Pharmac seeks feedback on proposal to fund nutritional supplements for metabolic conditions
Te Pātaka Whaioranga – Pharmac is consulting on a proposal to fund additional specialist nutritional supplements for people with genetic metabolic conditions, including phenylketonuria (PKU).
“We have heard from clinicians that people affected by inborn errors of metabolism could benefit from more treatment options,” says Pharmac’s Director Pharmaceuticals, Geraldine MacGibbon.
“Our role is to invest in new medicines and manage the supply of those currently funded. At the moment we can’t make any investments that would incur a cost to our budget. This proposal would allow us to expand the range of funded supplements in New Zealand, including some that would be made available for the first time without incurring additional cost.
Inborn errors of metabolism are genetic, often inherited, lifelong conditions, usually identified through screening at birth. The errors in metabolism can cause compounds in the body to build up to toxic levels, leading to adverse effects like cognitive impairment. People with inborn errors of metabolism often need to follow very restrictive diets so have to use specialist supplements meet their nutritional needs.
Pharmac has negotiated provisional agreements with two suppliers, Cortex Health and Vitaflo, to fund a range of nutritional supplements suitable for people with a of inborn errors of metabolism including:
- maple syrup urine disease
- 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 full year of funding, increasing at a rate of approximately 3-4 people each year.”
“Historically, some people with inborn errors of metabolism have accessed supplements through our exceptional circumstances, NPPA pathway. By listing treatments on the Pharmaceutical Schedule we can reduce the administration and time associated with this process and ensure access to all people with these conditions.”
Feedback in response to this consultation will help Pharmac decide how to progress this proposal. Consultation closes at 5:00pm, 28 November 2023 and feedback can be emailed to email@example.com