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:

  1. For the dietary management of inherited metabolic diseases; or
  2. 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.

Your feedback may be shared

Feedback we receive is subject to the Official Information Act 1982 (OIA). Please be aware that we may need to share your feedback, including your identity, in response to an OIA request. This applies to anyone providing feedback, whether they are providing feedback themselves or for an organisation, in a personal or professional capacity.

We can only keep feedback confidential as allowed under the OIA and other related laws. If you want any part of your feedback treated as confidential, you need to tell us. Please let us know if you want to keep part of your feedback confidential, and why. Is it commercially sensitive, confidential or proprietary, or personal information? Clearly state this and tell us which parts of your feedback you want to keep confidential for these reasons. We will consider your request under our OIA requirements.