvement.

Continuing application requirements

Dermatofibrosarcoma protuberans patients:

• the patient must demonstrate a response to treatment and the disease must remain unresectable.

Hypereosinophilic syndrome or chronic eosinophilic leukaemia patients:

• the patient must achieve and maintain a complete haematological response, with a normal eosinophil count as demonstrated by a copy of the full blood examination, which must be submitted
  and
• the disease must not have progressed whilst on imatinib mesylate treatment.

PDGFRB fusion gene-positive myelodysplastic or myeloproliferative disorder:

• the patient must achieve a complete haematological response as demonstrated by a copy of the full blood examination which must be submitted
  and 
• the disease must not have progressed whilst on imatinib mesylate treatment.

Aggressive systemic mastocytosis with eosinophilia patients:

• the patient must achieve a complete haematological response as demonstrated by a copy of the full blood examination, which must be submitted
  and
• the disease must not have progressed whilst on imatinib mesylate treatment.
  
  

All applicable pathology reports must be from an approved Australian pathology authority.

Lodgement details

All initial and continuing application requests must be in writing and sent to:

Medicare Australia
Prior written approval of specialised drugs
Reply Paid 9826
GPO Box 9826
Hobart TAS 7001

(no stamp required if posted in Australia)

Please ensure that all applications for authorisation include:

• a completed authority prescription form
• a completed supporting information form (for initial applications only includes signed patient and prescriber acknowledgements)
• all relevant pathology reports to support current criteria.

If you require further information please call 1800 700 270* and select option 3 (8 am to 5 pm EST Monday to Friday).

* Call charges apply from mobile and pay phones only. 

Application forms

• Rare diseases imatinib mesylate PBS authority application supporting information form—initial treatment—(Form 4218) [PDF, 213Kb]
  Lodge this form for a patient starting initial treatment of a qualifying rare disease. The patient and prescriber acknowledgements must also be completed.
• Rare diseases imatinib mesylate PBS authority application supporting information form—continuing treatment—(Form 4219) [PDF, 194Kb]
  Lodge this form for a patient undergoing continuing treatment of a qualifying rare disease.

Important: information on this website will be updated regularly. Please keep checking this site for updates.

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Last updated: 19 March, 2010

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