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

Medicines

Consultation Closed

PHARMAC are seeking feedback on proposals to widen funded access to medicines in the blood, alimentary and immunosuppressant therapy areas.

To provide feedback

Send us an email: consult@pharmac.govt.nz by Monday 6 January 2020.

We particularly seek your feedback on…

  • the number of patients who would use ruxolitinib if access was widened to include patients with intermediate-1 myelofibrosis.

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). 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.

Ticagrelor for prevention of thrombosis post-neurological stenting

What would the effect be?

Funded access to ticagrelor would be widened from 1 March 2020 to include the prevention of thrombosis in patients who have had a neurological stent and have demonstrated resistance to clopidogrel. The use of ticagrelor for thrombosis prevention following a neurological stent is not Medsafe approved.

This proposal would provide an alternative anti-platelet treatment for patients who, following a neurological stent, are unable to take the currently funded anti-platelet medication (clopidogrel) due to known resistance. We estimate that approximately 40 people each year would be eligible for treatment under the proposed criteria for funding. We note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

Prevention of subsequent neurological events following a neurological stent can reduce the likelihood of future hospitalisations, improving the health status of patients and reducing health care costs.

Who we think will be 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

About ticagrelor and thrombosis prevention post neurological stenting

Ticagrelor is an anti-platelet, or blood thinning, medication that prevents the formation of blood clots that could lead to a thrombotic event  (i.e. cardiovascular death, myocardial infarction or stroke). Ticagrelor is a tablet that is taken twice daily. Ticagrelor is currently funded for use in patients with ST-elevation or non-ST elevation acute coronary syndrome.

A neurological stent is a surgical procedure that is done following a stroke event to open a previously occluded blood-vessel in the brain. Following this surgery patients are required to take anti-platelet medications to prevent thrombosis of the stent and other complications. The majority of patients who have a neurological stent are treated with clopidogrel, another anti-platelet medication. However, approximately 20% of patients may not respond to this medication. The funding of ticagrelor would provide an alternative anti-platelet medication for these patients.

Details about our proposal

Access to ticagrelor (Brilinta) would 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):

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. Patient has demonstrated clopidogrel resistance using the P2Y12 (VerifyNow) assay and requires antiplatelet treatment with ticagrelor.

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).

Budesonide capsules for non-cirrhotic autoimmune hepatitis

What would the effect be?

Funded access to budesonide capsules would be widened from 1 March 2020 to include the treatment of non-cirrhotic autoimmune hepatitis. The use of budesonide for autoimmune hepatitis is not Medsafe approved.

The funding of budesonide capsules for patients with non-cirrhotic autoimmune hepatitis would provide an alternative corticosteroid in a different class to the currently funded corticosteroids, prednisone and prednisolone. For some patients, the use of budesonide may be better tolerated than currently funded corticosteroids due to the different side-effect profile.

We estimate that approximately 240 people each year would be eligible for treatment under the proposed criteria for funding. We note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

Who we think will be 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

About budesonide and non-cirrhotic autoimmune hepatitis

Budesonide is a corticosteroid, in a different class of corticosteroids than currently funded prednisone and prednisolone. Budesonide is a capsule taken once daily. Corticosteroids reduce inflammation and reduce immune system activity. Budesonide capsules are currently funded for use in auto-inflammatory conditions including Crohn’s disease, microscopic colitis and gut graft versus host disease.

Autoimmune hepatitis is a chronic condition where the body’s immune system causes damage to liver cells. Symptoms can include fatigue, abdominal discomfort, jaundice, joint pains and skin conditions. The exact cause of auto-immune hepatis is unclear. If left untreated, it can lead to cirrhosis and liver failure. The funding of budesonide would provide an alternative corticosteroid for approximately 240 patients with non-cirrhotic autoimmune hepatitis.

Details about our proposal

Access to budesonide capsules (Entocort CIR) would 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):

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).

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).

Etanercept for undifferentiated spondyloarthritis

What would the effect be?

Funded access to etanercept would be widened from 1 March 2020 to include the treatment of people with undifferentiated spondyloarthritis. The use of etanercept for undifferentiated spondyloarthritis is not Medsafe approved.

Etanercept would be funded for people with undifferentiated spondyloarthritis with active inflammation who have tried but not responded to an antimetabolite (i.e. methotrexate), an anti-inflammatory (i.e. sulphasalazine) and disease-modifying antirheumatic drug (i.e leflunomide).

For some people, this treatment would result in improvement of joint inflammation, decreasing pain and discomfort and increasing mobility.

People who use etanercept would be able to self-administer their treatment. We estimate that approximately 530 people each year would be eligible for treatment under the proposed criteria for funding.

Who we think will be 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

About etanercept and undifferentiated spondyloarthritis

Etanercept is a recombinant human tumour necrosis factor (TNF) inhibitor (a biologic medicine) that reduces chronic inflammation and immune response activation. It is administered as a weekly subcutaneous injection. The intent of treatment with etanercept in spondyloarthritis is to improve the quality of life of patients by reducing the number and severity of joints experiencing inflammation.

Spondyloarthropathies is the umbrella term for a collective group of chronic disease of the joints including ankylosing spondylitis, reactive arthritis, psoriatic arthritis and joint problems linked to inflammatory bowel disease.  While the majority of patients with undifferentiated spondyloarthropathies eventually differentiate into definite ankylosing spondylitis or psoriatic arthritis, a subset of patients remain undifferentiated in the long term (i.e. do not meet established classification criteria for named spondyloarthropathies). This is estimated to be 10%-30% of patients initially diagnosed with undifferentiated spondyloarthritis. There is no biologic medicine currently funded for patients with undifferentiated spondyloarthritis that remains undifferentiated over time. 

Details about our proposal

Access to etanercept (Enbrel) would 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. All of the following:

2.1 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
2.2 Patient has tried and not responded to at least three months of sulphasalazine at a dose of at least 2 g per day (or maximum tolerated dose); and
2.3 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

3. Any of the following:

3.1 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
3.2 Patient has an elevated erythrocyte sedimentation rate (ESR) greater than 25 mm per hour; or
3.3 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

2. 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

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

Ruxolitinib for lower-risk (intermediate-1) myelofibrosis

What would the effect be?

Funded access to ruxolitinib would be widened from 1 March 2020 to include the treatment of lower risk intermediate-1 myelofibrosis.

Widening access to funded ruxolitinib would provide an additional therapy for patients with lower-risk intermediate-1 myelofibrosis. For some people, this treatment would result in an improvement in disease symptoms and better quality of life. 

We expect that approximately 60 people each year would be eligible for treatment under the proposed criteria.

As part of this consultation, we are specifically seeking feedback on the number of patients who would use ruxolitinib if access was widened as proposed.

Who we think will be interested

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

About ruxolitinib and myelofibrosis

Ruxolitinib is a protein kinase inhibitor of genes that help control the number of blood cells produced. It is Medsafe approved for the treatment of disease-related splenomegaly or symptoms in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

Ruxolitinib is an oral tablet that is administered twice daily. The intent of treatment with ruxolitinib is to control symptoms and improve quality of life.

Ruxolitinib is currently funded for people with high risk and intermediate-2 myelofibrosis.

Myelofibrosis is a rare type of bone marrow cancer where the bone marrow is eventually replaced by fibrosis tissue as a result of excessive cell proliferation. Myelofibrosis symptoms include anaemia, fatigue, abdominal discomfort, pain under the ribs (enlarged spleen), satiety, muscle and bone pain, itching, and night sweats. People with myelofibrosis are stratified by various prognostic scoring systems into low, intermediate-1, intermediate-2 or high-risk myelofibrosis.

This proposal would allow patients with intermediate-1 myelofibrosis to access ruxolitinib in addition to patients with intermediate-2 and high-risk disease who are already able to access funded treatment.

Details about our proposal

Access to ruxolitnib (Jakavi) would 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  

3. 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.