Proposal to fund emicizumab for people with haemophilia A

Medicines Consultation Closed

PHARMAC is seeking feedback on a proposal to fund emicizumab (Hemlibra), a new treatment for people with haemophilia A, from 1 December 2020, through a provisional agreement with Roche Products (New Zealand) Limited.

Further details of this proposal, including how to provide feedback, eligibility criteria and background information, can be found below.

Consultation closes at 4pm Monday 5 October 2020 and feedback can be emailed to consult@pharmac.govt.nz.

What would the effect be?

From 1 December 2020, emicizumab (Hemlibra) would be funded for people with severe haemophilia A and inhibitors to a coagulation (clotting) factor (factor VIII) 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.

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. In turn, bleeding can be more difficult to control and there is a risk of permanent damage from bleeding into the joints or death from severe internal bleeding.

Our clinical advice indicates that treatment with emicizumab significantly reduces the frequency of bleeding episodes for people with haemophilia A who have severe disease and have developed inhibitors to standard treatment with factor VIII. Emicizumab is an injection that people could self-administer, and would reduce their need to go to hospital and improve their quality of life.

We estimate that approximately 10 - 15 patients per year would be eligible for treatment with emicizumab under the proposed criteria.

Currently, access to haemophilia treatments is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group (NHMG). It is intended that this new treatment would be managed in the same way; however, it would include the requirement for a Special Authority approval for access.

Who we think will be interested

  • People with haemophilia A and their family, 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

  • Suppliers of haemophilia A products

  • Hospital pharmacists

About haemophilia A and emicizumab

Haemophilia A is a hereditary, life-long bleeding disorder caused by a deficiency of factor VIII, which results in prolonged spontaneous and injury-related bleeding, joint damage, and in some cases can be life threatening. There are about 500 people in New Zealand with haemophilia A, including approximately 135 people who have severe haemophilia A and a strong tendency to spontaneously bleed, most of whom are managed appropriately with currently funded treatments.

There is a high health burden on people with haemophilia A and their whānau, especially if they are a young child, with significant impacts on everyday life.

Standard haemophilia A treatment consists of intravenous (IV) administration of factor VIII, which helps the blood to clot and stop bleeding. Treatment administration carries a risk of developing inhibitors to factor VIII, making standard treatments less effective and leading to issues with the body’s ability to form blood clots. In turn, bleeding can no longer be easily controlled, impairing people’s quality of life. Persistent factor VIII inhibitors can lead to permanent damage from bleeding into the joints or death from serious 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 and can be difficult to administer in children.

Emicizumab is a protein that helps the blood to clot. It works by replacing the function of factor VIII. 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. Emicizumab 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) [PDF, 253 KB].

Why we’re proposing this

A funding application for emicizumab for the treatment of people with haemophilia A and a documented history of factor VIII inhibitors was considered by the Haematology Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) in January 2019 and by PTAC in May 2019.

Both clinical advisory committees have recommended that emicizumab be funded with a high priority for routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in people with haemophilia A with factor VIII inhibitors, subject to certain clinical criteria.

More information, including links to the PTAC and Subcommittee minutes, can be found in the Application Tracker record for emicizumab(external link)(external link).

Details about our proposal

Emicizumab (Hemlibra) would 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 per ml, 1 ml vial

Hemlibra

1

$3,570

Emicizumab

Inj 60 mg per ml, 0.4 ml vial

Hemlibra

1

$7,138

Emicizumab

Inj 105 mg per ml, 0.7 ml vial

Hemlibra

1

$12,492

Emicizumab

Inj 150 mg per ml, 1 ml vial

Hemlibra

1

$17,846

A confidential rebate would apply to Hemlibra that would reduce the net price to the Funder. Hemlibra would have protection from delisting and subsidy reduction until 1 December 2023.

Hemlibra would 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:

2.1.   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
2.2.   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

  1. 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
  2. There is no immediate plan for major surgery within the next 12 months; and
  3. Either:

5.1.   Patient has failed immune tolerance induction (ITI) after an initial period of 12 months; or
5.2.   The Haemophilia Treaters Group considers the patient is not a suitable candidate for ITI; and

  1. Treatment is to be administered at a maximum dose of 3 mg/kg weekly for 4 weeks followed by 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.

If the proposal is approved, PHARMAC would 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.

To provide feedback

Send us an email:  consult@pharmac.govt.nz by 4pm Monday 5 October 2020.

In addition to seeking general feedback on the proposal, we are also interested in seeking views on the proposal, in relation to: 

  1. if there is a need to risk pool this new medicine in the same manner as currently occurs for haemophilia treatments;
  2. the retention of the current distribution arrangements for haemophilia treatments or whether an alternative mechanism could be appropriate (eg PCT);
  3. the proposed requirement for Special Authority approval.

All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) prior to making a decision on this proposal.

Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.

We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld. PHARMAC will give due consideration to any such request.