Decision to make changes to funded haemophilia treatments

Medicines

Decision

What we’re doing

We’re pleased to announce changes to funded haemophilia treatments that will result in two new extended half-life treatments being funded for the first time in New Zealand. These extended half-life treatments will be funded for all people requiring prophylaxis treatment and are expected to provide prolonged protection from bleeding and reduce the number of intravenous injections required.

Changes are also being made to the funding arrangements of the currently funded short half-life treatments and bypassing agents.

The changes will be implemented over a six-month transition period, commencing on 1 May 2019.

These decisions arose from a Request for Proposals (RFP) process for the supply of haemophilia treatments. In addition to expanding the range of funded haemophilia treatments, this process will release significant funds for PHARMAC to invest in other pharmaceuticals for the benefit of New Zealanders.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 29 January 2019.

There are no changes to the original proposal. We want to thank everyone who provided all their thoughtful feedback to this consultation, which has fed into this decision.

Who we think will be most interested

  • Patients with haemophilia and their family, whānau or caregivers. The Haemophilia Foundation of New Zealand and any others who support people and families affected by haemophilia.

  • Clinicians who treat patients with haemophilia, including the Haemophilia Treaters Group.

  • The National Haemophilia Management Group, Haemophilia Treatment Centres, laboratories, the New Zealand Blood Service and suppliers.

Detail about this decision

New extended half-life treatments

From 1 May 2019, if considered appropriate by the treating clinician, all patients with haemophilia requiring prophylaxis treatment will have funded access to extended half-life Factor VIII and Factor IX treatments (Adynovate and Alprolix, respectively).

Other funding changes

  • From 1 November 2019, Advate will be the Preferred Brand of short half-life recombinant Factor VIII, meaning that most patients with haemophilia A currently using Xyntha will need to change to Advate or Adynovate during a transition period commencing on 1 May 2019.
  • From 1 November 2019, RIXUBIS will have Sole Subsidised Supply status for short half-life recombinant Factor IX, meaning that all patients with haemophilia B currently using BeneFIX will need to change to RIXUBIS or Alprolix during a transition period commencing on 1 May 2019.
  • If a person is clinically unable to transition between brands of Factor VIII from Xyntha to Advate or Adynovate, an application will be required from the treating clinician to the Haemophilia Treaters Group prior to 1 November 2019 for the patient to remain on Xyntha. Patients will need to fulfil one or more of the following eligibility criteria set by PHARMAC for funded access to Xyntha:
    • previously had high titre inhibitor levels
    • are undergoing active or have undergone immune tolerance therapy
    • have a known product allergy
    • have recently commenced therapy (Previously Untreated Patients or PUPs)
    • live in the same residential setting with other people with haemophilia who are unable to switch, as it would be safer to have only one brand kept in the household.
  • Existing approvals for Kogenate FS will remain valid.
  • From 1 November 2019, FEIBA NF will be the Preferred Brand of bypassing agent for patients requiring greater than 14 days predicted use.

Summary

The table below provides a summary of the funding arrangements that will apply from 1 November 2019, following the six-month transition period:

rFVIII Short half-life Preferred Brand
(unrestricted access)
 Advate (Shire)
Rare Clinical Circumstances Brands
(access determined by Haemophilia Treaters Group on a named patient basis)
Xyntha (Pfizer)
Kogenate FS (Bayer)
Extended half-life Sole Subsidised Supply
(unrestricted access for prophylaxis only)
Adynovate (Shire)
rFIX Short half-life Sole Subsidised Supply
(unrestricted access)
RIXUBIS (Shire)
Extended half-life Sole Subsidised Supply
(unrestricted access for prophylaxis only)
Alprolix (Sanofi/Bioverativ)
Bypassing agents (FEIBA and rFVIIa) <14 days predicted use Multiple Supply
(unrestricted access)
FEIBA NF (Shire)
NovoSeven RT (Novo Nordisk)
>14 days predicted use Preferred Brand
(unrestricted access)
FEIBA NF (Shire)
Rare Clinical Circumstances Brand
(access determined by Haemophilia Treaters Group on a named patient basis)
NovoSeven RT (Novo Nordisk)

Please see the consultation letter for additional detail

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. 

A summary of the main themes raised in feedback and our responses is shown below.

Theme

PHARMAC response

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 a funding application for emicizumab in people with haemophilia A and inhibitors to FVIII 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.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.