Pharmac expands funded supplement options for people with inherited metabolic diseases

Media release Medicines

Pharmac – Te Pātaka Whaioranga is funding more nutritional supplements for people with inherited metabolic diseases from 1 February 2024.

“We understand that people living with inherited metabolic diseases, like phenylketonuria, will benefit from a greater choice of supplements to support their health and wellbeing,” says Pharmac’s Director, Pharmaceuticals, Geraldine MacGibbon.

Inherited metabolic diseases are genetic, lifelong conditions, usually identified through screening at birth. Metabolic diseases can cause compounds in the body to build up to toxic levels, leading to adverse effects such as cognitive impairment. People with inherited metabolic diseases often need to follow very restrictive diets and use specific supplements to meet their nutritional needs.

“We expect approximately 120 people in New Zealand will use these supplements in the first full year of funding, increasing by approximately 3-4 people each year.”

“We are pleased to have more treatment options available for this group of people, especially considering the significant constraints currently of our fixed budget and our ability to make new investments. Our team works hard to negotiate with suppliers to manage currently funded medicines and invest in new ones.”

The range of funded nutritional supplements will increase. This includes some that will be funded for the first time. The new treatments, supplied by Cortex Health and Vitaflo, will be suitable for people with a range of inherited metabolic diseases 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’re grateful for the feedback we received from advocacy groups and clinicians in response to the original consultation on our proposal to fund these supplements. It is important we use this feedback to inform our decisions. We’ve heard there is more to consider regarding access to these treatments, so we will continue these conversations with the health sector and the affected communities.”