Proposal to fund supplements for phenylketonuria and other inherited metabolic disease

What we’re proposing

We’re seeking feedback on a proposal to fund more nutritional supplements for people with phenylketonuria and other inherited metabolic diseases.

This proposal would also result in changes to the eligibility criteria for all currently funded foods and supplements for inherited metabolic diseases in Section D and Part II Section H of the Pharmaceutical Schedule. These changes are proposed to simplify the criteria and align it with current prescribing practice for these supplements.

Feedback to this consultation will help us decide how we progress this proposal. Consultation closes at 5:00 pm, Tuesday 5 July 2024 and feedback can be emailed to consult@pharmac.govt.nz.   

We’re interested in hearing from any suppliers of supplements for inherited metabolic disease who we are not already in contact with that may be interested in supplying the New Zealand market.

What would the effect be?

This proposal would expand the range of supplements available for inherited metabolic disease in New Zealand, including glycomacropeptide (GMP) based supplements for people with phenylketonuria.

From 1 August 2024, the proposed supplements would be funded for use in the community and in Health New Zealand – Te Whatu Ora Hospitals, subject to eligibility criteria. The proposed range of supplements would be funded 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 would use the supplements included in this proposal in the first year of funding. After the first year, 3-4 people would start on these supplements each year.

Who we think will be interested

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

About inherited metabolic disease

Inherited metabolic diseases (IMDs) are genetic conditions meaning they are often inherited, present at birth and lifelong. These disorders are often caused by differences in the body proteins. These proteins can do a range of things, including:

• breaking down nutrients from food and stopping compounds building up in the body at toxic levels or
• helping produce energy for the cells of the body to work properly.

Some IMDs can be managed effectively with dietary changes. These diets can be very restrictive and require the use of specialist supplements to meet peoples’ nutritional needs and support a person’s growth.

PKU is one of the diseases the new supplements would be funded for. People with PKU are unable to breakdown phenylalanine, one of the amino acids or ‘building blocks’ of protein. Phenylalanine then builds up in the blood and brain and can lead to brain damage if treatment is not started within the first few weeks of life.

Treatment for PKU includes a very low protein diet with strict avoidance of all high protein foods. This is similar to other amino acid breakdown disorders. People are usually prescribed a supplement without phenylalanine (or very low in phenylalanine) to meet ensure they are getting enough protein and micronutrients.

Why we’re proposing this

There are currently a limited number of funded supplements available for people with PKU and other inherited metabolic diseases. We’ve received clinical advice that expanding the range of supplements available would provide greater choice and improve the lives of this population.

In February 2024, we funded a new range of supplements. This proposal would provide additional options from another supplier. The proposed supplements would be funded at pricing that is cost neutral or lower to currently available options.

Currently some people receive these products  through Pharmac’s Exceptional Circumstances Framework (NPPA). Listing the proposed range of supplements on the Pharmaceutical Schedule would reduce the administrative burden for health care professionals and provide certainty of ongoing supply for these supplements.

Details about our proposal

We have negotiated a provisional agreement with Nutricia for supplements for inherited metabolic diseases.

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

Currently, some items are only listed in Section D or Section H. These would be listed in both Section D and Section H from 1 August 2024. This proposal also includes some items that are funded via NPPA. These would 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	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
Amino acid 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
Amino acid formula without phenylalanine and tyrosine	Liquid 125 ml bottles	Juicy Berries	TYR Lophlex LQ 20	30	$1,684.80
Amino acid 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	36	$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 amino acid formula	Powder 200 g	Neutral	Essential Amino acid Mix	200 g OP	$313.73

The Special Authority criteria for ‘Foods and Supplements for Inherited metabolic disease’ would be amended from 1 August 2024 in Section D 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 proposed to be funded as a result of this proposal.

The Hospital Restriction for ‘Metabolic Products’ would be amended from 1 August 2024 in Part II Section H 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.

When we consulted in November 2023 on a similar proposal, we heard that the funded supplements for inherited metabolic diseases would not be suitable for use in a ketogenic diet because of their high carbohydrate content. In response we’ve sought feedback from specialist clinicians who have confirmed these supplements are not currently being used by people on a ketogenic diet and would not be suitable in this setting because of their carbohydrate content. As a result, we have proposed to remove this part of the criteria. This change would not stop any people with inherited metabolic diseases from accessing these supplements. These changes would apply to all currently funded supplements for inherited metabolic diseases..

Supply of Nutricia’s newly funded supplements would be available to order from wholesalers from 12 July 2024. Nutricia’s currently funded supplements for metabolic diseases continue to be available during this consultation period.

To provide feedback

Send us an email: consult@pharmac.govt.nz by 5:00pm, Friday 5 July 2024.

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.

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