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.
Special Authority for Subsidy
Initial application from any relevant practitioner. Approvals valid without further renewal unless notified where patient requires dietary management of inherited metabolic disorders. for applications meeting the following criteria:
Either
1 Dietary management of inherited metabolic disorders.; or
2 for use as a supplement to the Ketogenic diet in patients diagnosed with epilepsy.
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.
Restricted
Initiation
Any of the following Either:
- For the dietary management of inherited metabolic diseases; or
- Patient has adrenoleukodystrophy.; or
3.for use as a supplement to the Ketogenic diet in patients diagnosed with epilepsy.
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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