Decision to fund supplements for phenylketonuria and other inherited metabolic diseases

What we’re doing

We're funding a range of supplements for phenylketonuria and other inherited metabolic diseases from 1 August 2024. Several supplements will be funded for use in the community and in Health New Zealand | Te Whatu Ora Hospitals for people with a range of inherited metabolic diseases including:

• phenylketonuria (PKU)
• maple syrup urine disease
• homocystinuria
• tyrosinemia
• glutaric aciduria
• organic acidaemias
• ECHS1-related disease or short chain enoyl coA hydratase deficiency
• urea cycle disorders
• fatty acid oxidation disorders

We expect approximately 120 people in New Zealand will use the supplements in the first year of funding. After the first year, we estimate 3-4 people will start on these supplements each year.

The consultation proposed changes to the eligibility criteria for supplements for inherited metabolic diseases. This was to reflect that the currently funded range of supplements are not suitable for people with epilepsy following a ketogenic diet because of their high carbohydrate content. This change in eligibility criteria has now been confirmed.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 20 June 2024. We haven’t made any changes to our proposal. We’re grateful for the feedback we received.

Who we think will be most interested

• People with inherited metabolic diseases and their whānau and caregivers
• Metabolic specialists, dietitians, and other health care professionals involved in the care of people with inherited metabolic diseases.
• Wholesalers and suppliers of supplements for people with inherited metabolic diseases.
• Pharmacies who supply special foods.

Detail about this decision

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

There will also be a price increase from 1 August 2024 for the MSUD Maxamum and XMET Maxamum brands of supplements for inherited metabolic diseases. These products will remain fully funded for people who meet the eligibility criteria.

Some of the supplements below are already listed in either Section D or Part II Section H of the Pharmaceutical Schedule. As a result of this proposal the metabolic supplements listed in Section D and Part II Section H of the Pharmaceutical Schedule will be aligned. Individual macros for the various supplements will continue to be listed in Part II Section H.

Some supplements for inherited metabolic diseases that are currently funded via the NPPA pathway will also be listed on the Pharmaceutical Schedule for the first time.

Phenylketonuria

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Glycomacropeptide and amino acid contains some phenylalanine	Powder 33.4 g sachets	Vanilla	PKU GMPro 20 Vanilla	30	$936.00
Glycomacropeptide and amino acid contains some phenylalanine	Powder 12.5 g sachets	Unflavoured	PKU GMPro Mix-In	30	$468.00
Glycomacropeptide and amino acid contains some phenylalanine	Liquid, 250 ml carton	Neutral	PKU GMPro LQ	18	$280.80
Amino acid formula without phenylalanine	Powder 12.5 g sachets	Unflavoured	PKU First Spoon	30	$234.00
Amino acid formula without phenylalanine	Tabs	N/A	Phlexy 10	75 OP	$99.00
Amino acid formula without phenylalanine	Powder 28 g sachets	Neutral, Berry, Orange	PKU Lophlex Powder	30	$936.00
Amino acid formula without phenylalanine	Liquid 62.5 ml bottles	Juicy Berries	PKU Lophlex LQ 10	60	$939.00
Amino acid formula without phenylalanine	Liquid 125 ml bottles	Juicy Tropical, Juicy Berries, Juicy Orange	PKU Lophlex LQ 20	30	$936.00
Amino acid formula without phenylalanine	Oral semi-solid (berries) 109 g pot	Berries	PKU Lophlex Sensation 20	36 OP	$1,123.20
Amino acid formula without phenylalanine	Liquid 250 ml cartons	Forest fruit	Easiphen Liquid	18 OP	$540.00
Amino acid formula without phenylalanine	Powder 500 g can	Unflavoured, Orange	XP Maxamum	500 g OP	$320.00
Amino acid formula without phenylalanine	Powder 36 g sachets	Neutral, Vanila, Chocolate, Orange	PKU Anamix Junior	30	$393.00
Amino acid formula without phenylalanine	Liquid 125 ml bottle	Berry, Orange	PKU Anamix Junior LQ	125 ml OP	$13.10
Amino acid formula without phenylalanine	Powder 400 g can	N/A	PKU Anamix Infant	400 g OP	$174.72

Tyrosineamia types 1, 2 and 3:

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Aminoacid formula without phenylalanine and tyrosine	Powder 36 g sachets	Neutral	TYR Anamix Junior	30	$471.00
Amino acid formula without phenylalanine and tyrosine	Powder 400 g can	N/A	TYR Anamix Infant	400 g OP	$260.00
Aminoacid formula without phenylalanine and tyrosine	Liquid 125 ml bottles	Juicy Berries	TYR Lophlex LQ 20	30	$1,684.80
Aminoacid formula without phenylalanine and tyrosine	Liquid 125 ml bottles	Orange	TYR Anamix Junior LQ	36	$941.40

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

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Amino acid formula without valine, leucine and isoleucine	Powder 500 g can	Neutral, Orange	MSUD Maxamum	500 g OP	$454.71
Amino acid formula without valine, leucine and isoleucine	Powder 400 g can	Unflavoured	MSUD Anamix Infant	400 g OP	$260.00
Amino acid formula without valine, leucine and isoleucine	Powder 36 g sachets	Neutral	MSUD Anamix Junior	30	$750.00
Amino acid formula without valine, leucine and isoleucine	Liquid 125 ml bottles	Orange	MSUD Anamix Junior LQ	36	$941.40
Amino acid formula without valine, leucine and isoleucine	Liquid 125 ml bottles	Juicy Berries	MSUD Lophlex LQ 20	30	$1,684.80

Homocystinuria:

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Amino acid formula without methionine	Powder 500 g can	Neutral	XMET Maxamum	500 g OP	$480.42
Amino acid formula without methionine	Powder 400g can	Unflavoured	HCU Anamix Infant	400 g OP	$260.00
Amino acid formula without methionine	Powder 36 g sachets	Neutral	HCU Anamix Junior	30	$750.30
Amino acid formula without methionine	Liquid 125 ml bottles	Orange	HCU Anamix Junior LQ	36	$941.40
Amino acid formula without methionine	Liquid 125 ml bottles	Juicy Berries	HCU Lophlex LQ	30	$1,684.80

Glutaric Aciduria type 1

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Amino acid formula without lysine	Powder 400 g can	N/A	GA Anamix Infant	400 g OP	$260.00
Amino acid formula without lysine	Powder 18 g sachets	Neutral	GA Anamix Junior	30	$750.30

Methylmalonic and Propionic Acidemias

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Aminoacid formula without isoleucine, methionine, threonine and valine	Powder 400 g can	N/A	MMA/PA Anamix Infant	400 g OP	$260.00
Amino acid formula without methionine, threonine and valine	Powder 18 g sachets	Neutral	MMA/PA Anamix Junior	30	$750.30

Fat Modified Products

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Elemental feed with high medium chain triglycerides	Powder 100 g sachets	Neutral	Emsogen	10	$47.01

Carbohydrate and Fat with added vitamins and minerals

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Protein free supplement containing carbohydrate, fat with added vitamins and minerals	Powder 400 g can	Neutral	Energivit	400 g OP	$49.29

Essential Amino Acids

Chemical	Formulation	Flavour	Brand	Pack size	Price and subsidy
Essential aminoacid formula	Powder 200 g can	Neutral	Essential Amino Acid Mix	200 g OP	$313.73

 

The following supplements for PKU will be delisted Section D and Part II Section H of the Pharmaceutical Schedule from 1 January 2025:

Chemical	Formulation	Flavour	Brand	Pack size
Amino acid formula without phenylalanine	Liquid 62.5 ml bottles	Juicy citrus	PKU Lophlex LQ 10	60
Amino acid formula without phenylalanine	Liquid 62.5 ml bottles	Juicy orange	PKU Lophlex LQ 10	60
Amino acid formula without phenylalanine	Liquid	Unflavoured	PKU Anamix Junior LQ	125 ml

The Special Authority criteria for ‘Foods and Supplements for Inherited metabolic disease’ will be amended from 1 August 2024 in Section D of the Pharmaceutical Schedule as follows (deletions in strikethrough, additions in bold). These changes would apply to currently funded products in the ‘Foods and Supplements for Inherited metabolic disease’ group and the supplements funded because of this proposal.

The Hospital Indication restriction for ‘Metabolic Products’ will be amended from 1 August 2024 in Part II of Section H of the Pharmaceutical Schedule as follows (deletions in strikethrough). These changes would apply to currently funded products in the ‘metabolic products’ group and the supplements proposed to be funded as a result of this proposal.

Our response to what you told us

We are grateful for the time you took to respond to this consultation. We have summarised the main themes you raised and our responses.

Theme	Pharmac comment
Respondents were supportive of the proposed changes to the eligibility criteria and the expansion of the range of supplements available for people with inherited metabolic disorders.	We’re pleased to hear support for the proposal and the benefit it will provide people who use the supplements.
A respondent raised administrative issues with prescribing funded supplements for inherited metabolic diseases. They requested these issues be resolved before new supplements are funded.	We’ve worked with the suppliers and Health New Zealand to resolve these issues. If new issues arise contact Pharmac via enquiry@pharmac.govt.nz
A respondent raised concerns that wholesalers or pharmacies might have to pay the difference for the price increase for XMET Maxamum and MSUD Maxamum. The price change was proposed to occur from 12 July 2024. The responder highlighted that wholesalers may purchase stock from the supplier at the new (higher) price, and pharmacies could only receive reimbursement at the current (lower) price.	We have discussed this with the supplier, Nutricia, who has agreed to delay the implementation of new pricing for these supplements until 22 July 2024. This reduces the risk that wholesalers or pharmacies would have to cover the difference in price.
Respondents requested additional supplements to be funded including low protein foods and Phlexyvits and Fruitivits.	We understand there are more products that could be funded and would provide health benefits to people with inherited metabolic diseases. We’re considering an funding application for low protein foods for inherited metabolic diseases. This has been ranked on our Options for Investment List. We also have funding applications for Phlexyvits and Fruitivits. We received clinical advice about these applications at the October 2023 Special Foods Advisory Committee meeting. Both items received positive funding recommendations. October 2023 Special Foods Advisory Committee meeting record [PDF, 592 KB]

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.