The Medicare Services Advisory Committee (MSAC) has recommended funding of emicizumab (Hemlibra) via the National Blood Authority for patients with moderate or severe haemophilia A without factor VIII inhibitors.

However, MSAC said in its advice to the Minister for Health the application for funding “did not justify the expense of emicizumab for all patients, and this supportive advice was subject to pricing negotiations” with manufacturer Roche.

The recommendation follows a previous rejection of the monoclonal antibody by MSAC in 2019, when the Committee concluded that there was not enough cost effectiveness data to support an application for subsidy of the therapy in Haemophilia A patients with inhibitors.

In its latest decision, MSAC noted there was a clinical need for emicizumab, as it reduced the risk of bleeds in severe haemophilia A, eliminated the peaks and troughs of factor VIII concentration currently seen when using factor VIII prophylaxis, and reduced the risk of developing FVIII inhibitors.

It also noted that emicizumab is both less invasive than factor VIII and less frequently injected when given as prophylaxis.

But the committee said the high price requested for  emicizumab was not justified in some subpopulations of patients with moderate HMA and/or patients currently receiving factor VIII on demand.

In its decision summary, MSAC said feedback from haematologists and haemophilia patient groups had been uniformly positive in supporting subsidised access to emicizumab.

It noted that patients with moderate/severe haemophilia A currently face a painful and stressful treatment burden of regular intravenous infusions twice weekly or even every second day to prevent bleeds.

Reduced current treatment compliance leads to worsening outcomes, late night presentations to emergency departments (after falls/injuries during the day) and longer term joint disease from bleeds (with associated surgeries and poorer quality of life).

“The novelty of emicizumab, which allows weekly (or less often, up to monthly) subcutaneous injections, with 50% of patients experiencing no bleeds over a six month period, facilitates freedom, normality and productivity (work and school),” the summary noted.

“Patients who participated in Australian emicizumab clinical trials talked about forgetting haemophilia for the first time, allowing them to get on with life, school and work.”

The committee said it did not believe there was a major risk of potential leakage of emicizumab  usage from the relatively small population of moderate–severe HMA patients to the relatively large population of mild HMA patients, despite factors such as a broader TGA-approved indication and greater convenience of subcutaneous administration, and the desirability of avoiding FVIII inhibitors.

Only 20% of mild HMA patients receive any FVIII treatment and the application was for emicizumab as prophylaxis, whereas mild patients are generally treated with FVIII on-demand.

“The Economic Sub-Committee] considered that haematologists are inherently conservative and are properly self-regulated, which decreases the likelihood of inappropriate emicizumab prescribing.”