Proposals for funding in the neurology, immunosuppressants, diabetes and antithrombotic therapy areas

Medicines Consultation Closed

We are seeking feedback on proposals for funding in the neurology, immunosuppressant, diabetes and antithrombotic therapy areas.

We are seeking feedback on proposals for funding in the neurology, immunosuppressant, diabetes and antithrombotic therapy areas.

In summary, the proposals would result in the following changes from 1 August 2019:

Further details of these proposals, including how to provide feedback and background information, can be found below. Hyperlinks have been provided above to make it easier for people to find the sections they are most interested in.

Consultation closes at 5pm on Friday, 14 June 2019 and feedback can be emailed to consult@pharmac.govt.nz.

To provide feedback

Send us an email: consult@pharmac.govt.nz by 5pm on 14 June 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.

Rituximab for neuromyelitis optica spectrum disorders (NMOSD)

What would the effect be?

Funded access to rituximab would be widened to include first-line treatment of a severe episode of NMOSD and second-line treatment of NMOSD that has not responded to treatment with oral agents.

Prevention of NMOSD attacks or breakthrough disease could lead to reduced morbidity and mortality for patients. Caregivers may also benefit due to an improvement in patients’ quality of life due to a reduction in attacks and subsequent disability.

We estimate that approximately 20 people per year would be eligible for treatment under the proposed criteria for funding and note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

There would be a cost to DHBs related to the infusion services needed to administer rituximab, however, it is likely that there would be an overall reduction in cost for DHBs associated with hospitalisations due to NMOSD attacks and subsequent disability.

Who we think will be interested

  • People with NMOSD and their whānau
  • Neurologists
  • Hospital pharmacies and DHBs

About rituximab and NMOSD

Rituximab is a monoclonal antibody, given by IV infusion, that destroys B cells by acting against CD20 proteins on their surface. Rituximab is approved by Medsafe for the treatment of non-Hodgkin’s lymphoma, CLL, rheumatoid arthritis and Wegner’s and Microscopic polyangitis. It is not approved for the treatment of NMOSD. Rituximab is currently listed on the HML to treat numerous haematological, rheumatoid and autoimmune conditions, which include both approved and unapproved (off-label) indications.

NMOSD are inflammatory disorders of the central nervous system characterised by severe, immune-mediated demyelination and axonal damage predominantly targeting optic nerves and spinal cord.

People with NMOSD experience acute attacks of bilateral or rapidly sequential optic neuritis (leading to severe visual loss) or transverse myelitis (often causing limb weakness, sensory loss and bladder dysfunction), with a typically relapsing course. Any relapse is potentially severe or fatal and may leave the patient with disability post relapse.

Why we’re proposing this

A funding application for rituximab for the treatment of NMOSD was reviewed by Pharmacology and Therapeutics Advisory Committee (PTAC) in November 2017 and the Neurological Subcommittee in February 2019, which recommended that access to rituximab be widened to include first line treatment of a severe episode of NMOSD and as a second line treatment for NMOSD that is not responsive to treatment with oral agents.

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

Details about our proposal

Access to rituximab (Mabthera) would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 August 2019 to include NMOSD, as follows (new criteria only shown below):

Special Authority for Subsidy

Initial application – (Neuromyelitis Optica Spectrum Disorder) 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. One of the following dose regimens is to be used: 2 doses of 1,000 mg rituximab administered fortnightly, or 4 doses of 375 mg/m2 administered weekly for four weeks; and
  2. Either:

2.1     The patient has experienced a severe episode or attack of NMOSD (rapidly progressing symptoms and clinical investigations supportive of a severe attack of NMOSD); or

2.2     All of the following:

2.2.1    The patient has experienced a breakthrough attack of NMOSD; and

2.2.2    The patient is receiving treatment with mycophenolate; and

2.2.3    The patients is receiving treatment with corticosteroids.


Renewal – (Neuromyelitis Optica Spectrum Disorder) only from a relevant specialist or medical practitioner on the recommendation of a relevant specialist. Approvals valid for 2 years for applications meeting the following criteria:

All of the following:

  1. One of the following dose regimens is to be used: 2 doses of 1,000 mg rituximab administered fortnightly, or 4 doses of 375 mg/m2 administered weekly for four weeks; and
  2. The patients has responded to the most recent course of rituximab; and
  3. The patient has not received rituximab in the previous 6 months.

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

Rituximab for severe refractory myasthenia gravis

What would the effect be?

Funded access to rituximab would be widened to include third-line treatment for patients with severe refractory myasthenia gravis.

Patients who do not remain stable on high-dose steroids and other immunosuppressants, or those that are unable to satisfactorily reduce steroid doses despite treatment with other immunosuppressants (severe refractory patients) would have access to treatment with rituximab.

Patients who achieve disease remission with rituximab may experience a large gain in health-related quality of life. Carer burden may also be reduced for patients who may have previously required help to manage everyday tasks. 

We estimate that approximately 60 people over 5 years would be eligible for treatment under the proposed criteria for funding and note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

There would be a cost to DHBs related to the infusion services needed to administer rituximab; however, it is likely that there would be an overall reduction in cost for DHBs associated with the health services used for management of uncontrolled disease.

Who we think will be interested

  • People with severe refractory myasthenia gravis and their whānau
  • Neurologists
  • Hospital pharmacies and DHBs

About rituximab and myasthenia gravis

Rituximab is a monoclonal antibody, given by IV infusion, that destroys B cells by acting against CD20 proteins on their surface. Rituximab is approved by Medsafe for the treatment of non-Hodgkin’s lymphoma, CLL, rheumatoid arthritis, and Wegner’s and Microscopic polyangitis. It is not approved for the treatment of myasthenia gravis. Rituximab is currently listed on the HML to treat numerous haematological, rheumatoid and autoimmune conditions, which include both approved and unapproved (off-label) indications.

Myasthenia gravis is a progressive autoimmune disease that is characterised by fluctuating skeletal muscle weakness. Weakness commonly affects ocular muscles, but it also involves a variable combination of bulbar, limb and respiratory muscles and fatigue. Patients with severe refractory myasthenia gravis have significant morbidity and mortality.

Why we’re proposing this

A funding application for rituximab for severe refractory myasthenia gravis was reviewed by the Pharmacology and Therapeutics Advisory Committee (PTAC) in August 2016, the Neurological Subcommittee in November 2016 and by PTAC again in February 2017, which recommended that access to rituximab be widened to include third-line treatment for severe refractory myasthenia gravis.

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

Details about our proposal

Access to rituximab (Mabthera) would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 August 2019 to include severe refractory myasthenia gravis, as follows (new criteria only shown below):

Special Authority for Subsidy

Initial application – (Severe Refractory Myasthenia Gravis) only from a neurologist or medical practitioner on the recommendation of a neurologist. Approvals valid for 24 months for applications meeting the following criteria:
Both:

  1. One of the following dose regimens is to be used: 375 mg/m2 of body surface area per week for a total of four weeks, or 500 mg once weekly for four weeks, or two 1,000 mg doses given two weeks apart; and
  2. Either:

2.1.    Treatment with corticosteroids and at least one other immunosuppressant for at least a period of 12 months has been ineffective; or

2.2.    Both:

2.2.1.  Treatment with at least one other immunosuppressant for a period of at least 12 months; and

2.2.2.  Corticosteroids have been trialed for at least 12 months and have been discontinued due to unacceptable side effects.

Renewal – (Severe Refractory Myasthenia Gravis). Only from a neurologist or medical practitioner on the recommendation of a neurologist. Approvals valid for 24 months for applications meeting the following criteria:

All of the following:

  1. One of the following dose regimens is to be used: 375 mg/m2 of body surface area per week for a total of four weeks, or 500 mg once weekly for four weeks, or two 1,000 mg doses given two weeks apart; and
  2. An initial response lasting at least 12 months was demonstrated; and
  3. Either:

3.1.    The patient has relapsed despite treatment with corticosteroids and at least one other immunosuppressant for a period of at least 12 months; or

3.2.    Both

3.2.1.  The patient’s myasthenia gravis has relapsed despite treatment with at least one immunosuppressant for a period of at least 12 months; and

3.2.2.  Corticosteroids have been trialed for at least 12 months and have been discontinued due to unacceptable side effects.

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

Bevacizumab and HPV vaccine for recurrent respiratory papillomatosis (RRP)

What would the effect be?

Funded access to bevacizumab and human papillomavirus (HPV) vaccine would be widened in DHB hospitals to include treatment of recurrent respiratory papillomatosis (RRP).

RRP is caused by infection with the human papillomavirus. When used as an adjunct to surgery, intra-lesional bevacizumab may reduce the growth of papillomas, improve a patient’s voice quality and increase the time interval between surgical procedures, thereby reducing the number of surgical procedures each year. The HPV vaccine may further reduce the size of future papilloma growths for those undergoing debulking surgery who have not yet had the vaccine.

We estimate that approximately 100 people over 5 years would be eligible for treatment under the proposed criteria for funding and note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

We estimate that there would be a reduction in cost for DHBs associated with the management of RRP due to a reduced number of surgical excisions and debridement of papillomas.

Who we think will be interested

  • People with RRP and their whānau
  • ENT surgeons, otolaryngologists, respiratory physicians
  • Hospital pharmacies and DHBs

About bevacizumab and HPV vaccine for RRP

Bevacizumab is a recombinant humanised monoclonal immunoglobulin G1 antibody that binds to and inhibits the biologic activity of human vascular endothelial growth factor by preventing receptor activation. There are a number of published studies showing improvements in patients’ RRP disease and symptoms with intra-lesional administration of bevacizumab; however, bevacizumab is not Medsafe approved for the treatment of RRP. Bevacizumab is currently listed on the HML to treat ophthalmological indications, which are also off label indications.

Gardasil 9 (the currently funded brand of HPV vaccine), is a recombinant vaccine that protects against 9 genotypes of HPV virus. It is indicated for both females and males in varying age groups for prevention of various cancers, precancerous or dysplastic lesions. Evidence suggests that HPV vaccination can increase the time between surgeries and decrease recurrence of RRP.

Why we’re proposing this

Following a number of requests from clinicians and NPPA applications, the Respiratory Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) reviewed the evidence for bevacizumab for the treatment of RRP in August 2017 and recommended that access be widened to this indication, when prescribed by an ENT specialist, with a high priority. In addition, the Respiratory Subcommittee considered that HPV vaccine should be available for people undergoing debulking surgery for RRP who have not yet had the vaccine since the vaccine may reduce the rate of recurrence and size of future papilloma growths.

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

Details about our proposal

Access to bevacizumab and HPV vaccine (Gardasil 9) would be widened in  Part II of Section H of the Pharmaceutical Schedule from 1 August 2019 to include recurrent respiratory papillomatosis, as follows (new criteria in bold):

BEVACIZUMAB

Restricted

Initiation – (Recurrent Respiratory Papillomatosis)

Only from an Otolaryngologist. Approvals valid for 12 months for applications meeting the following criteria:
All of the following:

  1. Maximum of 6 doses; and
  2. The patient has recurrent respiratory papillomatosis; and
  3. The treatment is for intra-lesional administration

Renewal – (Recurrent Respiratory Papillomatosis). Only from an Otolaryngologist. Approvals valid for 12 months for applications meeting the following criteria:

All of the following:

  1. Maximum of 6 doses; and
  2. The treatment is for intra-lesional administration; and
  3. There has been a reduction in surgical treatments or disease regrowth as a result of treatment.

Initiation – (ocular conditions)

Either:

  1. Ocular neovascularisation; or
  2. Exudative ocular angiopathy.

HUMAN PAPILLOMATOSIS (6, 11, 16, 18, 31, 33, 45, 52 AND 58) VACCINE

Restricted

Initiation – (Recurrent Respiratory Papillomatosis)

All of the following:

  1. Either:

1.1.    Maximum of two doses for children aged 14 years and under; or

1.2.    Maximum of three doses for people aged 15 years and over.

  1. The patient has recurrent respiratory papillomatosis; and
  2. The patient has not previously had an HPV vaccine.

Initiation – Children aged 14 years and under

Therapy limited to 2 doses

Children aged 14 years and under.

Initiation – other conditions

Either:

  1. Up to 3 doses for people aged 15 to 26 years inclusive; or
  2. Both:

2.1       People aged 9 to 26 years inclusive; and

2.2       Any of the following:

2.2.1      Up to 3 doses for confirmed HIV infection; or

2.2.2      Up to 3 doses for transplant (including stem cell) patients; or

2.2.3      Up to 4 doses for Post chemotherapy.

Adalimumab for Behçet's disease

What would the effect be?

Funded access to adalimumab would be widened to include treatment of Behçet’s disease, as a second-line biologic treatment after infliximab.

Adalimumab would be funded for patients who are either intolerant to, or have had inadequate benefit from, infliximab. For some patients, this treatment would result in improvements in mucocutaneous, ocular, gastrointestinal and central nervous forms of the disease.

We estimate that approximately 5 to 15- people per year would be eligible for treatment under the proposed criteria for funding and note that some people have been funded in the past via Named Patient Pharmaceutical Assessment (NPPA) applications.

Preventing disease progression for patients would likely result in reduced healthcare costs. There may be a reduction in infusion costs for DHBs if patients move from infliximab to adalimumab.

Who we think will be interested

  • People with Behçet’s disease and their whānau
  • Ophthalmologists, dermatologists, rheumatologists, paediatricians
  • Community and hospital pharmacies
  • DHBs

About adalimumab, ocular inflammation and Behçet’s disease

Adalimumab is a recombinant human tumour necrosis factor (TNF) inhibitor that reduces chronic inflammation and immune response activation. It is administered by subcutaneous injection. Adalimumab is currently funded to treat a number of autoimmune inflammatory conditions, which include both approved and unapproved (off-label) indications.

Behçet’s disease is a rare multi-system, relapsing, chronic inflammatory disorder of the blood vessels. Common symptoms include recurrent oral ulcers, ocular inflammation, genital ulcers, inflammatory arthritis and skin lesions. Serious and life-threatening complications can occur including blindness, inflammation of the brain and nervous symptoms, gastrointestinal disease, blood clots and aneurysms. Behçet’s disease significantly increases morbidity and mortality, with the leading cause of morbidity being eye involvement and subsequent visual loss.

Why we’re proposing this

A funding application for TNF inhibitors for the treatment of Behçet’s disease for patients who are refractory to conventional therapy has been reviewed by the Dermatology Subcommittee in December 2013, the Ophthalmology Subcommittee in October 2014 and by PTAC in August 2012, May 2014 and May 2015. PTAC recommended widening access to adalimumab to patients with Behçet’s disease who are intolerant to infliximab, or who have received inadequate benefit from infliximab, with a medium priority.

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

Details about our proposal

Access to adalimumab (Humira) would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 August 2019 to include Behçet’s disease as follows (new criteria only shown below):

Special Authority for Subsidy

Initial application – (severe Behcet's disease) from any relevant practitioner. Approvals valid for 3 months for applications meeting the following criteria:

All of the following:

  1. The patient has severe Behcet's disease that is significantly impacting the patient’s quality of life (see Notes); and
  2. Either:

2.1      The patient has severe ocular, neurological, gastrointestinal, rheumatological, mucocutaneous and/or vasculitic symptoms and has not responded adequately to treatment with infliximab (see Notes); or

2.2      The patient has severe ocular, neurological, gastrointestinal, rheumatological, mucocutaneous and/or vasculitic symptoms and has experienced intolerable side effects from treatment with infliximab; and

  1. The patient is experiencing significant loss of quality of life; and
  2. Adalimumab to be administered at doses no greater than 40 mg every 14 days.

Notes: Behcet's disease diagnosed according to the International Study Group for Behcet's disease. Lancet 1990;335(8697):1078-80. Quality of life measured using an appropriate quality of life scale such as that published in Gilworth et al, J Rheumatol. 2004;31:931-7

Renewal  – (severe Behcet's disease) from any relevant practitioner. Approvals valid for 6 months for applications meeting the following criteria:

Both:

  1. Patient has had a good clinical response to initial treatment with measurably improved quality of life; and
  2. Adalimumab to be administered at doses no greater than 40 mg every 14 days.

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

Quantity of funded insulin needles

What would the effect be?

Currently a maximum of 100 needles (insulin syringes with attached needles or insulin pen needles) are funded per prescription. This proposal would result in the quantity of insulin syringes and pen needles funded on prescription increasing to allow funding for a further 100 needles per prescription. This means prescribers would be able to prescribe 100 needles plus one repeat; and, patients would have access to 200 funded needles from a single prescription if needed. 

We estimate that around 50,000 people in the community use insulin needles and/or syringes each year.

Who we think will be interested

  • People with diabetes who use insulin and their whānau
  • Community pharmacists
  • Diabetes specialists, diabetes nurse specialists, GPs, nurses
  • DHBs
  • Suppliers of insulin and insulin needles and syringes

About insulin needles

Insulin syringes with attached needles are used to administer insulin from a vial into a person. Insulin pen needles are used to administer insulin from a cartridge via a device (usually an insulin pen) into a person.

Why we’re proposing this

The Diabetes Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) considered the funded needle quantity in September 2013 and March 2019 and recommended increasing the quantity of funded needles to include one repeat per prescription.

Full details of the clinical advice is available here:

PTAC diabetes subcommittee minutes March 2019 [PDF, 328 KB]

PTAC diabetes subcommittee minutes December 2013 [PDF, 30 KB]

Details about our proposal

The maximum quantity of insulin syringes with attached needles and insulin pen needles would be amended in Section B of the Pharmaceutical Schedule from 1 August 2019 as follows (additions in bold and deletions in strikethrough):

INSULIN PEN NEEDLES – Maximum of 100 dev per prescription

  1. a) Maximum of 200 dev per prescription
  2. b) Maximum of 100 dev per dispensing

INSULIN SYRINGES, DISPOSIBLE WITH ATTACHED NEEDLE– Maximum of 100 dev per prescription

a) Maximum of 200 dev per prescription

b) Maximum of 100 dev per dispensing

Intravenous aspirin for acute interventional cardiology and neuro-radiology procedures

What would the effect be?

Intravenous aspirin would be funded in DHB hospitals for use during acute interventional cardiology and neuro-radiology procedures.

Dispersible aspirin tablets are currently used in hospitals for the prevention of blood clots during acute interventional cardiology and neuro-radiology procedures, such as placement of an intracranial stent or coil. Funding an intravenous formulation of aspirin would mean that an immediate antiplatelet effect could be achieved without the delay associated with systemic absorption and placement of an orogastric or nasogastric tube if required.

We estimate that an intravenous formulation of aspirin would lead to a reduction in the time required for some acute interventional cardiology and neuro-radiology procedures. This could also reduce time for an intervention by around 30 minutes per patient per procedure.

We estimate that approximately 200 people per year would be eligible for treatment under the proposed criteria for funding.

DHB hospitals would be able to purchase any brand of intravenous aspirin (lysine acetylsalicylate), in any pack size. PHARMAC would monitor usage and would be amenable to pursuing a national contract should an approved (Medsafe registered) product become available.

Who we think will be interested

  • Cardiologists, interventional neuro-radiologists, neurologists, neurosurgeons and stroke physicians
  • Hospital pharmacies and DHBs.

About aspirin and acute interventions

Aspirin is used for the prevention of blood clots during interventional procedures. Intravenous aspirin may provide an immediate antiplatelet effect in these situations and allow for a faster interventional procedure.

Intravenous aspirin has not been approved by Medsafe, so it would need to be supplied under Section 29 of the Medicines Act 1981. Details regarding the use and supply of unapproved medicines is available from the Medsafe website(external link).

Why we’re proposing this

A funding application for intravenous aspirin for use during interventional neuro-radiology procedures was reviewed by the Cardiology Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) in September 2017, which recommended that intravenous aspirin be funded for acute interventional cardiology and neuro-radiology procedures. PTAC accepted the recommendation of the Subcommittee and recommended that restrictions should apply.

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

Details about our proposal

Lysine acetylcysteine 500 mg injection (intravenous aspirin) would be listed in Part II of Section H of the Pharmaceutical Schedule from 1 August 2019, with no brand name, pack size or price specified. An example brand name would be included (Aspegic), but hospitals would be able to purchase any brand of intravenous aspirin, in any pack size.

Lysine acetylcysteine 500 mg injection (intravenous aspirin) would be listed subject to the following Hospital Restriction:

Restricted
Initiation

Both:

  1. For use when an immediate antiplatelet effect is required prior to an urgent interventional neuro-radiology or interventional cardiology procedure; and
  2. Administration of oral aspirin would delay the procedure.