Decision to make changes to funded haemophilia treatments

Responders were generally supportive of the proposal to fund extended half-life Factor VIII and Factor IX. Responders noted that whilst some patients will find the change unsettling, it is likely that most (particularly those with severe haemophilia A and B) will want to change to extended half-life Factor VIII and Factor IX, particularly if the right patient education is in place.

This is consistent with our view on likely uptake of extended half-life treatments.

We will focus our implementation activities on supporting people with haemophilia to change to the different treatments, ensuring Treaters have sufficient information on the changes and the new products to be able to support their patients to change treatments. We will work with the Haemophilia Foundation to support their members during the transition period.

Choice of particular products/brands

There are differences in the half-lives of the extended half-life Factor VIII treatments available and this should be considered in the evaluation.

At its meeting on 4 October 2017, the Haematology Subcommittee advised that PEG-rFVIII (Adynovate) could be considered to be clinically equivalent to rFVIIIFc (Eloctate) for the purposes of funding an extended half-life Factor VIII. This was based on its view of the available evidence that both PEG-rFVIII and rFVIIIFc have the same or similar therapeutic efficacy in both adults and children, with the same or similar risks. This advice was considered in the RFP process.

There is limited data on long term safety of regular PEG-rFVIII (Adynovate), particularly in infants and children.

Adynovate is registered by Medsafe for use in children in New Zealand and in many other overseas jurisdictions including Australia, Canada and the United States.

However, there would be no requirement to treat children with Adynovate. The short-acting product, Advate, will be an available funded alternative if treaters or patients consider that Adynovate is not appropriate for them.

We acknowledge that there is a lack of long-term data confirming the safety of this and many other pharmaceuticals used in children. The Haematology Subcommittee noted, in its consideration of Adynovate, that there were no polyethylene glycol (PEG) related adverse reactions in the clinical trials.

We note that in Canada, Adynovate will be the only brand of extended half-life Factor VIII funded. The Canadian Blood Services outlined the widespread and long-term use of PEG containing pharmaceuticals during implementation of that decision, noting that no safety concerns have been raised. We will also address this topic in more detail during our implementation activities.

Other out-of-scope haemophilia treatments 

Emicizumab could radically change how haemophilia A is treated, both for patients with and without inhibitors.

Novel agents such as the as subcutaneously administered prophylaxis treatment emicizumab were specifically excluded from the RFP. This was primarily for the reason that, at the time, we had not yet received clinical advice or performed our assessment on any of these products.

Any products excluded from the scope of the RFP could be listed at any time during the RFP period, but would require funding application assessment and ranking against all of PHARMACs other options for investment.

Since the release of the RFP afunding application for emicizumab in people with haemophilia A and inhibitors to FVIII(external link) has been received and was considered at the recent meeting of the Haematology Subcommittee in January 2019. Minutes of this meeting are not yet available, but will be published on the PHARMAC website once finalised.