Rare diseases

The Pharmaceutical Benefits Scheme (PBS) subsidises imatinib mesylate for patients with rare diseases.

For patients with rare diseases, treatment with imatinib mesylate can be subsidised through the PBS under section 85 of the National Health Act 1953.

Initial treatment

The following rare diseases are eligible for initial treatment with imatinib mesylate:

  • dermatofibrosarcoma protuberans (DFSP)
  • hypereosinophilic syndrome or chronic eosinophilic leukaemia (HES/CEL)
  • platelet-derived growth factor receptor (PDGFR) B fusion gene-positive myelodysplastic or myeloproliferative disorder (MDS/MPD), and
  • aggressive systemic mastocytosis (ASM) with eosinophilia

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

Continued treatment

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

Schedule item details

Dose - The maximum dose that will be approved for:

  • dermatofibrosarcoma protuberans is 800 mg per day
  • hypereosinophilic syndrome or chronic eosinophilic leukaemia is 400 mg per day
  • PDGFR B fusion gene-positive myelodysplastic or myeloproliferative disorder is 400 mg per day, and
  • aggressive systemic mastocytosis with eosinophilia is 400 mg per day

The table below is a guide to prescribing imatinib mesylate for rare diseases.

Name and dose per day Daily dose Tablet strength Authority prescription Quantity Maximum repeats
Dermatofibrosarcoma protuberans (maximum 800 mg per day) 400 mg 400 mg 1 x 400 mg 30 2
100 mg 4 x 100 mg 120 2
600 mg 100 mg 6 x 100 mg 180 2
or      
400 mg 1 x 400 mg 30 2
and      
100 mg 2 x 100 mg 60 2
800 mg 400 mg 2 x 400 mg 60 2
  100 mg 8 x 100 mg 240 2
Hypereosinophilic syndrome or chronic eosinophilic leukaemia (maximum 400 mg per day) 400 mg 400 mg 1 x 400 mg 30 2
  100 mg 4 x 100 mg 120 2
PDGFR B fusion gene-positive myelodysplastic or myeloproliferative disorder (maximum 400 mg per day) 400 mg 400 mg 1 x 400 mg 30 2
  100 mg 4 x 100 mg 120 2
Aggressive systemic mastocytosis with eosinophilia (maximum 400 mg per day) 400 mg 400 mg 1 x 400 mg 30 2
  100 mg 4 x 100 mg 120 2

Important: 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 criteria as indicated in the restrictions and be eligible for the Pharmaceutical Benefits Scheme.

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, and
  • the radiology, nuclear medicine, respiratory function or anatomical pathology report(s) providing details of organs involved

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

  • the pathology report confirming the PDGFR gene re-arrangement, and
  • the bone marrow biopsy report that 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, and
  • 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 while on imatinib mesylate treatment

PDGFR B 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 while 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 while on imatinib mesylate treatment

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

Lodging an application

All initial and continuing application requests must be in writing and sent to the Complex drugs address on the contact us page.

For a patient:

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

Further information

For more information contact us on the Complex Drugs Program Enquiry line.

Page last updated: 1 October 2016

This information was printed Sunday 4 December 2016 from humanservices.gov.au/health-professionals/enablers/rare-diseases It may not include all of the relevant information on this topic. Please consider any relevant site notices at humanservices.gov.au/siteinformation when using this material.