Leukaemia Foundation

Leukaemia, Lymphoma, Myeloma & Related Blood Disorders.

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Continuation of reforms to TGA and PBS to improve access to new medicines for Australians with blood cancers.

The Issue 

The Leukaemia Foundation is concerned that our regulator of new medicines (TGA) and medicines access scheme (PBS) continue to evolve to remain fit for purpose in the age of new genetics-based, targeted medicines for specific disease subtypes.

What we know 

  • Our understanding of cancer is changing rapidly and improving significantly our ability to identify, name and treat cancer by its genetic characteristics. Increasingly cancers are being recognised as subtypes of disease rather than simply the organ in which they occur.
  • The genetic features of subtypes are being used as targets for the development of new and specific medicines – called personalised medicine or precision medicine.
  • Patients welcome precision medicines as it means that they are able to receive a specific treatment developed for their particular blood cancer subtype and avoid heavy duty chemo with its immediate and ongoing detrimental side effects.
  • However, subtype therapies can magnify the challenges of obtaining the quality clinical data required to demonstrate cost effectiveness to the PBAC for rare subtypes of already rare blood cancers.
  • In the last 10 years, there have been around 20 new blood cancer therapies added to the PBS (Medicines Australia Facts Book 2) and about 300 therapies are under development (PhRMA, 2015).
  • The Leukaemia Foundation is concerned that the current PBS system in Australia will not effectively cope with the number of new generation treatments.
  • Blood cancer patients often express concern that drugs made available on the PBS for the common cancers, exclude people with rare forms of cancer, even if their cancer has the same genetic target.
  • The Leukaemia Foundation believes that there should be a score allocated for patient-reported quality of life as part of PBAC assessment.
  • The PBAC’s ability to assess national treatment outcome data would be significantly enhanced with a national clinical quality blood cancer registry. Such registries enable the evaluation of the appropriateness and effectiveness of treatments and other interventions in the healthcare (ACSQHC, 2014).

Election commitments sought 

Where evidence demonstrates clear genetics-based activity of a targeted therapy, initiate target specific indications for therapies in rare diseases to enable equitable access to targeted therapies.

An evidence-based quality of life scoring system based on patient reported experience to form part of the formal assessment process used by PBAC to evaluate the cost effectiveness of medicines.

Next action: Commitment to ensuring the Clinical Trials Action Group recommendations are implemented in full as soon as possible.