Decision for funding in the blood, alimentary and immunosuppressant therapy areas

Medicines

Decision

We're pleased to announce a decision to approve proposals relating to medicines in the blood, alimentary and immunosuppressant therapy areas.

Ticagrelor for prevention of thrombosis post-neurological stenting

Any changes to the original proposal?

As a result of consultation feedback and additional clinical advice, the Special Authority Criteria and restrictions for ticagrelor were amended to allow patients to demonstrate clopidogrel resistance by the occurrence of a new cerebral ischemic event as well as by the VerifyNow assay. This amendment acknowledges that the VerifyNow assay is not available in all DHBs.

The changes to the Special Authority are outlined below

Who we think will be most interested

  • People who have had a neurological stent and their whānau
  • Clinicians who perform neurological stents and are involved in the patient’s subsequent care and management, including:
    • neurosurgeons
    • interventional neurologists
    • neurologists
    • primary care prescribers
    • rehabilitation physicians
  • Community and hospital pharmacies
  • DHBs

Details about this decision

Access to ticagrelor (Brilinta) will be widened in Section B of the Pharmaceutical Schedule from 1 March 2020 to include use post-neurological stenting as follows (new criteria only shown below, amendment following consultation in bold):

Special Authority for Subsidy

Initial application – (thrombosis prevention post neurological stenting) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria.

Both:

  1. Patient has had a neurological stenting procedure* in the last 60 days; and
  2. Either

2.1  Patient has demonstrated clopidogrel resistance using the P2Y12 (VerifyNow) assay and requires antiplatelet treatment with ticagrelor; or
2.2  Clopidogrel resistance has been demonstrated by the occurrence of a new cerebral ischemic event.

Renewal – (thrombosis prevention post neurological stenting) from any relevant practitioner. Approvals valid for 12 months for applications meeting the following criteria.

Both:

  1. Patient is continuing to benefit from treatment; and
  2. Treatment continues to be clinically appropriate.

Note: Indications marked with * are unapproved indications

The same changes to the restrictions for ticagrelor (Brilinta) would apply in Part II of Section H of the Pharmaceutical Schedule (the Hospitals Medicine List; HML).

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. Responses were supportive of the proposal. A summary of the main theme raised in feedback, our response and changes we have made after listening to you are outlined below. 

Theme

PHARMAC Comment

The clopidogrel resistance assay is not available in all DHBs and implementing this assay for small patient numbers is not likely cost-effective. Resistance would be demonstrated by the occurrence of a new cerebral ischemia (functional failure of clopidogrel). Consider including this in the criteria.

The ticagrelor criteria have been amended to incorporate this feedback. PHARMAC considered the proposed change to be a pragmatic solution to address the availability of the VerifyNow assay in some DHBs.

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.

Budesonide capsules for non-cirrhotic autoimmune hepatitis

Any changes to the original proposal?

As a result of consultation feedback, the Special Authority Criteria and restrictions for budesonide capsules were amended to include adolescents with poor linear growth where conventional corticosteroid use may limit further growth.

The changes to the Special Authority are outlined below

Who we think will be most interested

  • People with non-cirrhotic autoimmune hepatitis and their whanau
  • Clinicians who care of patients with autoimmune hepatitis, including:
    • gastroenterologists
    • immunologists
    • general practitioners.
  • Community and hospital pharmacies
  • DHBs

Details about our proposal

Access to budesonide capsules (Entocort CIR) will be widened in Section B of the Pharmaceutical Schedule from 1 March 2020 to include non-cirrhotic autoimmune hepatitis as follows (new criteria only shown below, changes following consultation in bold):

Special Authority for Subsidy

Initial application - (non-cirrhotic autoimmune hepatitis) from any relevant practitioner. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has autoimmune hepatitis*; and
  2. Patient does not have cirrhosis; and
  3. Any of the following:

3.1 Diabetes; or
3.2 Cushingoid habitus; or
3.3 Osteoporosis where there is significant risk of fracture; or
3.4 Severe acne following treatment with conventional corticosteroid therapy; or
3.5 History of severe psychiatric problems associated with corticosteroid treatment; or
3.6 History of major mental illness (such as bipolar affective disorder) where the risk of conventional corticosteroid treatment causing relapse is considered to be high; or
3.7 Relapse during pregnancy (where conventional corticosteroids are considered to be contraindicated).
3.8 Adolescents with poor linear growth (where conventional corticosteroid use may limit further growth) 

Note: Indications marked with * are unapproved indications

Renewal - (non-cirrhotic autoimmune hepatitis) from any relevant practitioner. Approvals valid for 6 months where the treatment remains appropriate and the patient is benefitting from the treatment.

The same changes to the restrictions for budesonide capsules (Entocort CIR) would apply in Part II of Section H of the Pharmaceutical Schedule (the Hospitals Medicine List; HML).

Our response to what you told us

We’re really grateful for the time people took to respond to this consultation. Responses were supportive of the proposal.

A summary of the main theme raised in feedback and changes we have made after listening to you are outlined below. 

Theme

PHARMAC Comment

The proposal addresses an area of unmet clinical need.

Suggested an additional criterion to allow use in adolescents with poor linear growth of whom prednisone may limit their growth

The Special Authority criteria have been amended to specifically include adolescents with poor linear growth.

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.

Etanercept for undifferentiated spondyloarthritis

Any changes to the original proposal?

No changes were made to this proposal as a result of consultation feedback. Some minor formatting changes were made to the criteria.

This decision was subject to a consultation letter dated 9 December 2019

Who we think will be most interested

  • People with undifferentiated spondyloarthritis and their whanau
  • Clinicians who care for patients with undifferentiated spondyloarthritis, including:
    • rheumatologists
    • immunologists
    • general practitioners.
  • Community and hospital pharmacies
  • DHBs

Details about this decision

Access to etanercept (Enbrel) will be widened in Section B of the Pharmaceutical Schedule from 1 March 2020 to include undifferentiated spondyloarthritis as follows (new criteria only shown below):

Special Authority for Subsidy

Initial application — (undifferentiated peripheral spondyloarthritis) only from a rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has undifferentiated peripheral spondyloarthritis* with active peripheral joint arthritis in at least four joints from the following: wrist, elbow, knee, ankle, and either shoulder or hip; and
  2. Patient has tried and not responded to at least three months of oral or parenteral methotrexate at a dose of at least 20 mg weekly or a maximum tolerated dose; and
  3. Patient has tried and not responded to at least three months of sulfasalazine at a dose of at least 2 g per day (or maximum tolerated dose); and
  4. Patient has tried and not responded to at least three months of leflunomide at a dose of up to 20 mg daily (or maximum tolerated dose); and
  5. Any of the following:

5.1 Patient has a C-reactive protein level greater than 15 mg/L measured no more than one month prior to the date of this application; or
5.2 Patient has an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour measured no more than one month prior to the date of this application; or
5.3 ESR and CRP not measured as patient is currently receiving prednisone therapy at a dose of greater than 5 mg per day and has done so for more than three months.

      Note: Indications marked with * are unapproved indications

      Renewal — (undifferentiated peripheral spondyloarthritis) only from a rheumatologist or medical practitioner on the recommendation of a rheumatologist. Approvals valid for 6 months for applications meeting the following criteria:

      All of the following:

      1. Either:

      1.1 Applicant is a rheumatologist; or
      1.2 Applicant is a Practitioner and confirms that a rheumatologist has provided a letter, email or fax recommending that the patient continues with etanercept treatment; and

      1. Either:

      2.1 Following 3 to 4 months’ initial treatment, the patient has at least a 50% decrease in active joint count from baseline and a clinically significant response to treatment in the opinion of the physician; or
      2.2 The patient demonstrates at least a continuing 30% improvement in active joint count from baseline and a clinically significant response to prior etanercept treatment in the opinion of the treating physician; and

      1. Etanercept to be administered at doses no greater than 50 mg dose every 7 days.

      The same changes to the restrictions for etanercept (Enbrel) will apply in Part II of Section H of the Pharmaceutical Schedule (the Hospitals Medicine List; HML).

      Our response to what you told us

      We’re really grateful for the time people took to respond to this consultation. Responses were supportive of the proposal. A summary of the main theme raised in feedback, our response and changes we have made after listening to you are outlined below. 

      Theme

      PHARMAC Comment

      Request to include patients with associated uveitis for whom they believe 1st line adalimumab would be a better treatment option than etanercept.

      Adalimumab and infliximab are currently funded for patients with severe or chronic uveitis. We consider patients that have active uveitis that meet the funding criteria for this indication would already be able to access funded adalimumab or infliximab, addressing this clinical need.

      Request for adalimumab and infliximab to be funded for patients who cannot tolerate or do not respond to etanercept.

      Widening access to adalimumab for undifferentiated spondyloarthritis remains an option for consideration in the future.

      At this time PHARMAC do not have positive clinical advice regarding infliximab for this patient population. We would seek further clinical advice following the emergence of evidence to support infliximab’s efficacy and safety in this patient population.

      There would be an increase in clinical services time as a result of initiating and supporting patients on etanercept subcutaneous injections.

      PHARMAC acknowledge the impact on clinical services to initiate and support patients with subcutaneous treatments. However, we also acknowledge that this patient group will already be engaging regularly with specialist health services and this is unlikely to significantly change with the funding of etanercept.

      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.

      Ruxolitinib  - Myelofibrosis, lower-risk (Intermediate-1)

      Any changes to the original proposal?

      No changes were made to this proposal as a result of consultation feedback.

      This decision was subject to a consultation letter dated 9 December 2019

      Who we think will be most interested

      • People with myelofibrosis and their whanau
      • Clinicians who care of patients with myelofibrosis, including:
        • haematologists
        • general practitioners.
      • Community and hospital pharmacies
      • DHBs

      Details about this decision

      Access to ruxolitnib (Jakavi) will be widened in Section B of the Pharmaceutical Schedule from 1 March 2020 to include lower-risk intermediate-1 myelofibrosis as follows (additions in shown in bold, deletions shown in strikethrough):

      Special Authority for Subsidy

      Initial application only from a haematologist. Approvals valid for 12 months for applications meeting the following criteria:

      All of the following:

      1. The patient has primary myelofibrosis or post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis; and
      2. Either

      2.1 A classification of risk of intermediate-2 or high-risk myelofibrosis according to either the International Prognostic Scoring System (IPSS), Dynamic International Prognostic Scoring System (DIPSS), or the Age-Adjusted DIPSS; and or
      2.2 Both

      2.2.1 A classification of risk of intermediate-1 myelofibrosis according to either the International Prognostic Scoring System (IPSS), Dynamic International Prognostic Scoring System (DIPSS), or the Age-Adjusted DIPSS; and
      2.2.2 Patient has severe disease-related symptoms that are resistant, refractory or intolerant to available therapy; and  

      1. A maximum dose of 20 mg twice daily is to be given.

      Renewal 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:

      Both:

      1. The treatment remains appropriate and the patient is benefiting from treatment; and
      2. A maximum dose of 20 mg twice daily is to be given.

      The same changes to the restrictions for ruxolitnib (Jakavi) will apply in Part II of Section H of the Pharmaceutical Schedule (the Hospitals Medicine List; HML).

      Our response to what you told us

      We’re really grateful for the time people took to respond to this consultation. Responses were supportive of the proposal. A summary of the main theme raised in feedback and our response  are outlined below. 

      Theme

      PHARMAC Comment

      Supportive of funding proposal noting unmet health need and that this would align the indications with Australia.

      Other haematology indications are of a greater priority – specifically Midostaurin for AML patients and Ibrutinib for CLL patients.

      A clinician application for midostaurin for AML was received in August 2019. The next step for this application is for PHARMAC to seek clinical advice. This will occur at the next available opportunity.

      More information can be found in the Application Tracker records for midostaurin(external link)

      A proposal to fund ibrutinib for CLL is currently ranked and remains a future option for investment.  We have not yet specifically sought advice on groups of patients with CLL that would benefit most from ibrutinib now that venetoclax is funded. We will likely seek advice from CaTSoP on this at a future committee meeting.

      More information, including links to the PTAC and Subcommittee minutes, can be found in the Application Tracker records for ibrutinib(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 66 00 50.