PBAC supports reforms to CML medicine listings

Blood cancers

By Geir O'Rourke

1 Feb 2024

Prescribing TKIs for the treatment of chronic myeloid leukaemia looks set to become a little more straightforward, after listing reforms were endorsed by the Pharmaceutical Benefits Advisory Committee.

The proposed changes would impact all forms and strengths of ponatinib, asciminib, dasatinib, imatinib and nilotinib, although the recommendations differ somewhat between each drug.

Firstly, the PBAC recommended removing ‘accelerated phase’ from the PBS restriction criteria for all five TKIs to align with the latest World Health Organization classification.

However, it did stress that ‘not blast phase (BP)’ continue to be included in the restriction criteria for asciminib, and that prescribers should be referred to the WHO website for the current definition of BP.

In addition, for ponatinib, it called for the removal of the criteria specifying the patient “must not be eligible for PBS-subsidised treatment with nilotinib because the patient has a blast crisis”.

It also supported changes to the PBS restriction criteria for the imatinib, dasatinib and nilotinib listings for CML:

  • remove specification of line of treatment and phase
  • allow all strengths of nilotinib to be available for dose titration regardless of line of therapy.

The recommendations were made at an intracycle meeting of the committee, held late last year and released last week (link here).

In an outcome statement, the committee also advised removing prescribing and administrative advice from the criteria, as well as the definition of nonresponse, given the select group of clinicians – i.e. specialist haematologists – who prescribed these targeted therapies.

“The PBAC considered these changes would not result in any material changes to the patient population eligible for TKIs listed on the PBS for CML and therefore would not result in increased costs to the PBS,” it added.

hATTR amyloidosis treatment gets the nod

Meanwhile, the committee also came out in support of listing patisiran (Onpattro) for the treatment of patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.

In its outcomes statement, it noted there was a high clinical need for effective treatment of the disease.

There was also evidence of clinical benefits from patisiran in terms of delaying disease progression, reducing neuropathy symptoms, and improving health-related quality of life compared to placebo, based on the primary and secondary outcomes in the APOLLO trial over its 18-month trial period.

Imported by Alnylam Australia, the therapy would be available as concentrate for IV infusion 10 mg in 5 mL.

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