The anti-CD38 mAb daratumumab (Darzalex) has received a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) for PBS listing as a second line treatment in combination with bortezomib and dexamethasone for patients with multiple myeloma.
The drug was recommended on the basis that it should be available only under special arrangements under the Section 100 Efficient Funding of Chemotherapy program.
The recommendation has been a long time coming for clinicians and their patients.
Professor Andrew Spencer, Head of our Malignant Haematology & Stem Cell Transplantation Service at Alfred Health, told the limbic the delay was actually disgraceful.
“This is the fourth submission now. It’s a bit of a joke really. In the past five years there have been six or seven new drugs approved for use in other countries and none of them approved in Australia.”
“It’s an exercise which has really highlighted the widening gap between drug access here and other allegedly first world countries. Countries like Greece and others have had it for a couple of years and yet Australian patients have not had access,” he said.
He said daratumumab was the first new agent with a different mode of action against myeloma potentially reimbursed in Australia for 13 years.
“It’s all about cost. The PBAC model doesn’t allow you to pay for high cost drugs. They have these QALYS etc which are set at financial levels that are 30-40 years out of date.”
“So the pharma companies, no matter how they model the data, they can’t fit into the extremely stringent financial parameters that the PBAC demands.”
Professor Spencer said the IV drug was extremely well tolerated and reimbursement – when it comes – will be very welcome.
“There is also a subcutaneous preparation which has now been approved in a couple of other countries so that will be another major advantage when it can be given subcutaneously.”
The PBAC meeting outcome also noted that daratumumab monotherapy would be provided by Janssen on a compassionate basis to all eligible relapsed and/or refractory MM patients who have no other PBS funded treatment options.
A recently published study has confirmed and extended the previously known effectiveness and tolerability of daratumumab plus bortezomib/dexamethasone in treating relapsed or refractory MM.
In the primary analysis of the CASTOR study, the addition of daratumumab to bortezomib/dexamethasone prolonged progression-free survival (16.7 v 7.1 months) and improved overall survival (85% v 63%) compared to just bortezomib/dexamethasone.
A subsequent sub-group analysis, co-authored by Professor Spencer has found the benefit occurs regardless of cytogenetic risk status.
Median PFS was 16.6 months in patients with standard cytogenetic risk (v 6.6 months with bortezomib/dexamethasone; p < 0.0001) and 12.6 months in patients with high cytogenetic risk (v 6.2 months with bortezomib/dexamethasone; p = 0.0106).