Decision to decline inactive funding applications

Glucose solution with complex carbohydrate biscuit – hypoglycemia

Not supportive. The comparator treatment has changed since the application has been assessed.

We are not declining this application as, based on the feedback received, we understand there may be a need to update our assessment if the alternative treatment we compared this treatment to has changed.

Ceftolozane with tazobactam – significant infections due to multi-drug resistant aerobic gram-negative organisms

Not supportive. This treatment is important in hospital care for a small number of urgent multi-resistant infection cases.  The Anti-infectives Advisory Committee’s recommendation to decline in 2016 [PDF, 112 KB] may have been due to the limited published evidence at that time

We are not declining this application as, based on the feedback received, we plan to seek further clinical advice on this application from our Anti-infectives Advisory Committee and the supplier.

Albendazole – giardiasis

Giardia is an infectious condition where there is no coherent approach to treatment in New Zealand.

Although there is limited evidence to guide second-line regimens, using albendazole which has a different mechanism of action to first line treatments, is logical when alternative treatments have not worked .

The medicine is not Medsafe approved and is not always effective, however it is well tolerated and is more effective in combination with nitroimidazole.

We are not declining this application as, based on the feedback as we received, we plan to seek further clinical advice on this application from the Anti-infectives Advisory Committee.

Rifampicin/isoniazid/pyrazinamide/ethambutol - fixed dose combinations – tuberculosis

Not supportive. A fixed dose, combination option would ensure patients receive adequate doses of treatment. This medicine would also increase treatment adherence in a highly vulnerable population.

We are not declining this application as we have sought additional clinical advice which indicates:

• updated cost analysis is required.
• patients with tuberculosis (TB) requiring this treatment are under close care of public health services.
• clinical need and suitability advantages are significant.

We are currently working with tuberculosis clinical groups to understand the issues and explore potential solutions.

Miglustat – Niemann Pick Type C (NPC)

Miglustat is currently funded through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA). NPPA is appropriate for this ultra-rare disorder.

We are not declining this application as we have received additional information for consideration. We will take this information to our Rare Disorders Advisory Committee in the future for further consideration. Clinicians can continue to apply for this medicine through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA).

Cannabidiol with tetrahydrocannabinol – multiple sclerosis spasticity, epilepsy, pain and spasticity

A consumer group indicated they would like to provide more information to support this application for cannabidiol with tetrahydrocannabinol.

We are not declining this application, based on the feedback received. We await updated information.  

If no updated information is able to be provided,  we may reconsider declining the application in the future.

Zolmitriptan – migraine - acute migraine with or without aura

Not supportive. New evidence has been published regarding the effectiveness and tolerability , in particular for the use of a nasal spray which has a very fast onset of action. The evidence for comparative efficacy of zolmitriptan should be revisited.

We are not declining this application, based on the feedback received, as we understand there is new evidence regarding effectiveness and tolerability. We will take this information to one of our Advisory Committees in the future for further consideration.

Everolimus – breast cancer, advanced, hormone receptor positive, HER-2 negative

Not supportive. The treatment is recommended in the New Zealand clinical guidelines for advanced breast cancer (2022) [PDF](external link) and is strongly supported by clinical experts involved in the development of the guideline.

Significant inequities exist for Māori and Pacific women with breast cancer that result in poor survival rates. The lack of treatment alternatives for NZ women is significantly contributing to the poor survival rates.

We are not declining this application based on the feedback we received. We have been in contact with the submitter to discuss the additional information we would need for us to reassess the application.  We await updated information. 

If no updated information is able to be provided, we may reconsider declining the application in the future.

Nivolumab with ipilimumab – melanoma, unresectable or metastatic

Not supportive. More evidence has been published since the Pharmacology and Therapeutics Advisory Committee (PTAC) provided advice in 2016. This treatment was included in the Te Aho o Te Ka–u - Cancer Control Agency medicines gap analysis [PDF](external link) A further submission should be sought from the supplier or from the Melanoma Special Interest Group (SIG).

We are not declining this application, based on the feedback received. We have met with the supplier and will take updated information to the Cancer Treatments Advisory Committee (CTAC) at a future meeting.

Pembrolizumab – combination with chemotherapy for those with recurrent or metastatic head and neck squamous cell carcinoma irrespective of Combined Positive Score (ie. an ‘all-comers’ population)

The supplier submitted additional evidence/information to support this application for pembrolizumab.

We will not be declining this application, based on the feedback received. Staff note that a more recent consultation has been issued proposing to fund pembrolizumab for a number of indications (including for recurrent or metastatic head and neck squamous cell carcinoma). Pending the outcome of that consultation and any resulting decisions this application will therefore not be declined at this time.

Bevacizumab – second-line treatment of high-risk advanced ovarian cancer

Not supportive. Recent evidence that relates to the use of bevacizumab in ovarian cancer needs to be considered.

A number of personal accounts from women with advanced ovarian cancer were also provided.

We will not be declining this application, based on the feedback received. The Pharmacology and Therapeutics Advisory Committee (PTAC) considered new evidence submitted in support of this application in May 2024. The record of this meeting will be published soon and then we’ll determine the next steps.  

We are grateful to those who shared their personal experiences with us and have heard what the devastating effects of ovarian cancer are for those people with ovarian cancer and their families/whānau. The information provided helps us understand the health need of people with the illness and the impact of the medicines we're considering for funding.

Atropine 0.01% eye drops – myopia

The supplier, Aspen New Zealand, told us they received Medsafe approval as of 21 December 2023(external link) for atropine sulphate 0.01% eye drops  

We are not declining this application, based on the information received, as the initial reasoning for proposing this to decline was because there was no approved product. We will progress this application for further assessment.

Hepatitis A vaccine – immunisation of people living with HIV

The meeting records relevant to this application are not publicly available so it is not possible to give meaningful feedback to this consultation.

We are not declining this application, based on the feedback received.

We have now published the sections of the February 2015 records of the Immunisation Advisory Committee meeting that relate to the application for Hepatitis A vaccine.  We will contact the submitter to discuss further.

Tacrolimus suppository – rectal inflammation due to inflammatory bowel disease (IBD)

Not supportive. This is a high health need group, with limited alternatives. Extemporaneous compounding with the oral capsules into a rectal preparation is possible.  

We understand there is a high health need for people with rectal inflammation due to IBD.

As there is no approved product available in New Zealand, we have declined the application.

We have considered the feedback about extemporaneous compounding. However, we understand that compounding of tacrolimus oral capsules requires a ventilation hood and although possible in some hospitals, would not be possible in community pharmacies.

If a Medsafe approved proprietary product becomes available, we would be happy to reopen the funding application.

Paracetamol 500mg with codeine 30mg – pain

Supportive of the decline. .

We have declined this application as we have not received any new evidence or information that would support further assessment at this time.

Our expert clinical advisors (the Analgesic Subcommittee) recommended the application be declined as they considered this presentation would not fill an unmet clinical need. We would be happy to reopen the application if information that addresses the Analgesic Subcommittee’s reasons for decline is provided.

Peginterferon alfa-2a – myeloproliferative neoplasms

Clinical need for this medication is met via the current Special Authority.

We have declined this application as we understand the health need for people with myeloproliferative neoplasms is already met through currently funded treatment with peginterferon alfa-2a.

Whole thyroid extract, normal release T3 and extended release T3  – hypothyroidism

Not supportive. Prescribing and use of whole thyroid extract is being conducted internationally ahead of further randomised controlled trials that are planned or currently underway. There is a large clinical need for this treatment and Pharmac should broaden its understanding of the need for this medication.  More evidence and information on prescribing and use of whole thyroid extract could be available in the future.

We have declined this application as we have not received any new evidence or information which would support further assessment at this time. The latest information we received was reviewed by the Endocrinology Advisory Committee in August 2022 which recommended the application be declined [PDF, 235 KB].

We understand this will be disappointing for people with hypothyroidism. We would be happy to re-open the application for assessment should new information be submitted in the future.

Tocilizumab – grade 3 or 4 cytokine release syndrome

Not supportive. Tocilizumab is a standard treatment for cytokine release syndrome (which is seen post CAR-T cell treatment). It is also being used in trials following the use of bi-specific antibodies (which are in a compassionate access programme and which are likely to be funded in the near future).

We have declined this application, but note that this will have no effect on the current access to tocilizumab.

Tocilizumab is already funded for cytokine release syndrome (CRS) with Special Authority criteria. These criteria include those enrolled in the ENABLE trial, experiencing CAR T-Cell related CRS and those enrolled in the AALL 1731 trial.

Oseltamivir – treatment and prophylaxis of influenza during influenza outbreaks in long-term care facilities

 The evidence for efficacy in different contexts (community/hospital) is probably weak, there may be a role in the setting of a new virulent epidemic strain, such as within aged residential care.

We have declined this application. We appreciate the feedback provided and note that oseltamivir is already included as one of the influenza antivirals held in the Ministry of Health’s emergency response supplies.

We note that the Ministry of Health is responsible for establishing and maintaining national reserve supplies. National reserve supplies ensure health services have continued access to specific critical supplies during emergencies that generate unusual demands on stocks or supply chains. This includes pandemic supplies of antiviral medications such as oseltamivir.

More information about the national reserve supplies on the Ministry of Health website(external link).

Tenofovir alafenamide – chronic hepatitis B adults

Have the latest clinical trials been included in our assessment of tenofovir alafenamide?

The patent on tenofovir alafenamide expired in 2022.

We have declined this application as no further published evidence has been provided to suggest there is a health benefit over and above the already funded treatments. We also note that there is currently no tenofovir alafenamide with Medsafe approval for use in New Zealand.

Our Pharmacology and Therapeutics Advisory Committee (PTAC) considered three clinical trials at its August 2018 meeting [PDF, 665 KB]. We will contact the submitter directly to confirm which clinical trials have been considered by PTAC.

We would be happy to reopen the application if updated trial evidence addresses the reasons for PTAC’s recommendation or if there is a generic supplier willing to seek regulatory approval in the future.

Esketamine hydrochloride – major depressive disorder with active suicidal ideation with intent (MDSI)

Esketamine – treatment-resistant depression

Queries about whether esketamine hydrochloride and esketamine could still be funded through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA), if we declined the application. In addition could self-funding still continue?

We have declined the application for use in major depressive disorder with active suicidal ideation with intent as PTAC recommended that the application be declined [PDF, 672 KB] (November 2020) due to lack of clinically relevant benefit and poor generalisability to the New Zealand context .

We have declined the application for use in treatment- resistant depression as PTAC recommended that the application be declined [PDF, 553 KB] (February 2021)

Applications for esketamine can still be assessed through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA).  NPPA is for exceptional circumstances therefore an application would need to demonstrate how a person’s circumstances are  different to the group considered for funding.

There is another funding application for esketamine for treatment resistant depression, post augmentation(external link) under assessment. This was also recommended for decline by PTAC at its February 2024 meeting [PDF, 847 KB].

Self-funding of a treatment is unaffected by a Pharmac decision to decline funding.

Miglustat – Gaucher disease - type 1 (mild to moderate)

An alternative treatment (taliglucerase) to miglustat is already funded for the treatment of Gaucher disease.

If a patient required an alternative to taliglucerase requesting funding via the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA),  would be appropriate.

We have declined this application as an alternative treatment (taliglucerase) is funded.

We can consider individual applications for alternative treatments via the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA), provided the principles of the policy are met, specifically that the person has exceptional circumstances that differ to the group considered.

Miglustat – Gaucher disease

An alternative treatment (taliglucerase) to miglustat is already funded for the treatment of Gaucher disease.

If a patient required an alternative to taliglucerase requesting funding via the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA),  would be appropriate.

We have declined this application as an alternative treatment (taliglucerase) is funded.

We can consider individual applications for alternative treatments via the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA), provided the principles of the policy are met, specifically that the person has exceptional circumstances that differ to the group considered.

Alglucosidase alfa – late onset Pompe disease

Not supportive. There should be a general review of funded treatments for late-onset Pompe disease (LOPD). Childhood onset should be considered differently from adult onset.

Clarification sought that a decline for alglucosidase alfa would not have any impact on a potential future Pharmac assessment for avalglucosidase alfa (brand name Nexviazyme).

We have declined this application as we have not  received any further evidence to support reassessment. We understand there is a high health need for people with Pompe disease and that this decline will be disappointing.

The Rare Disorders Subcommittee considered alglucosidase alfa at its November 2018 [PDF, 713 KB] meeting, and recommended it for decline. At this time the Subcommittee indicated it would welcome an application for alglucosidase alfa targeting treatment to those individuals considered to have juvenile-onset Pompe disease. We have not received a funding application for alglucosidase alpha in this setting.

However, an application for the funding of an alternative treatment, avalglucosidase alfa, for people with either infantile (juvenile) or late onset Pompe disease is under assessment. Declining the application for alglucosidase alfa will not impact our assessment for avalglucosidase alfa. It also wouldn’t prevent us from reconsidering the application for alglucosidase alfa in future if we receive new information.

More information on avaglucosidase alfa on the Application Tracker(external link)

Laronidase – Hurler’s disease (mucopolysaccharidosis 1 or MPS 1)

Not supportive. Laronidase is effective. The information considered by our expert advisors from 2011 is likely outdated. Any future consideration of funding for treatments for MPS should ensure this application is revisited.

We have declined this application as the Pharmacology and Therapeutics Advisory Committee (PTAC) recommended that this application be declined (November 2011) [PDF, 304 KB]. No new evidence to support reassessment has been provided.

We could be happy to reopen this application if we receive further evidence. We would welcome information which addressed the comments PTAC made during its discussion in the November 2011 meeting.

Sapropterin – hyperphenylalaninaemia due to phenylketonuria (PKU) in non-pregnant PKU patients

There is an unmet health need for non-pregnant people with PKU. Funding of sapropterin would be life changing for some people.

New treatments for PKU are being developed, however sapropterin is an option that should also be available. The evidence for sapropterin was last reviewed eight years ago and consideration of new research should occur.

In August 2018 our Pharmacology and Therapeutics Advisory Committee (PTAC) considered an application for sapropterin for patients with PKU at risk of cognitive impairment. However, the recommendation to fund sapropterin with a low priority subject to criteria was made for all patients with PKU, therefore encompassing non-pregnant patients with PKU. That application has now been renamed to reflect the wider patient group considered.

We have declined the application for non-pregnant PKU patients as it has been superseded by another application. This is named sapropterin -  phenylketonuria(external link) and this application is on our Options for Investment list, meaning it is something we would like to fund if we have budget available.

Quinacrine – systemic lupus erythematosus (SLE)

Not supportive. This treatment offers low toxicity and has a role particularly in patients (especially those with skin disease alone) who are intolerant of the alternative treatments.

Patients are able to access funded treatment through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA) in these scenarios.

We have declined this application as there is no Medsafe approved product available in New Zealand and based on the clinical advice we have received we understand there is low evidence to support efficacy. Full details of the clinical advice we received is available in the Application tracker(external link).

We understand this will be disappointing for people with systemic lupus erythematosus who are unable to use other funded alternatives. The decision to decline this application would not stop applications continuing to be considered through the exceptional circumstances pathway, called Named Patient Pharmaceutical Assessment (NPPA).

Lenalidomide – in combination with dexamethasone for the first-line treatment of transplant eligible patients with multiple myeloma

The combination of lenalidomide and dexamethasone with bortezomib would be standard of care internationally for previously untreated transplant eligible patients.

We have declined this application based on the clinical advice we have received that data indicates it is inferior to the current standard of care.

We received advice to support this decision in 2021, from our Cancer Treatments Subcommittee (CaTSoP) [PDF, 878 KB]. It noted the treatment is an inappropriate induction regimen.

We would be happy to reopen this proposal if additional evidence is provided to address CaTSoP’s concerns.

Obinutuzumab – follicular lymphoma, 1st line

Obinutuzumab is funded in the UK, as there is a small benefit, however the priority for funding in New Zealand compared to other non-funded treatments is uncertain.

We have declined this application as the Cancer Treatments Subcommittee of PTAC recommended(external link) (November 2021) that this medicine should only be funded if it was cost neutral ie costs the same, or less, than the funded comparable medicine (November 2021) and that such pricing has not been achievable.

Tobramycin podhaler (TOBI) – cystic fibrosis

It is disappointing that the application is not being progressed, however understand that it has not been possible to achieve a cost-neutral price.

The Pharmacology and Therapeutics Advisory Committee (PTAC) recommended that this medicine should only be funded if it was cost neutral ie cost the same, or less, than the funded comparable medicine (February 2020) [PDF, 588 KB].

We understand that cost neutral pricing for TOBI is not achievable and the application has been declined.

Dexamethasone implant – diabetic macular oedema, first-line

Verteporfin – small subfoveal choroidal neovascularisation due to age-related macular degeneration

Supportive. Access to these treatments would however be welcomed for other indications, including chronic central serous chorioretinopathy.

We have declined these applications as both applications have received decline recommendations. Dexamethasone implant was recommended to be declined by the Ophthalmology Subcommittee in February 2016 [PDF, 99 KB]. And Verteporfin was recommended for decline by PTAC in May 2006. [PDF, 41 KB]

We are seeking clinical advice on a number of funding applications for macular oedema due to retinal vein occlusion, and non-infectious uveitis. We would welcome any supportive information from clinicians that would help us identify the target populations, intended health benefits and comparator treatments.

We would also welcome a funding application for the treatment of chronic central serous chorioretinopathy or any other indications where there is an unmet clinical need.

Ranibizumab – diabetic macular oedema, 2nd line treatment and 3rd line treatment

Supportive.

Since this application was received, we have funded an alternative medicine, aflibercept, as the second line treatment option for diabetic macular oedema.

PTAC recommended that ranibizumab as the third line treatment for diabetic macular oedema be declined (May 2017) [PDF, 534 KB].

The application has therefore been declined.

Ketotifen fumarate – ocular allergy

Advice from an anterior segment specialist should be sought.

The Pharmacology and Therapeutics Advisory Committee (PTAC) recommended that this medicine should only be funded if it was cost neutral [PDF, 205 KB] ie cost the same, or less, than the funded comparable medicine (May 2008).

We understand that this pricing is not achievable and the application has been declined.

We note that our Ophthalmology Advisory Committee includes experts in anterior segment conditions.

We would be happy to reopen this proposal if additional evidence became available.

Influenza vaccine - ring protection for high risk groups [3]

Influenza disproportionately affects  both Māori and Pacific peoples. There are existing health inequities  and a higher risk of influenza in Māori and there is also continued impact on generational health and well-being.

Since receiving this feedback, we have clarified that the application should not reference either Pacific peoples or Māori as these groups have been considered in a separate application for “Influenza vaccine - Māori and Pacific people, 50 to 64 years of age(external link)” that has been ranked on our Options for Investment List, meaning it is something we would like to fund if we have budget available.

This application has therefore been renamed to “Influenza vaccine - ring protection for high risk groups” to reflect the advice received. It is only the ‘ring protection’ of high risk groups (and not the groups themselves) that has been declined for funding.

We did not receive any feedback relating to declining the ‘ring protection’ aspect of the proposal.

Pneumococcal polysaccharide vaccine - immunisation of those aged over-65 years

Not supportive. There are high rates of invasive pneumococcal diseases (IPD), particularly in Māori over the age of 65 and the statistics presented in the proposal were likely to have undercounted the true extent of Māori health inequities.

There are increased rates of IPD compared to when Pharmac’s Immunisation Advisory Committee last considered this vaccine. There are also  higher incidence rates in Pacific peoples and Māori in particular in those people living in socioeconomic deprivation.

We have declined this application as both our Pharmacology and Therapeutics Advisory Committee (PTAC) and the Immunisation Advisory Committee(external link) have recommended declining this application on two occasions. This is due to the low-quality evidence and imprecise results for the efficacy of PPV23 against non-bacteraemic pneumococcal pneumonia. They also considered the lack of evidence of benefit for the Māori and Pacific populations and the lack of data in people over the age of 60 who had risk factors other than hospitalisation.

We acknowledge the feedback and note that we are seeking advice on pneumococcal vaccines at a meeting of the Immunisation Advisory Group scheduled for September 2024. We could reopen this proposal depending on the advice received.

Adjuvanted inactivated quadrivalent influenza vaccine – influenza vaccination for people aged 65 years and over

Not supportive. Adjuvanted influenza vaccines have been shown to be more effective. The risk of hospitalisations, complications, and death resulting from influenza increases in adults aged over 65.

We have declined this as it has been superseded, and an alternative influenza vaccine has been funded. That application was submitted as part of the annual flu vaccination procurement process. 

We would welcome new applications for any future competitive process for influenza vaccines.