Decision to fund supplements for phenylketonuria and other inherited metabolic diseases

What we’re doing

We're pleased to announce our decision to fund a range of supplements for phenylketonuria and other inherited metabolic diseases from 1 February 2024. A number of supplements will be funded for people with a range of inherited metabolic diseases including:

• phenylketonuria 
• maple syrup urine disease
• homocystinuria
• tyrosinaemia
• glutaric aciduria 
• organic acidaemias
• ECHS1-related disease or short chain enoyl coA hydratase deficiency
• urea cycle disorders
• glycogen storage disease    

We expect approximately 120 people in New Zealand will benefit from this proposal in the first year of funding, increasing by approximately 3-4 people each year.

Any changes to the original proposal?

This decision was subject to a consultation document dated 14 November 2023. 

Your feedback told us that ‘inherited metabolic diseases’ is the term used internationally to refer to the genetic conditions that these supplements would be funded for. We have changed our language for this notification and will continue to use this term to describe this group of genetic conditions in the future.

The consultation proposed in error that people with adrenoleukodystrophy receiving care in Te Whatu Ora hospitals would no longer be able to access these supplements. People with adrenoleukodystrophy in hospital will still be able to access these supplements. We are sorry for any concern or confusion this might have caused.

Feedback highlighted some of the supplements listed on the Special Authority form for inherited metabolic disease are no longer available in New Zealand. Feedback requested these were removed. We have removed the supplements that are no longer available from the form.

In the consultation, we asked if people with epilepsy who follow a ketogenic diet use these supplements. We received mixed feedback about this. We have not changed funded access for this group as part of our decision. We will engage with stakeholders to understand whether these supplements should be used by people with epilepsy who follow a ketogenic diet. We would consult before making a decision about any potential changes to funded access for people with epilepsy who follow a ketogenic diet.

Who we think will be most interested

• People with inherited metabolic diseases and their whānau, family and caregivers
• Metabolic specialists and clinicians
• Dietitians, healthcare professionals in primary care and other healthcare settings involved in the care of people with inherited metabolic diseases.
• Suppliers of supplements for people with inherited metabolic diseases.

Details about this decision

From 1 February 2024 the following supplements for phenylketonuria and other inherited metabolic diseases will be listed in Section D and Part II of Section H of the Pharmaceutical Schedule:

Phenylketonuria

Chemical	Formulation	Flavour	Brand	Supplier	Pack size	Price and subsidy
Glycomacropeptide and amino acid contains some phenylalanine	Powder 40 g sachets	Neutral	Camino Pro Bettermilk	Cortex Health	30	$673.92
Glycomacropeptide and amino acid contains some phenylalanine	Liquid, 250 ml carton	Chocolate, neutral	PKU Glytactin RTD 15	Cortex Health	30 OP	$684.45
Glycomacropeptide and amino acid contains some phenylalanine	Liquid, 250 ml carton	Vanilla, Coffee Mocha	PKU Glytactin RTD 15 Lite	Cortex Health	30 OP	$684.45
Glycomacropeptide and amino acid contains some phenylalanine	Powder 16 g sachets	Neutral	PKU Build 10	Cortex Health	30	$449.28
Glycomacropeptide and amino acid contains some phenylalanine	Powder 32 g sachets	Chocolate, Vanilla, Smooth, Raspberry Lemonade	PKU Build 20	Cortex Health	30	$898.56
Glycomacropeptide and amino acid contains some phenylalanine	Powder 20 g sachet	Berry, Orange	PKU Restore Powder	Cortex Health	60	$449.28
Glycomacropeptide and amino acid contains some phenylalanine	Powder 35 g sachets	Red Berry, Vanilla, Chocolate, Banana, Lemon	PKU Sphere 20	Vitaflo	30	$930.00
Amino acid formula without phenylalanine	Powder 400 g	Neutral	PKU Start	Vitaflo	400 g OP	$178.79
Amino acid formula without phenylalanine	Powder 12.5 g sachets	Neutral	PKU Explore 5	Vitaflo	30	$220.88
Amino acid formula without phenylalanine	Powder 25 g sachets	Raspberry, Orange	PKU Explore10	Vitaflo	30	$441.75
Amino acid formula without phenylalanine	Powder 34 g sachets	Neutral, Orange, Lemon, Tropical	PKU Express 20	Vitaflo	30	$883.50

 Tyrosineamia types 1, 2 and 3:

Chemical	Formulation	Flavour	Brand	Supplier	Pack size	Price and subsidy
Amino acid formula without phenylalanine and tyrosine	Powder 12.5 g sachets	Neutral	TYR Explore 5	Vitaflo	30	$349.65
Glycomacropeptide and amino acid contains some tyrosine and phenylalanine	Powder 35 g sachets	Red Berry, Vanilla	TYR Sphere 20	Vitaflo	30	$1,398.60

Maple Syrup Urine Disease, Short chain enoyl coA hydratase deficiency:

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
Amino acid formula without valine, leucine and isoleucine	Powder 12.5 g sachets	MSUD Explore 5	Vitaflo	30	$349.65
Amino acid formula without valine, leucine and isoleucine	Powder 25 g sachets	MSUD Express 15	Vitaflo	30	$1,048.95

Homocystinuria:

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
Amino acid formula without methionine	Powder (unflavoured) 12.5 g sachets	HCU Explore 5	Vitaflo	30	$349.65
Amino acid formula without methionine	Powder 25 g sachets	HCU Express 15	Vitaflo	30	$1,048.95

Glutaric Aciduria type 1

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
Amino acid formula without lysine	Powder 12.5 g sachets	GA Explore 5	Vitaflo	30	$349.65
Amino acid formula without lysine	Powder (25 g sachets)	GA Express 15	Vitaflo	30	$1,048.95

Methylmalonic and Propionic Acidemias

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
Amino acid formula without methionine, threonine and valine	Powder 12.5 g sachets	MMA/PA Explore 5	Vitaflo	30	$349.65
Amino acid formula without methionine, threonine and valine	Powder 25 g sachets	MMA/PA Express 15	Vitaflo	30	$1,048.95

Glycogen Storage Disease

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
High amylopectin corn-starch	Powder 60 g sachets	Glycosade	Vitaflo	30	$241.62

Single dose amino acids:

Chemical	Formulation	Brand	Supplier	Pack size	Price and subsidy
Phenylalanine	Powder 4 g sachets	Phenylalanine50	Vitaflo	30	$141.05
Valine	Powder 4 g sachets	Valine50	Vitaflo	30	$141.05
Isoleucine	Powder 4 g sachets	Isoleucine50	Vitaflo	30	$141.05
Leucine	Powder 4 g sachets	Leucine100	Vitaflo	30	$141.05
Arginine	Powder 4 g sachets	Arginine2000	Vitaflo	30	$211.45
Tyrosine	Powder 4 g sachets	Tyrosine1000	Vitaflo	30	$211.45
Citrulline	Powder 4 g sachets	Citrulline1000	Vitaflo	30	$211.45

The Special Authority criteria for ‘Foods and Supplements for Inherited Metabolic Disease’ from 1 February 2024 in Section D of the Pharmaceutical Schedule will be as follows. 

These Special Authority criteria will apply to currently funded products in the ‘Foods and Supplements for Inborn Errors of Metabolism’ group and the supplements funded from this decision. The name of the group will change to ‘Foods And Supplements For Inherited Metabolic Disease’ from 1 February 2024.

The Hospital Restriction Indication for ‘Metabolic Products’ will be amended from 1 February 2024 in Part II of Section H as follows. These changes will apply to currently funded products in the ‘Metabolic Products’ group and the supplements funded from this decision.

The criteria above will also apply to PKU Build 20 and PKU Sphere 20 which are currently funded in the ‘Other supplements for PKU’ Group in Section D(external link) and Part II Section H(external link) of the Pharmaceutical Schedule.

PKU GMPro Ultra Lemonade, which is supplied by Nutricia, was not in scope of this decision. It will continue to be available in the ‘Other supplements for PKU’ Group in Section D and Part II Section H of the Pharmaceutical Schedule under SA2229 (Community) [PDF](external link) or RS1972 (Hospital) [PDF](external link), if required.

Accessing supply of the funded supplements

Cortex Health

Stock of Cortex Health’s range of supplements will initially be held in Australia. It will be delivered regularly to New Zealand. Stock will be able to be ordered directly from Cortex Health by community pharmacies and Te Whatu Ora hospitals. This includes organisations acting on their behalf.

To cover the costs associated with the delivery of the supplements to New Zealand, Cortex Health will be entitled to charge community pharmacies a distribution fee of 4% of the total value of the supplements being ordered plus GST. This distribution fee aligns with the 4% margin for procurement and stockholding that community pharmacies are currently reimbursed under the current Integrated Community Pharmacy Services Agreement(external link) (ICPSA) for special foods.

Supplements from Cortex Health will be delivered within a maximum of 14 calendar days, where stock is required to be delivered from Australia, or 21 calendar days where stock is required to be delivered from the United States of America.

Cortex Health’s supplements will initially be supplied directly to community pharmacies and Te Whatu Ora hospitals rather than utilising a wholesaler. This arrangement will allow Cortex to closely manage its supplements and their expiry dates, so supplements are not wasted. Demand may be uncertain in the months immediately following the introduction of Cortex Health’s supplements in New Zealand.

Cortex Health’s supplements will be provided to New Zealand with at least six months shelf life remaining.

This arrangement is a temporary exception to normal stock ordering processes via wholesalers. Cortex Health and Pharmac will agree a timeframe for stock to be held in New Zealand within 12 months of Cortex’s supplements being listed on the Pharmaceutical Schedule. It is anticipated that stock would be ordered via wholesalers in the same way as other Special Foods in the future.

Vitaflo

Supply of Vitaflo’s frequently purchased supplements will be held in New Zealand. Supply of infrequently purchased supplements will be held in Australia. These will be supplied to wholesalers in New Zealand within a maximum of 10 business days of an order being received by Vitaflo.

Vitaflo’s range of supplements will be available to order by community pharmacies and Te Whatu Ora hospitals through the wholesaler supply chain via CDC Pharmaceuticals. This includes organisations acting on behalf of Te Whatu Ora hospitals.

Vitaflo’s proposed range of supplements will be supplied to New Zealand with a minimum of 3 months shelf life remaining. Where a supplement with a shelf life of less than 6 months is supplied to New Zealand and expires before it can be used, Vitaflo will provide replacement stock at no extra cost.

These supply arrangements will be reviewed by Pharmac and Vitaflo within 12 months of Vitaflo’s supplements being available in New Zealand. We will amend these arrangements if required.

Our response to what you told us

We’re grateful for the time people took to respond to this consultation. A summary of the main themes raised in feedback, our responses to the feedback received and the changes we have made after listening to you, are available below.

Theme	Pharmac comment
Support for the funding of additional supplements for people with inherited metabolic diseases in New Zealand including glycomacropeptide (GMP) based supplements and single dose amino acids. Responses noted: more variety would allow people to select and use appropriate supplements smaller pack sizes would be more portable and convenient than currently funded options.	We’re pleased to be listing a wide range of supplements to provide more options for people with inherited metabolic diseases.

Stock holding and supply location

Respondents raised concern with the proposal for Cortex’s supplements to be supplied from Australia and the USA. Respondents considered the proposed lead times for delivery may risk an affected individual’s health if supply did not arrive as expected. It was noted this would be a particular concern for infants and young people.

This feedback has been shared with Cortex prior to the release of this notification. We will work closely with Cortex to ensure people can access the supplements they need. The arrangements are temporary to allow time for Cortex to understand likely demand volumes. It is expected that within 12 months of Cortex’s supplements being available in New Zealand timeframes for New Zealand based distribution would be agreed.

Proposed eligibility criteria 

A respondent noted that people with epilepsy who follow a ketogenic diet would not require access to supplements for inherited metabolic diseases as they are not currently used for this purpose. Another respondent supported the funding of supplements for inherited metabolic diseases being retained for people with epilepsy who follow a ketogenic diet.

Following the consultation, we sought further advice from the National Metabolic Service about eligibility for these supplements. The Service told us that the currently funded and proposed supplements for inherited metabolic diseases are not appropriate for use in the ketogenic diet because the carbohydrate content is too high. We are proposing to retain access for people with epilepsy following a ketogenic diet for now. This is based on the mixed feedback that we’ve received. This will ensure people are able to continue receiving funded supplements while we consider this further.

A respondent noted that the current Special Authority form for inherited metabolic diseases (SA1108) includes details of many products that are no longer funded. It was suggested these should be removed.

We appreciate receiving this feedback. We’ve reviewed the supplements included on the Special Authority form and removed supplements that are no longer funded.

Accessibility of proposed supplements 

Respondents supported supplements for inherited metabolic diseases being available on three monthly prescriptions. They considered that current access is inconsistent, with some people receiving monthly supply while others receive three monthly supplies at one time.

We understand there are inconsistencies with the supply of special foods and the volumes that people receive. These are currently intended to be dispensed on a one monthly basis. We will continue to work with Te Whatu Ora to ensure consistent access through New Zealand.

Other feedback

A respondent considered the language ‘Inborn errors of metabolism’ was not people centric and should be updated.	We’ve discussed this feedback with the National Metabolic Service. They told us that ‘inherited metabolic disease’ is the term used internationally. From 1 February 2024 the language in the Pharmaceutical Schedule will be updated accordingly. We will use this language in future communications.
Respondents requested the funding of low protein food options, including the funding of the Camino Pro Complete bar offered by Cortex Health.	We acknowledge the benefits that low protein food options, including those offered by Cortex Health, could offer for people with inherited metabolic diseases.  We have a funding application for low protein foods(external link), including a range of foods offered by Cortex Health. This proposal is currently ranked on our options for investment list. This means it is something we would like to fund when we have available budget.
A respondent requested funding of a paediatric multivitamin and mineral product suitable for a ketogenic diet.	We have a funding application for multivitamin and mineral products(external link) (branded as Phlexyvits and Fruitivits) for people with inherited metabolic diseases and epilepsy. It was considered by the Special Foods Advisory Committee at its meeting in October 2023. Records of this discussion are expected to be available in early 2024.
A respondent highlighted that there were some spelling mistakes in the names of some inherited metabolic diseases.	This feedback is appreciated. These will be corrected in future documents.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll-free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.