Proposal for changes to funded haemophilia treatments

We are proposing to make changes to the funding of haemophilia treatments from 1 May 2019, including funding of new extended half-life treatments. These changes would mean that most people with haemophilia would need to change brands, but there would be an increase in the range of funded treatment options available with extended-half-life treatments becoming funded for the first time.

If the proposal is approved, the following changes would occur:

Haemophilia A

• The preferred funded brand of short-half-life recombinant Factor VIII would change from Xyntha to Advate, following a six-month transition period starting on 1 May 2019.
• A new, extended-half-life, recombinant Factor VIII treatment (Adynovate) would be funded from 1 May 2019 for any patients requiring prophylaxis.
• Xyntha would remain available for patients not able to be effectively or safely treated with Advate or Adynovate.
• Kogenate FS would remain available for patients who currently have an approval for Kogenate FS.
• FIEBA NF and NovoSeven RT would continue to be funded for patients with inhibitors to Factor VIII requiring bypassing agents, although FIEBA NF would be the preferred funded brand for longer-term use.

Haemophilia B

• The funded brand of short-half-life recombinant Factor IX would change from BeneFIX to RIXUBIS, following a six-month transition period starting on 1 May 2019.

• A new, extended-half-life recombinant Factor IX treatment (Alprolix) would be funded from 1 May 2019 for any patients requiring prophylaxis.

The arrangements outlined above would apply until at least 30 June 2022.

In addition, PHARMAC’s Haemophilia Treatments Panel would be disestablished and its function of determining named patient eligibility for funded access to the rare clinical circumstances brands (applying PHARMAC specified criteria) would shift to the Haemophilia Treaters Group.

PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 4pm on 18 February 2019 to:

Andrew Park, Senior Therapeutic Group Manager, PHARMAC.  

Email: andrew.park@pharmac.govt.nz.

Who we think will be 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 and the New Zealand Blood Service. 
• Suppliers of haemophilia treatments.

What would the effect be?

For patients

• The preferred brand of short half-life recombinant Factor VIII would be changed from Xyntha (supplied by Pfizer) to Advate (supplied by Shire), meaning that most patients with haemophilia A would need to change brands during a transition period commencing on 1 May 2019.
• Sole-subsidised supply status for short half-life recombinant Factor IX would be awarded to RIXUBIS (supplied by Shire) meaning that all patients with haemophilia B currently using BeneFIX (supplied by Pfizer) would need to change brands during a transition period commencing on 1 May 2019.
• From 1 May 2019, if considered appropriate by the treating clinician, all patients with haemophilia requiring prophylaxis treatment would have funded access to extended half-life Factor VIII and Factor IX treatments (Adynovate and Alprolix, respectively). These treatments are expected to reduce the number of intravenous injections required and may provide health benefits.
• From 1 November 2019, the six-month transition period would end, meaning that all patients who are able to change brands would need to be using Advate, Adynovate, RIXUBIS or Alprolix.
• If a person is clinically unable to transition between brands of Factor VIII from Xyntha to Advate or Adynovate, an application would be required by the treating clinician for the patient to remain on Xyntha. Existing approvals for Kogenate FS would remain valid.
• Preferred brand status would be awarded to FEIBA NF (supplied by Shire) from 1 November 2019 date for patients requiring a bypassing agent for greater than 14 days predicted use.

For treating clinicians

• From 1 May 2019, a six-month transition period would begin, requiring your assistance to transition and support most patients currently using Xyntha to change to Advate or Adynovate and all patients currently using BeneFIX to change to RIXUBIS or Alprolix.
• PHARMAC’s Haematology Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) has noted a best-practice switch to an extended half-life treatment is likely to require initial education, a pharmacokinetic assessment, trough levels after 5-10 treatments, new inhibitor screening and additional follow-ups for the first three months. [PDF, 317 KB]
• From 1 November 2019, the six-month transition period would end, meaning that the following funding arrangements would apply:

• The proposed arrangements above would apply until at least 30 June 2022, with two optional extension periods of one year each until 30 June 2023 and 30 June 2024 respectively, to be exercised at PHARMAC’s sole discretion.
• Prior to 1 November 2019, the Haemophilia Treaters Group would need to have received applications for and considered the clinical circumstances of each patient with haemophilia A on Xyntha who their treating clinician considers inappropriate to transition to Advate or Adynovate. Patients would 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.
• Patients who already have an approval for Kogenate FS would continue to be able to access this brand. Clinicians could also apply to the Haemophilia Treaters Group for funded access to Kogenate FS as a Rare Clinical Circumstances Brand for any new patient who moves to New Zealand where their clinician considers that it would be inappropriate to transition the patient on Kogenate FS to Advate or Adynovate.  In addition to being eligible for funded treatment in New Zealand, patients would need to fulfil one or more of the above eligibility criteria set by PHARMAC in order to access funded Kogenate FS.

Why we’re proposing this

PHARMAC released a Request for Proposals (RFP) for the supply of Recombinant Factor VIII, Recombinant Factor IX and bypassing agents for the treatment of haemophilia in New Zealand on 23 July 2018.

As a result of the RFP and our analysis of the proposals received, PHARMAC has entered into the following provisional agreements:

• Shire New Zealand Ltd for the sole-supply of Adynovate and RIXUBIS, and preferred supply of Advate and FEIBA NF (>14 days predicted bypassing agent use);
• Sanofi/Bioverativ for the sole-supply of Alprolix;
• Pfizer for the supply of Xyntha in rare clinical circumstances;
• Bayer for the supply of Kogenate FS in rare clinical circumstances;
• Novo Nordisk for the ongoing supply of NovoSeven RT.

This combination of agreements would enable broad access to the new extended half-life treatments, whilst still providing substantial savings to the pharmaceutical budget that can be invested in other medicines. Feedback to this consultation will help us to decide if these agreements should be confirmed.

Details about our proposal

New listings

Extended half-life rFVIII

Rurioctocog alfa pegol [recombinant factor VIII] (Adynovate) would be listed in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2019 at the following prices and subsidies (ex-manufacturer, excluding GST):

• A confidential rebate would apply to Adynovate that would reduce its net price to the funder.

• Adynovate would be listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of NHMG claiming only) subject to the ‘Xpharm’ rule and the following eligibility criteria until at least 30 June 2022:

For patients with haemophilia A receiving prophylaxis treatment. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

A funding application for Adynovate was reviewed by our Haematology Subcommittee in October 2017. The Subcommittee recommended that Adynovate be considered as clinically equivalent to rFVIIIFc (which had previously been assessed by PTAC) and that an extended half-life rFVIII be funded for haemophilia A, with a low priority. Detailed information about Adynovate dosing and administration can be found in the Medsafe datasheet(external link).

Extended half-life rFIX

Efmoroctocog [recombinant factor IX] (Alprolix) would be listed in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2019 at the following prices (ex-manufacturer, excluding GST):

• A confidential rebate would apply to Alprolix that would reduce its net price to the Funder.

• Alprolix would be listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of NHMG claiming only) subject to the ‘Xpharm’ rule and the following eligibility criteria until at least 30 June 2022:

For patients with haemophilia B receiving prophylaxis treatment. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

A funding application for Alprolix was reviewed by our Pharmacology and Therapeutics Advisory Committee (PTAC) in August 2016. The Committee recommended that Alprolix be funded only if cost-neutral to the currently listed brands of short-acting rFIX. The Haematology Subcommittee in October 2017 recommended that an extended half-life rFIX be funded for haemophilia B, with a medium-high priority.

Detailed information about Alprolix dosing and administration can be found in the Medsafe datasheet(external link).

Amended listings

Short half-life rFVIII

The listing subsidy and price of octocog alfa [recombinant factor VIII] (Advate) would be amended in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2019 to reflect the following new prices (ex-manufacturer, excluding GST):

• A confidential rebate would apply to Advate that would reduce its net price to the Funder.

• Advate would be listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of claiming) subject to the following eligibility criteria:

from 1 May 2019 until 31 October 2019:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

from 1 November 2019 until 30 June 2022:

For patients with haemophilia. Preferred Brand of short half-life recombinant factor VIII. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

The listing subsidy and price of moroctocog alfa [recombinant factor VIII] (Xyntha) would be amended in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 November 2019 to reflect the following new prices (ex-manufacturer, excluding GST):

Xyntha and Kogenate FS would be listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of claiming) subject to the following eligibility criteria:

from 1 May 2019 until 31 October 2019:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

from 1 November 2019 until 30 June 2022:

For patients with haemophilia. Rare Clinical Circumstances Brand of short half-life recombinant factor VIII. Access to funded treatment is by named patient application to the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group, subject to criteria.

Short half-life rFIX

The listing subsidy and price of nonacog gamma [recombinant factor IX] (RIXUBIS) would be amended in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2019 to reflect the following new prices (ex-manufacturer, excluding GST):

• A confidential rebate would apply to RIXUBIS that would reduce its net price to the Funder.

• The 250 iu strength would be delisted from 1 May 2019 due to low use.

• RIXUBIS would be listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of claiming) subject to the following eligibility criteria:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

All strengths of nonacog alfa [recombinant factor IX] (BeneFIX) would be delisted from the Pharmaceutical Schedule on 1 November 2019.

Bypassing agents

The listing subsidy and price of factor eight inhibitor bypassing fraction (FEIBA NF) would be amended in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 May 2019 to reflect the following new prices (ex-manufacturer, excluding GST):

• A confidential rebate would apply to FEIBA NF that would reduce its net price to the Funder.

• FEIBA NF would remain listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of claiming) subject to the following eligibility criteria:

from 1 May 2019 until 31 October 2019:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

from 1 November 2019 until 30 June 2022:

For patients with haemophilia. Preferred Brand of bypassing agent for >14 days predicted use. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

NovoSeven RT would remain listed in Part II of Section H of the Pharmaceutical Schedule and Section B (for the purposes of claiming) subject to the following eligibility criteria:

from 1 May 2019 until 31 October 2019:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group.

from 1 November 2019 until 30 June 2022:

For patients with haemophilia. Access to funded treatment is managed by the Haemophilia Treaters Group in conjunction with the National Haemophilia Management Group. Rare Clinical Circumstances Brand of bypassing agent for >14 days predicted use. Access to funded treatment for >14 days predicted use is by named patient application to the Haemophilia Treaters Group, subject to access criteria. 

To provide feedback

Send us an email to andrew.park@pharmac.govt.nz by 4pm on Monday, 18 February 2019.

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.