Decision to fund emicizumab for patients with severe haemophilia A and inhibitors of factor VIII

Medicines Decision

What we’re doing

We're pleased to announce a decision to fund emicizumab (Hemlibra), a new treatment for people with severe haemophilia A and inhibitors of factor VIII through an agreement with Roche Products (New Zealand) Limited.

We estimate that 10–15 patients will benefit from this treatment. 

What does this mean for people?

This decision means that people with severe haemophilia A and inhibitors to a coagulation (clotting) factor (factor VIII) will have access to emicizumab (Hemlibra), subject to certain clinical criteria. 

Some people with haemophilia A have a greater tendency to spontaneously bleed and are considered to have more severe haemophilia A. Some of these people can also develop resistance to standard haemophilia treatments. This happens when people develop proteins, called inhibitors, that make standard treatments less effective. When people with haemophilia A develop inhibitors, they cannot form blood clots effectively and bleed more often. This puts them at risk of permanent damage from bleeding into the joints or death from severe internal bleeding. 

Currently, people with haemophilia A who develop inhibitors to factor VIII receive a bypassing agent (FEIBA or NovoSeven) as needed. These bypassing agents are delivered by IV infusion, can be difficult to administer in children and can become ineffective over time. 

Emicizumab is a protein that helps the blood to clot. It is highly effective in preventing bleeding and reduces the frequency of traumatic bleeding episodes in people with haemophilia A who have inhibitors to factor VIII. Emicizumab has a low level of administration-related risk compared to current treatment options and a comparatively low healthcare resource use. 

Emicizumab is Medsafe-approved and is administered subcutaneously at a recommended dose for routine prophylaxis of 3 mg/kg once weekly for the first 4 weeks, followed by a maintenance dose of 1.5 mg/kg weekly. More information about emicizumab dosing and administration is available from the Medsafe datasheet(external link).

Any changes to the original proposal?

This decision was subject to a consultation letter dated 18 September 2020. There have been no major changes as a result of the consultation feedback, other than a small change to the Special Authority criteria to reflect the potential for emicizumab to be administered less frequently than once weekly, if deemed appropriate by the treating clinician.

Who we think will be most interested

  • People with haemophilia A and their whānau or caregivers
  • DHBs and other organisations who deliver services and support for people and families affected by haemophilia A
  • Healthcare professionals who treat patients with haemophilia A
  • Suppliers of haemophilia A products
  • Hospital pharmacists

Detail about this decision

Emicizumab (Hemlibra) will be listed in Section B and in Part II of Section H of the Pharmaceutical Schedule from 1 December 2020 at the following prices and subsidies (ex-manufacturer, excluding GST):

 

Chemical

Formulation

Brand

Pack size

Proposed price and subsidy

Emicizumab

Inj 30 mg in 1 ml vial

Hemlibra

1

$3,570

Emicizumab

Inj 60 mg in 0.4 ml vial

Hemlibra

1

$7,138

Emicizumab

Inj 105 mg in 0.7 ml vial

Hemlibra

1

$12,492

Emicizumab

Inj 150 mg in 1 ml vial

Hemlibra

1

$17,846

A confidential rebate will apply to Hemlibra that will reduce the net price to the Funder. Hemlibra has subsidy and delisting protection until 1 December 2023.

Hemlibra will be listed in Section B and Part II of Section H subject to the ‘Xpharm’ rule and the following eligibility criteria:

Special Authority for Subsidy

Initial application - only from a haematologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has severe congenital haemophilia A and history of bleeding and bypassing agent usage within the last six months; and
  2. Either:
    1. Patient has had greater than or equal to 6 documented and treated spontaneous bleeds within the last 6 months if on an on-demand bypassing agent regimen; or
    2. Patient has had greater than or equal to 2 documented and treated spontaneous bleeds within the last 6 months if on a bypassing agent prophylaxis regimen; and
  3. Patient has a high-titre inhibitor to Factor VIII (greater than or equal to 5 Bethesda units per ml), which has persisted for six months or more; and
  4. There is no immediate plan for major surgery within the next 12 months; and
  5. Either:
    1. Patient has failed immune tolerance induction (ITI) after an initial period of 12 months; or
    2. The Haemophilia Treaters Group considers the patient is not a suitable candidate for ITI; and
  6. Treatment is to be administered at a maximum dose of 3 mg/kg weekly for 4 weeks followed by the equivalent of 1.5 mg/kg weekly. 

Renewal - only from a haematologist. Approvals valid for 6 months for applications meeting the following criteria:

Both:

  1. Patient has had no more than two spontaneous and clinically significant treated bleeds after the end of the loading dose period (i.e. after the first four weeks of treatment until the end of the 24-week treatment period); and
  2. The treatment remains appropriate and the patient is benefiting from treatment.

PHARMAC intends to work with the NHMG, DHBs, haemophilia treatment centres, the Haemophilia Foundation and the supplier (Roche) to ensure adequate support for clinicians, patients and their families during implementation of this change. 

Things to be aware of about this decision:

  • This decision means that the management of these patients by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group (NHMG) remains. However, access to emicizumab treatment will require Special Authority approval.
  • Patients can self-administer emicizumab, reducing their need to go to hospital and improving their quality of life.
  • In order to realise the impact of this treatment, the supplier has organised a direct distribution arrangement to a patients nominated local pharmacy. This would avoid the need to travel to one of the 6 haemophilia treatment centres across New Zealand to receive ongoing treatment, further reducing the burden of haemophilia for these patients.
  • Roche will provide healthcare professionals and patients with information about emicizumab to support its use and implementation. This will include:
    • Videos, booklets, injection logbooks and dosage reminder cards to help patients understand the changes that could be expected when receiving emicizumab
    • Booklet on dosage calculation and administration for healthcare professionals
    • An application (Hemisphere App) for patients to aid in the administration of emicizumab including a reminder function to take their dose.

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 to the feedback received are set out below.  

Theme

Comment

Responders were supportive of funding of emicizumab for patients with haemophilia A and inhibitors of factor VIII. However, not supportive of exclusively funding emicizumab for this severe group of patients only.

PHARMAC staff note the responses in support of funding of emicizumab for this patient group and would welcome a funding application for the wider group of patients.

Responders considered that the proposed Special Authority criteria are restrictive and that access for patients with severe haemophilia A and inhibitors should be less restricted, including:

  • Reduced bleed frequency requirement
  • Access for patients who experience one large bleed

 

PHARMAC has received and assessed an application for this particular patient group with severe haemophilia A and inhibitors of factor VIII.

Members of the Haematology Subcommittee considered that, given the understood efficacy of treatment, there could be many additional patients that could receive emicizumab treatment if these changes were implemented and once a patient commences treatment, treatment is likely to last for a long duration, thus would incur a significant cost.

PHARMAC would welcome a funding application to widen access to emicizumab for less severe patients with or without inhibitors.

PHARMAC staff also note that, should a patient be ineligible for treatment via the proposed Special Authority criteria, it may be appropriate to consider whether an application for funding via PHARMAC’s exceptional circumstances pathway would be appropriate.

Responders proposed that the requirement be changed so that the onus is on the Haemophilia Treaters Group if they consider emicizumab the most appropriate therapy as opposed to ITI, and/or use of bypassing agents.

We consider that objective access criteria, in particular in relation to the frequency of bleeds during access to bypassing agents, for this treatment is the most appropriate way to ensure that those who need emicizumab the most and would receive the most benefit from emcizumab, receive funded access to treatment. In addition, we note that this is the patient group that was assessed in the funding application submitted by the supplier. 

This means that the management of these patients by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group (NHMG) remains. However, access to emicizumab treatment will require Special Authority approval. Please note that the assessment of value of ITI remains a criterion that can be assessed by the Haemophilia Treaters Group.

Responders highlighted that treatment can be administered up to once every 4 weeks and that there is data to support this.

PHARMAC staff acknowledge that treatment can be administered less frequently and in higher doses.

In light of this feedback, we are making this change to the Special Authority criteria to enable this flexibility for patients and prescribers.

Some responders requested access for all patients with severe haemophilia A as emicizumab is considered to be life changing, stopping their bleeds and any ongoing physical crippling damage. Furthermore, it out performs replacement therapy with factor VIII and removes the trauma that can arise from intravenous access.

 

 

PHARMAC staff appreciate the feedback regarding making emicizumab available to all patients with severe haemophilia A.

We understand that emicizumab is highly effective in preventing bleeds. However, at this time, we have not received nor assessed an application for emicizumab for patients with severe haemophilia without inhibitors of factor VIII.

PHARMAC would welcome an application to widen access to emicizumab for patients with severe haemophilia A with or without inhibitors of factor VIII so that this can be assessed through our usual decision-making processes.

A responder did not consider it to be a good idea for patients to self-administer treatment as some people do not like to deal with needles. They also do not have any training in self injecting process.

PHARMAC staff note that while this treatment has the potential to be self-administered, it is not intended that this medicine would have to be self-administered by patients who were not comfortable with the process. PHARMAC staff understand that patients receiving emicizumab would be adequately trained in the process of self-administration.

A responder indicated that the cost for pharmacies to increase the fridge insurance should be highlighted.

The product would be distributed XPharm, so it would not be dispensed by community pharmacy (other than those nominated pharmacies that agree to hold product for their patients to collect). 

Responders highlighted their previous experience with the National Haemophilia Management Group (NHMG). They consider the Haemophilia Treaters Group in conjunction with the NHMG is managing product delivery very efficiently and well for patients with haemophilia. Furthermore, this group has, over time, led to significant savings to the system of treatment and care for people with inherited bleeding disorders in New Zealand

 

PHARMAC staff note and appreciate the supportive feedback regarding the current arrangements with the NHMG for haemophilia products and the provision of a full blood service for these patients.

In addition, we acknowledge the view that the management service has had an important role in limiting the expenditure of haemophilia treatments over the years.

PHARMAC consider that, given the relative cost of emicizumab, it remains appropriate for access to be determined by a Special Authority criteria to manage the cost of this funding decision. Furthermore, this will better inform a future funding decision regarding emicizumab.

Some responders described their experience or the experience of their children with severe haemophilia A and noted that since commencing treatment with emicizumab their quality of life has improved dramatically and that they have not experienced bleeds.

Responders considered that emicizumab is the closest thing to a cure, which will ensure quality of life as a preventative to painful bleeding episodes.

PHARMAC staff note the detailed information regarding the efficacy, suitability and impact of emicizumab on the lives of patients who have received emicizumab prior to this decision and thank the respondents for sharing their personal stories.

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 660 050.