Proposal to fund supplements for phenylketonuria and other inborn errors of metabolism

What we’re proposing

We are seeking feedback on a proposal to fund a range of nutritional supplements from Cortex Health and Vitaflo for people with phenylketonuria (PKU) and other inborn errors of metabolism from 1 February 2024.

This proposal would also result in changes to the eligibility criteria for currently funded foods and supplements for inborn errors of metabolism in Section D and Part II Section H of the Pharmaceutical Schedule to simplify access for people with inborn errors of metabolism.

Feedback to this consultation will help us decide whether to progress this proposal. Consultation closes at 5:00pm, 28 November 2023 and feedback can be emailed to consult@pharmac.govt.nz

We are working with other suppliers of supplements for inborn errors of metabolism and hope to consult on additional proposals in the future. We are also interested in hearing from any suppliers of supplements for inborn errors of metabolism 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 significantly expand the range of supplements for inborn errors of metabolism in New Zealand, including glycomacropeptide (GMP) based supplements for some inborn errors of metabolism that would be made available for the first time.

From 1 February 2024 the supplements would be funded for use in the community and Te Whatu Ora Hospitals, subject to access criteria that target funding to people with inborn errors of metabolism.

The proposed range of supplements would be listed in Section D of the Pharmaceutical Schedule (known as the community schedule) and Section H of the Pharmaceutical Schedule (known as the hospital medicines list).

The proposed range of supplements would be funded for people with a range of inborn errors of metabolism including:

• phenylketonuria
• maple syrup urine disease
• homocystinuria
• tyrosinemia
• glutaric aciduria
• propionic acidaemia
• 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 would use the supplements included in this proposal in the first year of funding, increasing at a rate of approximately 3-4 people each year.

Who we think will be interested

• People with inborn errors of metabolism and their whānau, family and caregivers
• Metabolic specialists and clinicians
• Dietitians, General Practitioners and other healthcare professionals involved in the care of people with inborn errors of metabolism.
• Suppliers of supplements for inborn errors of metabolism.

About inborn errors of metabolism

Inborn errors of metabolism (IEM) are genetic conditions meaning they are often inherited, present at birth and lifelong. These disorders are often caused by defects in the body proteins (usually an enzyme or a transporter protein) involved in the metabolism of protein, fat or carbohydrate from food. These may help break down nutrients from food and stop compounds building up in the body at toxic levels or may be involved in helping produce energy for the cells of the body to work properly. 

Early diagnosis of treatable IEMs is vital to the outcome. Some IEMs can be identified through newborn screening in the first week of life. Without treatment, IEMs can lead to a range of negative consequences, the most significant being damage to the developing brain. With early identification and treatment, many of the adverse health effects of IEMs can be avoided.

Some IEMs can be treated effectively with dietary changes. These diets can be very restrictive and require the use of specialist supplements to meet nutritional needs and allow adequate growth.

One of the IEMs that new supplements would be funded for is phenylketonuria (PKU). People with PKU are unable to breakdown phenylalanine, one of the amino acids or ‘building blocks’ of protein. Phenylalanine 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, similar to other amino acid breakdown disorders, includes a very low protein diet with strict avoidance of all high protein foods. A supplement free from phenylalanine or very low in phenylalanine is prescribed to meet protein and micronutrient requirements.

Why we’re proposing this

There are currently a limited number of funded supplements available for people with PKU and other inborn errors of metabolism. We have received feedback from stakeholders that expanding the range of supplements available would provide greater choice and significantly improve the lives of people with inborn errors of metabolism in New Zealand.

Supplements for PKU which contain glycomacropeptide are currently ranked on Pharmac’s ‘Only if Cost Neutral or Cost Saving List(external link)’ This proposal would result in supplements for PKU that contain glycomacropeptide being funded at pricing that is cost neutral or lower to currently available supplements for PKU.

In addition, a number of supplements for inborn errors of metabolism are frequently provided  through Pharmac’s Exceptional Circumstances Framework.

Listing the proposed range of supplements on the Pharmaceutical Schedule would reduce the administrative load associated with this process for metabolic specialists and healthcare practitioners and provide certainty of ongoing supply for these supplements.

We have negotiated provisional agreements with two suppliers of supplements for inborn errors of metabolism: Cortex Health Pty Ltd and Vitaflo Australia Pty Limited.

Details about our proposal

From 1 February 2024 the following supplements for phenylketonuria and other inborn errors of metabolism would 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
Amino acid formula without phenylalanine	Powder 49 g sachets	Neutral	Camino Pro Bettermilk	Cortex Health	30	$673.92
Amino acid formula without phenylalanine	Liquid, 250 ml carton	Chocolate, neutral	PKU Glytactin RTD 15	Cortex Health	30	$684.45
Amino acid formula without phenylalanine	Liquid, 250 ml carton	Vanilla, Coffee Mocha	PKU Glytactin RTD 15 Lite	Cortex Health	30	$684.45
Amino acid formula without phenylalanine	Powder 16 g sachets	Neutral	PKU Build 10	Cortex Health	30	$449.28
Amino acid formula without phenylalanine	Powder 32 g sachets	Chocolate, Vanilla, Smooth, Raspberry Lemonade	PKU Build 20	Cortex Health	30	$898.56
Amino acid formula without phenylalanine	Powder 20 g sachet Hydration beverage	Berry, Orange	PKU Restore Powder	Cortex Health	60	$449.28
Amino acid formula without 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	Unflavoured	TYR Explore 5	Vitaflo	30	$349.65
Amino acid formula without phenylalanine and tyrosine	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 Inborn Errors of Metabolism’ would be amended from 1 February 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 Inborn Errors of Metabolism’ 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 February 2024 in Part II Section H as follows (deletions in strikethrough, additions in bold). 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.

The criteria above would also apply to PKU Build 20 and PKU Sphere 20 which are currently funded in the ‘Other supplements for PKU’ Group in Section D and Part II Section H of the Pharmaceutical Schedule Funding for these supplements from 1 February 2024 would be via SA1108 (Community) [PDF](external link) or RS1232 (Hospital) [PDF](external link) . We understand there aren’t any people currently who would require a new SA number as a result of the proposed changes; however we welcome feedback on this.

PKU GMPro Ultra Lemonade, which is supplied by Nutricia and is not in scope of this proposal  would 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.

We have received feedback that the currently funded foods and supplements for inborn errors of metabolism and the range of supplements that would be funded as a result of this proposal may not be being used as a supplement to the Ketogenic diet in people with epilepsy, and therefore this aspect of the criteria may not be required.

Currently, we have proposed to retain this aspect of the criteria however we are seeking your feedback on the appropriateness of this.

Accessing supply of the proposed supplements

Cortex Health

Stock of Cortex Health’s range of supplements would initially be held in Australia and would be delivered regularly to New Zealand. Stock would be able to be ordered directly from Cortex Health by Community Pharmacies and Te Whatu Ora Hospitals (including organisations acting on their behalf).

To cover the costs associated with the delivery of the supplements to New Zealand it is proposed, through a contractual arrangement with Pharmac, that Cortex Health would 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 would be delivered within a maximum of 14 days where stock is required to be delivered from Australia, or 21 days where stock is required to be delivered from the USA.

Cortex Health’s supplements would initially be supplied directly to Community Pharmacies and Te Whatu Ora Hospitals rather than utilising a wholesaler. Cortex Health has requested this arrangement so that it can closely manage its supplements and their expiry dates to ensure supplements are not wasted, particularly in the months immediately following availability in New Zealand while demand will be uncertain.

Cortex Health’s supplements would be provided to New Zealand with a minimum shelf life remaining 6 months.

We note that the above arrangement is a temporary exception to how stock is usually ordered via wholesalers. If this proposal is progressed Cortex Health and Pharmac would 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 at this point stock would be able to be ordered via wholesalers in the same way as other special foods.

Vitaflo

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

Vitaflo’s range of supplements would be available to order by Community Pharmacies and Te Whatu Ora Hospitals (including organisations acting on their behalf) through the wholesaler supply chain via CDC Pharmaceuticals.

Vitaflo’s proposed range of supplements would 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 would provide replacement stock at no extra cost.

If this proposal is approved, these supply arrangements would be reviewed by Pharmac and Vitaflo within 12 months of Vitaflo’s supplements being available in New Zealand and amended if required.

To provide feedback

Send us an email: Consult@Pharmac.govt.nz by 5:00pm, Tuesday, 28 November 2023.

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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