Pharmac proposes funding for rare disorder treatment

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Source: PHARMAC

Pharmac is proposing to fund nitisinone for people with the rare disorders – tyrosinemia type 1 and alkaptonuria.

Nitisinone is currently funded through Pharmac’s Named Patient Pharmaceutical Assessment (NPPA) pathway. Under this proposal, it would be listed on the Pharmaceutical Schedule, making it easier for clinicians to prescribe and for people to access treatment.

If approved following the public consultation, approximately 10 people with these rare disorders would benefit from access to the medicine in the first year of funding.

 “This proposal would make it easier for people living with these rare conditions to access nitisinone,” says Pharmac’s Acting Manager of Pharmaceutical Funding, Claire Pouwels.

“It would also make it easier for clinicians to prescribe this treatment, without needing to apply through NPPA each time.”

Tyrosinemia type 1 and alkaptonuria are lifelong conditions that affect how the body breaks down tyrosine, an amino acid. Without treatment, harmful substances can build up and cause serious health problems. Nitisinone, alongside a special diet, can help prevent or reduce these effects.

People currently receiving nitisinone through NPPA would continue to get their treatment as usual but may need to change to the LogixX Pharma brand from 1 February 2026. If people experience issues with the new brand, there are pathways in place within the Exceptional Circumstances framework to access an alternative if clinically necessary.

Pharmac wants to hear from clinicians, consumer advocacy groups, and others about how this proposal could work in practice. Feedback can be provided through Pharmac’s website until 5pm Friday 12 December.

Following public consultation, Pharmac will make a decision on the funding proposal. If approved, it would be funded from 1 February 2026.

MIL OSI

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