The Medical Services Advisory Committee has rejected a funding proposal for haemophilia A and B treatment marstacimab, while backing subsidies for a small group of haemophilia B patients to access the daily injection concizumab.

Marstacimab maker Pfizer had applied for the therapy, a once-weekly prophylatic injection, to be funded for patients aged over 12 who do not have inhibitors.

But the committee advised Minister for Health Mark Butler that there were significant uncertainties with the evidence base, marstacimab’s efficacy compared with other therapies and its cost effectiveness.

“Several prophylaxis treatment options are already available for patients with haemophilia who do not have inhibitors,” the committee noted.

The application had proposed the use of marstacimab, instead of emicizumab, which is already subsidised in Australia, or factor replacement therapy.

However, MSAC noted issues with the data presented for the therapy, including the small number of patients included and differences between study cohorts and the Australian haemophilia population.

“Most Australian patients use extended half-life clotting factor – which may be better at preventing bleeds,” the committee said in its public summary document.

“These issues meant that MSAC could not be certain that marstacimab was as safe, or effective, as emicizumab or factor replacement prophylaxis.”

These concerns about evidence made it difficult to evaluate the cost, with the committee concluding it “could not be certain that marstacimab was good value for money”. Because the treatment was subcutaneous rather than intravenous, the uptake among patients could also be higher than estimated, it said.

In its response, Pfizer said it was considering resubmission in light of the “evidentiary requirements and expectations” of MSAC.

“Patients with severe haemophilia, particularly those with severe [haemophilia B], represent a small population. Access to a subcutaneous treatment option could substantially improve quality of life and reduce inequity of access relative to [haemophilia A],” the company said.

Concizumab backed, but only for HMB with inhibitors 

Meanwhile, Novo Nordisk had argued for funding of its haemophilia B medication concizumab for patients with or without inhibitors, but MSAC could only be convinced for the small group of patients with inhibitors.

“For patients with haemophilia B with inhibitors, MSAC noted the results of the studies did show that patients appeared to have fewer bleeding episodes when they used concizumab compared with on-demand treatment with bypassing agents,” the public summary document said.

“MSAC considered that concizumab reduced treatment burden in the small haemophilia B with inhibitors population who currently do not have access to a regular preventative treatment option and have a high unmet clinical need.”

However, the committee flagged the evidence favouring concizumab for these patients could be due to chance.

For those without inhibitors, the MSAC ruled the evidence did not stack up to say concizumab was more effective than existing Factor IX prophylactic treatment. Studies suggested patients who switched to concizumab from Factor IX therapies experienced more bleeds, MSAC said.

In response, Novo Nordisk welcomed the funding support for patients with inhibitors, but said the committee’s approach to pricing of the drug was not suitable.

Although “MSAC accepted concizumab as a long-term prophylactic therapy, the pricing approach did not adequately reflect this, with an episodic on-demand care framework applied instead,” the company said.

“Novo Nordisk does not consider this appropriate and remains committed to working constructively with the Department to secure timely patient access through a fair and sustainable pricing arrangement.”