Proposals to fund treatments for people with kidney disease, osteoporosis, cancers, hyperparathyroidism and injuries from burns

What we’re proposing

We are seeking feedback on proposals to:

• Fund tolvaptan, a new treatment for people with kidney disease, from 1 December 2022
• Widen funded access to cinacalcet for people with hyperparathyroidism from 1 December 2022
• Remove all funding restrictions from zoledronic acid for people with osteoporosis, Paget’s disease, hypercalcaemia, breast cancer and bone metastases from 1 March 2023
• Fund selenium and copper supplementation for people in hospital with major burns from 1 December 2022

Further details of these proposals, including proposed eligibility criteria, background information and how to provide feedback can be found below.

Consultation closes at 5pm Friday 16 September and feedback can be emailed to consult@pharmac.govt.nz 

Your feedback may be shared

Feedback we receive is subject to the Official Information Act 1982 (OIA). Please be aware that we may need to share your feedback, including your identity, in response to an OIA request. This applies to anyone providing feedback, whether they are providing feedback themselves or for an organisation, in a personal or professional capacity. 

We can only keep feedback confidential as allowed under the OIA and other related laws. If you want any part of your feedback treated as confidential, you need to tell us. Please let us know if you want to keep part of your feedback confidential, and why. Is it commercially sensitive, confidential or proprietary, or personal information? Clearly state this and tell us which parts of your feedback you want to keep confidential for these reasons. We will consider your request under our OIA requirements. 

Tolvaptan for kidney disease

We are proposing to fund tolvaptan (Jinarc), for people with autosomal dominant polycystic kidney disease (ADPKD), subject to eligibility criteria, from 1 December 2022 through a provisional agreement with Otsuka Australia Pharmaceutical Pty Ltd.

Who we think will be interested

• People with ADPKD, their whānau and carers
• Te Whatu Ora hospitals and other organisations who deliver services and support for people, and their whānau who are affected by ADKPD
• Healthcare professionals who treat patients with ADKPD
• People interested in the funded of medicines for people with kidney disease
• Pharmaceutical suppliers
• Pharmacies and wholesalers

What would the effect of this proposal be?

This proposal would mean that people with ADPKD who meet the eligibility criteria described below would have funded access to tolvaptan (Jinarc). It is expected that treatment with tolvaptan would slow kidney cyst growth and kidney function decline; treatment may also reduce kidney pain, delay the progression to end stage renal disease, delay the need for dialysis and transplantation and reduce the risk of early death.

We estimate that approximately 370-400 people would be eligible for treatment with tolvaptan each year.

The clinical advice we have received is that ADPKD is a disease that predominantly affects people aged between 18 and 50 years. We are aware from the clinical advice received that there are often delays in receiving dialysis treatment for Māori with kidney disease, and that Māori with ADPKD are likely to have poorer outcomes than non-Māori with ADPKD. We consider that funding a treatment that delays progression of kidney disease and time to dialysis would therefore be of benefit to Māori and their whānau.

We estimate that approximately 7% of people with ADPKD eligible for treatment with tolvaptan would be Māori. We do not have any data on the percentage of Pacific people who would be eligible.

About autosomal dominant polycystic kidney disease and tolvaptan

ADPKD is a genetic condition characterised by an increase in the number and size of fluid-filled cysts in the kidney. ADPKD leads to end stage renal disease, with patients requiring renal replacement therapy by the age of 58 years on average, resulting in early death (Spithoven Kidney Int. 2014 Dec;86(6):1244-52(external link)). Current treatment is limited to management of chronic kidney disease and symptom relief. There are no funded treatments available which treat the underlying disease. For those people who develop end stage renal disease, dialysis and/or kidney transplantation are the only treatments. 

Tolvaptan works by preventing a hormone called vasopressin from binding to receptors in the kidney. By blocking the effect of vasopressin, tolvaptan slows the development of kidney cysts, slows the rate of kidney function decline, reduces some symptoms of the disease, and increases urine volume. 

Tolvaptan (Jinarc) is approved by Medsafe to slow the progression of cyst development and renal insufficiency of ADPKD in adults with stage 1 to 4 chronic kidney disease when treatment is started and who have rapidly progressing disease. 

Tolvaptan is a tablet taken by mouth twice daily. We have been advised there are dosing considerations and precautions associated with tolvaptan. People taking tolvaptan need regular blood tests and monitoring by their health care professional. Full details of recommended dosing, as well as specific precautions, are available from the Medsafe datasheet(external link) [PDF]. 

We understand the supplier of tolvaptan, Otsuka, would provide a risk management programme to support pharmacists and prescribers to understand, monitor and manage any potential adverse effects. Further details on the risk management programme would be available in the coming months. 

Why we’re proposing this

The Pharmacology and Therapeutics Advisory Committee (PTAC) and the Nephrology Advisory Committee recommended that tolvaptan be funded with a high priority for the people with ADPKD who have an estimated glomerular filtration rate (eGFR) of between 25 and 65 mL/min/1.73 m² at treatment initiation, subject to eligibility criteria. The Committees considered the high health need of people with ADPKD and the evidence of benefit from tolvaptan.

PTAC considered that, in addition to treating people with more advanced disease, there would also be benefit in treating people with stage 1 CKD (eGFR >90 ml/min/1.73m²). While stage 1 CKD would be expected to progress slowly, PTAC considered there may be a high level of benefit in early treatment of people with ADPKD. We have further assessed treatment for patients with stage 1 CKD and are pleased to be including this group as part of this funding proposal.

More information, including links to the Advisory Committee records, can be found in the Application Tracker record for tolvaptan.(external link)

We note that our clinical advisors highlighted the following monitoring and risk requirements as part of the eligibility criteria:

Tolvaptan must be monitored under the supervision of physicians with expertise in managing ADPKD, and a full understanding of the risks of tolvaptan therapy including hepatic toxicity and monitoring requirements (liver function tests are required monthly for the first 18 months and 3-monthly thereafter; concurrent monitoring for symptoms of possible liver injury is recommended)

We are not proposing to include this information as part of the eligibility criteria. We consider this information is better accessed via the New Zealand Formulary(external link).

We also note our clinical advisors have recommended Special Authority renewals every three months, and we welcome your feedback on whether this frequency would be practical.

Details about our proposal

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

A confidential rebate would apply to Jinarc that would reduce the net price to Pharmac. Jinarc would have subsidy and delisting protection until 31 December 2025. 

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

Zoledronic acid – removing funding restrictions

We are proposing to widen access to all funded presentations of zoledronic acid by removing the eligibility criteria restrictions from 1 March 2023. This would apply to the inj 0.05 mg per ml, 100 ml vial/bags and inj 4 mg per 5 ml, vials.

Who we think will be interested

• People with osteoporosis, Paget’s disease, hypercalcaemia, breast cancer and bone metastases requiring treatment with zoledronic acid and their whānau and carers
• Te Whatu Ora hospitals, community-based infusion services and other health providers who deliver infusion services
• Healthcare professionals
• Pharmaceutical suppliers
• Pharmacies and wholesalers 

What would the effect of this proposal be?

All presentations of zoledronic acid would be funded without restrictions (“open-listed”). This would mean that the funded brands of zoledronic acid could be prescribed for any relevant use. 

This change would mean this medicine is funded for many more people, particularly people with osteoporosis. We anticipate that this change would allow 6,000 additional people to access zoledronic acid over the next 5 years. 

We estimate that approximately 3% of people who would use zoledronic acid, should this proposal be approved, would be Māori and 2% would be Pacific people. 

Zoledronic acid is given by intravenous (IV) infusion. We understand that infusion services in hospitals are limited and that this may result in access to zoledronic acid being limited despite removal of the funding restrictions. Community-based infusion services (where they currently exist) generally require a patient service-related co-payment, in addition to any medicine co-payments, which may impact equitable access to treatment. We are proposing a delayed implementation date of 1 March 2023 to allow time for Te Whatu Ora to assess and collaborate with others in the health sector to identify ways to ensure that eligible people would have equitable access to zoledronic acid. This may include increasing hospital or community-based infusion capacity or other measures.

About zoledronic acid

Zoledronic acid is in a group of medicines called bisphosphonates. These medicines reduce bone resorption and the rate of bone turnover. The medicine is administered by IV infusion under the supervision of a healthcare professional. 

Zoledronic acid inj 4 mg per 5 ml is currently funded for use in bone metastases, early breast cancer and symptomatic hypercalcaemia. Zoledronic acid inj 0.05 mg per ml, 100 ml is currently funded for Paget’s disease and osteoporosis. 

You can read the current eligibility criteria on the Pharmaceutical Schedule.(external link)

The current funded brands of zoledronic acid are only Medsafe approved for certain indications. If zoledronic is to be prescribed for unapproved indications, it would need to be prescribed and used in accordance with Section 25 of the Medicines Act 1981. 

You can read more about Section 25 of the Medicines Act 1981 on the Medsafe website.(external link)

Why we’re proposing this

In March 2021 our Endocrinology Advisory Committee recommended removing the funding restrictions for zoledronic acid(external link), with a high priority.

When we consulted on widening access to zoledronic acid in February 2022, we also received feedback requesting we remove all the restrictions.

We have recently secured a price reduction from $60.00 to $22.53 for zoledronic acid 0.05 mg per ml, 100 ml as a result of Pharmac’s 2021/22 Annual Tender process. This has enabled us to widen access to zoledronic acid, as well as reinvest additional savings into funding other medicines.

Details about our proposal

All strengths of zoledronic acid would continue to be listed on the Pharmaceutical Schedule, however all funding restrictions would be removed. 

Cinacalcet for hyperparathyroidism

We are proposing to widen access to cinacalcet, from 1 December 2022, for people with primary, secondary and tertiary hyperparathyroidism.

Who we think will be interested

• People with hyperparathyroidism and their whānau and carers
• Te Whatu Ora and other organisations who deliver services and support for people, and their families and whanau who are affected by hyperparathryoidism
• Healthcare professionals who treat people with hyperparathroidism
• People interested in the funded of medicines for people with kidney and endocrine disease
• Pharmaceutical suppliers
• Pharmacies and wholesalers 

What would the effect of this proposal be?

This proposal would mean that people with hyperparathyroidism who meet the eligibility criteria described below would have funded access to cinacalcet (brand name Cinacalcet Devatis). It is expected that treatment with cinacalcet would reduce serum calcium levels and the risk of cardiovascular events. It may also reduce death from any cause.

We estimate that approximately 725 people would be eligible for treatment in the first year of funding, and this would increase to approximately 945 people per year after five years.

We consider that funding a treatment that may reduce the risk of cardiovascular events and death would be of benefit to Māori, Pacific peoples and their whānau, noting the following points:

• We understand, from the advice we have received, that Māori are less likely to receive curative treatments for hyperparathyroidism such as parathyroidectomy and transplant, due to a higher prevalence of comorbidities.
• We also understand that the incidence and prevalence rates of end-stage chronic kidney disease (the underlying cause of most cases of secondary and tertiary hyperparathyroidism) are higher for Pacific peoples than for any other ethnic group.
• Of the people eligible for treatment we estimate that:
  • approximately 18% of those with primary hyperparathyroidism, would be Māori and 12% would be Pacific people
  • approximately 33% of those with secondary hyperparathyroidism, would be Māori and 29% would be Pacific people
  • approximately 22% of those with tertiary hyperparathyroidism would be Māori, and 17% would be Pacific people.

About cinacalcet

Cinacalcet is in a class of medicines called calcimimetics. Calcimimetics mimic some actions of calcium in the body. Cinacalcet is an oral tablet which decreases the amount of calcium in the blood. Cinacalcet is approved by Medsafe for the following indications:

• as an additional treatment for the signs and symptoms of secondary hyperparathyroidism in people with end stage renal disease who are receiving dialysis.
• for the treatment of hypercalcemia in patients with parathyroid carcinoma.
• to treat the signs and symptoms of primary hyperparathyroidism in adults for whom parathyroidectomy is not a treatment option.

Further information regarding dosing and administration can be found in the Medsafe datasheet [PDF](external link).

Cinacalcet is currently funded for people with parathyroid carcinoma or calciphylaxis who meet specific eligibility criteria [PDF](external link).

Why we’re proposing this

Primary hyperparathyroidism

The Endocrinology Advisory Committee has supported the funding cinacalcet for people with primary hyperparathyroidism, subject to eligibility criteria. The Committee told us that cinacalcet could result in meaningful benefits from a reduction in calcium for people with primary hyperparathyroidism who are unable to have parathyroidectomy surgery.

PTAC recommended that cinacalcet for primary hyperparathyroidism be funded with a medium priority, subject to eligibility criteria. PTAC told us this was because of the high health need of people who are not candidates for surgery, and the lack of available treatments for this group. PTAC considered there were concerns regarding the indirect evidence of benefit, however a reduction in serum calcium from cinacalcet should translate into a reduction in hospitalisations (for treatment of symptomatic hypercalcaemia and its complications) and an improvement in quality of life.

More information, including links to the Advisory Committee records, can be found in the Application Tracker record for cinacalcet for primary hyperparathyroidism(external link).

Secondary and tertiary hyperparathyroidism

The Nephrology Advisory Committee recommended cinacalcet be funded for secondary and tertiary hyperparathyroidism, subject to eligibility criteria, with a high priority. The Committee told us there us a high unmet health need for people with secondary and tertiary hyperparathyroidism.

PTAC recommended funding of cinacalcet for secondary and tertiary hyperparathyroidism subject to eligibility criteria, with a medium priority. PTAC told us there was indirect evidence of reduced mortality/death and considered cinacalcet would offer another medicine for management of calcium for a condition which can be difficult to manage.

More information, including links to the Advisory Committee records, can be found in the Application Tracker record for cinacalcet for secondary(external link) and tertiary hyperparathyroidism(external link)

Details about our proposal

Access to cinacalcet would be widened in Section B and Part II of Section H of the Pharmaceutical Schedule from 1 July 2022 as follows (new criteria shown only):  

Selenium and copper chloride supplementation for people in hospital with major burns

We are proposing to fund selenium and copper chloride supplementation for people in hospital with major burns.  

Who we think will be interested

• People with burns and their whānau/carers
• Te Whatu Ora hospitals and other organisations who deliver services and support for people, and their families and whanau who are injured by burns
• Healthcare professionals who treat patients with burns
• Pharmaceutical Suppliers
• Pharmacies and wholesalers 

What would the effect of this proposal be?

This proposal would mean that hospitals could purchase any brand of selenium oral drops and copper chloride injection and these supplements would be funded for people in hospital with major burns.

It is expected that supplementation with selenium and copper would result in improved wound healing, a decrease in infectious episodes, and is likely to be associated with a reduced risk of infection-related death.

We understand that there are higher reported rates of hospital admission for treatment of burns for Māori, Pacific and socioeconomically deprived populations (Mistry et al. Burns. 2010;36:403-8(external link)). We consider that funding a treatment that may reduce infections and infection-related death would therefore be of benefit to these population groups with burns and their whānau.

We estimate that approximately 195 people would receive supplementation with selenium oral drops and copper chloride injection each year.

As noted below, copper chloride is administered via an intravenous (IV) infusion. However, as patients are already being managed in hospital, we consider any additional infusion requirements are unlikely to represent a significant extra resource requirement. We understand that these agents are already being used by some burns centres across New Zealand via the hospital assessment Named Patient Pharmaceutical Application (NPPA)-rapid process.(external link) Funding selenium oral drops and copper chloride injection would mean hospitals no longer need to complete these forms, for patients who meet the eligibility criteria.

About selenium and copper chloride

Copper and selenium are critical trace elements in several cellular pathways in growth and metabolism, including those involved in wound healing, and are disproportionately lost in patients with major burns due to fluids loss through the wound.

We understand that selenium oral drops and copper chloride injection administered via a daily infusion are used in combination with other funded multivitamins to provide the required supplementation.

Why we’re proposing this

Multivitamin and mineral supplements are currently funded for patients hospitalised with burns. However, funded supplements contain levels of copper and selenium significantly below the levels required for repletion and improved wound healing for severe burn injuries. We understand from the clinical advice we have received that micronutrient deficiencies can result in suboptimal wound healing and an increased risk of infection.

PTAC has recommended that copper chloride and selenium be funded for patients hospitalised with moderate to severe burns with a medium priority, subject to eligibility criteria that require treatment be limited to a three month time period: PTAC told us there is a high health need for patients admitted to hospital with major burns, and that there is strong biological plausibility for the role of trace supplementation in wound healing and reducing the risk of infection following severe burns.

More information, including links to the PTAC records, can be found in the Application Tracker record for selenium and copper chloride(external link).

Details about our proposal

Selenium oral drops and copper chloride injection would be listed in Part II of Section H of the Pharmaceutical Schedule from 1 December 2022, with an example brand for selenium oral drops and no brand for copper chloride, meaning that hospitals could purchase any brand of either formulation. 

Selenium oral drops and copper chloride injection would be listed subject to the following restrictions in Part II of Section H of the Pharmaceutical Schedule: