Decision to fund treatments for people with breast and blood cancers

Medicines Decision

We’re pleased to announce the funding of treatments for people with breast and blood cancers.

What we’re doing

We're pleased to announce decisions to:

  • Widen access to trastuzumab emtansine (Kadcyla) for people with early breast cancer; and
  • Fund gemtuzumab ozogamicin (Mylotarg) for people with newly diagnosed, CD33-positive acute myeloid leukaemia (AML) through an agreement with Pfizer New Zealand Ltd; and
  • Widen access to azacitidine for treatment related myelodysplastic syndromes (MDS) and AML 

A decision to widen access to obinutuzumab (Gazyva) for some people with indolent non-Hodgkin lymphoma, as consulted, has been delayed. We received extensive and useful consultation feedback on the access criteria and want to take the time to consider this feedback in detail to understand the impact of the requested changes.

Who we think will be most interested

  • People with breast cancer, acute myeloid leukaemia, non-Hodgkin lymphoma and myelodysplastic syndromes and their whānau/carers
  • DHBs and other organisations who deliver services and support for people, and their families and whānau who are affected by breast cancer, acute myeloid leukaemia, non-Hodgkin lymphoma and myelodysplastic syndromes
  • Healthcare professionals who treat patients with breast cancer, acute myeloid leukaemia, non-Hodgkin lymphoma and myelodysplastic syndromes
  • People interested in the funding of medicines for people with cancer
  • Pharmaceutical suppliers
  • Hospital pharmacies and DHBs

Trastuzumab emtansine for early breast cancer

What does this mean for people?

From 1 July 2022, people with early stage HER2-positive breast cancer, with residual disease after initial treatment to shrink the tumour, will have access to trastuzumab emtansine post-surgery. 

In New Zealand, around 80% of people with breast cancer are diagnosed with either stage I or stage II disease, with similar proportions for Māori. This proportion is slightly lower for Pacific people. Around 15% of people with breast cancer are HER2-positive, with similar proportions for Māori. The proportion of people with HER2-positive breast cancer is higher for Pacific peoples (Understanding the Gap: an analysis of the availability of cancer medicines in Aotearoa – Te Aho o Te Kahu(external link)). 

We received feedback which suggested that there may not be as much as many as 110 patients who would receive trastuzumab emtansine in this setting. We have taken this on board and anticipate that around 70 people per year will benefit from treatment. Additionally, around 10% fewer people will need treatment for metastatic breast cancer and there will also be a reduction in the number of people relapsing following treatment post adjuvant therapy. 

Any changes to the original proposal?

This decision was subject to a consultation letter dated 19 May 2022. There have been no changes to this proposal as a result of consultation feedback. All consultation feedback received has been summarised at the end of this notification letter. 

Detail about this decision

Access to trastuzumab emtansine would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 July 2022 as follows (additions in bold):

Special Authority for Subsidy - PCT only – Specialist

Initial application - (early breast cancer) only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. Patient has early breast cancer expressing HER2 IHC3+ or ISH+; and
  2. Documentation of pathological invasive residual disease in the breast and/or axillary lymph nodes following completion of surgery; and
  3. Patient has completed systemic neoadjuvant therapy with trastuzumab and chemotherapy prior to surgery; and
  4. Disease has not progressed during neoadjuvant therapy; and
  5. Patient has left ventricular ejection fraction of 45% or greater; and
  6. Adjuvant treatment with trastuzumab emtansine to be commenced within 12 weeks of surgery; and
  7. Trastuzumab emtansine to be discontinued at disease progression; and
  8. Total adjuvant treatment duration must not exceed 42 weeks (14 cycles). 

Initial application – (metastatic breast cancer) only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist.

Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has metastatic breast cancer expressing HER2 IHC 3+ or ISH+ (including FISH or other current technology); and
  2. Patient has previously received trastuzumab and chemotherapy, separately or in combination and
  3. Either:
    1. The patient has received prior therapy for metastatic disease*; or
    2. The patient developed disease recurrence during, or within six months of completing adjuvant therapy*; and
  4. Patient has a good performance status (ECOG 0-1); and
  5. Either:
    1. Patient does not have symptomatic brain metastases; or
    2. Patient has brain metastases and has received prior local CNS therapy; and
  6. Patient has not received prior funded trastuzumab emtansine treatment; and
  7. Treatment to be discontinued at disease progression

Renewal - (metastatic breast cancer) only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist.

Approvals valid for 6 months for applications meeting the following criteria:

Both:

  1. The cancer has not progressed at any time point during the previous approval period whilst on trastuzumab emtansine; and
  2. Treatment to be discontinued at disease progression. 

Note: * Prior or adjuvant therapy includes anthracycline, other chemotherapy, biological drugs, or endocrine therapy.

Gemtuzumab ozogamicin for newly diagnosed CD33 positive AML

What does this mean for people?

From 1 July 2022, people with newly diagnosed CD33-positive AML (a type of blood cancer) will have access to gemtuzumab ozogamicin (Mylotarg). It is expected that treatment with gemtuzumab ozogamicin will improve survival and  reduce the likelihood of relapse for people with AML. 

We estimate that approximately 70 people per year will benefit from treatment with gemtuzumab ozogamicin. We know that Māori and Pacific peoples have an increased risk of AML compared with non-Māori and non-Pacific peoples, and worse outcomes once diagnosed. So we are pleased to be funding a new treatment that will benefit patients with newly diagnosed AML. 

Any changes to the original proposal?

This decision was subject to a consultation letter dated 19 May 2022. 

Following consultation feedback, we have amended the Special Authority criteria to enable the use of two vials of gemtuzumab ozogamicin per patient to enable more flexible dosing. We consider that this change provides clinical flexibility to dose as required in line with the institutional guidelines. 

Detail about this decision

Gemtuzumab ozogamicin (Mylotarg) would be listed in Section B and in Part II of Section H of the Pharmaceutical Schedule from 1 July 2022 at the following price and subsidy (ex-manufacturer, excluding GST): 

Chemical

Formulation

Brand

Pack size

Proposed price and subsidy

Gemtuzumab ozogamicin

Inj 5 mg vial

Mylotarg

1

$12,973.00

A confidential rebate will apply to Mylotarg that will reduce the net price to the Funder.

Mylotarg will be listed as a PCT only - Specialist pharmaceutical in Section B of the Pharmaceutical Schedule, meaning that only public hospitals will be able to make a subsidy claim.

Mylotarg would be listed in Section B and Part II of Section H of the Pharmaceutical Schedule subject to the following eligibility criteria:

Special Authority for Subsidy

Initial application - only from a haematologist. Approvals valid for 3 months for applications meeting the following criteria:

All of the following:

  1. Patient has not received prior chemotherapy for this condition; and
  2. Patient has de novo CD33-positive acute myeloid leukaemia; and
  3. Patient does not have acute promyelocytic leukaemia; and
  4. Gemtuzumab ozogamicin will be used in combination with standard anthracycline and cytarabine (AraC); and
  5. Patient is being treated with curative intent; and
  6. Patient’s disease risk has been assessed by cytogenetic testing to be good or intermediate; and
  7. Patient must be considered eligible for standard intensive remission induction chemotherapy with daunorubicin and cytarabine (AraC); and
  8. Gemtuzumab ozogamicin to be funded for one course only; and
  9. Either:
    1. Gemtuzumab ozogamicin to be administered as one dose at 3 mg per m2 body surface area; or
    2. Up to 10 mg of gemtuzumab ozogamicin to be administered 

Notes: Acute myeloid leukaemia excludes acute promyelocytic leukaemia and acute myeloid leukaemia that is secondary to another haematological disorder (eg myelodysplasia or myeloproliferative disorder).

Obinutuzumab for the treatment of relapsed or refractory marginal zone and follicular lymphoma

What we’re doing

A decision to widen access to obinutuzumab (Gazyva) for some people with indolent non-Hodgkin lymphoma, as consulted, has been delayed. 

We received extensive and useful consultation feedback on the access criteria and want to take the time to consider this feedback in detail to understand the impact of the requested changes.

Azacitidine for treatment related MDS/AML

What does this mean for people?

From 1 July 2022, more people with some types of blood cancer will be able to access azacitidine. 

We estimate that approximately 10 more people per year with therapy related MDS/AML will be eligible for treatment with azacitidine. We anticipate that this will result in improved survival for these people. 

Any changes to the original proposal?

This decision was subject to a consultation letter(external link) dated 19 May 2022. 

Following consultation feedback, we have removed the proposed addition to the criteria which required azacitidine to be the primary therapy for AML/MDS. This change was made to improve clarity with regard to the intent of the access criteria. 

Detail about this decision

Access to azacitidine would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 July 2022 as follows (deletions in strikethrough):

Special Authority for Subsidy

Initial application only from a haematologist or medical practitioner on the recommendation of a haematologist. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. Any of the following:
    1. The patient has International Prognostic Scoring System (IPSS) intermediate-2 or high-risk myelodysplastic syndrome; or
    2. The patient has chronic myelomonocytic leukaemia (10%-29% marrow blasts without myeloproliferative disorder); or
    3. The patient has acute myeloid leukaemia* with 20-30% blasts and multi-lineage dysplasia, according to the World Health Organization (WHO) classification; and
  2. The patient has performance status (WHO/ECOG) grade 0-2; and
  3. The patient does not have secondary myelodysplastic syndrome resulting from chemical injury or prior treatment with chemotherapy and/or radiation for other diseases; and
    The patient has an estimated life expectancy of at least 3 months 

Renewal only from a haematologist or medical practitioner on the recommendation of a haematologist. Approvals valid for 12 months for applications meeting the following criteria:

Both:

  1. No evidence of disease progression; and
  2. The treatment remains appropriate, and patient is benefitting from treatment.

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, our responses to the feedback received, and changes we have made after listening to you are summarised below.  

Theme

Comment

General

Responders were supportive of these proposals

Pharmac is pleased to be funding proposals that will improve the health outcomes of New Zealanders.

Concerns around capacity and staffing resource to provide new cancer treatments

We appreciate that the there is a substantial impact on clinical services required to initiate and support patients with cancer. 

We consider that these proposals would not significantly impact current resource utilisation.

  • Trastuzumab emtansine will largely replace current treatment with other agents
  • Gemtuzumab ozogamicin would be offered early on, alongside chemotherapy
  • Azacitidine will be used to treat a small group of patients (approximately 10 per year).

Trastuzumab emtansine

Supportive of funding trastuzumab emtansine in this setting.

We are pleased to be widening access to trastuzumab emtansine for people with early breast cancer.

Concerned with limiting patients with HER2-positive breast cancer to one treatment with trastuzumab emtansine per lifetime.

We note that there is a desire to allow for use of trastuzumab emtansine in the metastatic setting, post treatment in the neo-adjuvant setting.

The Cancer Treatments Subcommittee (CaTSoP, which is now the Cancer Treatments Advisory Committee) previously considered that there was no evidence for activity on re-treatment with trastuzumab emtansine to date and noted that in the metastatic setting clinicians would still be able to use trastuzumab. Full details of the clinical advice we received on this topic is available on the application tracker(external link).

We would be happy to receive and assess clinical evidence regarding re-treatment, should this support the use of trastuzumab emtansine in prior responders to treatment, in the metastatic setting.

Sought confirmation that a patient who had previously been treated with trastuzumab emtansine in either the adjuvant (or even metastatic) setting would be permitted to receive it again in the adjuvant setting in the event of a second primary breast cancer.

We note that use of a second course of trastuzumab in the event of a second primary has been considered previously via our waivers process. We consider that a waiver would remain the appropriate funding pathway to assess funding for trastuzumab emtansine should this occur. Information on how to submit a ‘waiver’ is available on the Pharmac website(external link).

Gemtuzumab ozogamicin

Supportive of funding gemtuzumab ozogamicin, noting the poor prognosis of people with AML and the significant benefits of treatment for patients and the health system.

We are pleased to be funding a treatment that will benefit patients with newly diagnosed AML and that it may alleviate health sector resource requirements.

Clinical trial data supports additional doses for a total of five doses and aligns with standard of care dosing in other jurisdictions. The proposed criteria could be inhibitory for NZ patients’ ability to participate in clinical trials for AML.

If unable to fund more than one course, would be supportive of more flexible criteria to allow two doses of up to 5 mg per person.

The proposed Special Authority criteria/Hospital restrictions are based on clinical advice received from our expert advisors. In July 2020 [PDF, 481 KB], CaTSoP considered that the incremental health benefit of five doses was not sufficient to warrant the additional doses. CaTSoP considered that there was likely only small additional benefit in relapse rate and survival from five doses compared with that provided by one dose of gemtuzumab ozogamicin, and that the potential for treatment-related toxicity may also preclude subsequent transplant.

We have made amendments to allow for the use of two vials per patient to enable more flexible dosing of gemtuzumab ozogamicin where it is administered according to institutional guidelines.

Disappointed that midostaurin for newly diagnosed FLT3-positive AML was not approved for funding at the same time as gemtuzumab ozogamicin.

We appreciate that there is a desire for having midostaurin funded for those patients with a FLT-3 mutation (see application tracker(external link)). It remains an option for investment for consideration in the future.

Azacitidine

Supportive due to poor prognosis in this patient group.

We are pleased to be funding a treatment that will have a positive effect on patients with treatment related MDS/AML.

Request for widened access:

  • the Special Authority criteria to be removed completely
  • widen access to patients with a blast count >30% and/or all AML patients who are not fit for intensive induction/conventional therapy

We have assessed a funding application for widened access to azacitidine for all AML patients with a blast count above 30%. This remains an option for investment for consideration in the future (see application tracker(external link)). 

Widening access to open list azacitidine would constitute a substantial investment that has not been assessed. We would welcome a funding application to remove restrictions for azacitidine.

Suggest removal of the criterion “azacitidine is to be used as primary therapy for AML/MDS” could cause confusion and could be interpreted to not be used as a bridge to allogenic stem cell transplantation.

We acknowledge the ambiguity and assure that this was not the intent of the criterion. We have since removed this to reduce ambiguity in intent.

Note that internationally azacitidine is available for older patients in combination with venetoclax. Considered that the proposal to fund azacitidine alone will have minimal impact and requested that venetoclax be included in this proposal.

We are aware of the desire  for venetoclax in combination with azacitidine. Widened access to both venetoclax and azacitidine to enable use in line with the VIALE-A trial is currently an option for investment for consideration in the future (see application tracker(external link)).

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.