Proposal to widen access to cystic fibrosis medicines welcomed

Rare Disorders NZ is thrilled that Pharmac is proposing to fund Trikafta and new treatment Alyftrek for all children with cystic fibrosis, and fund Kalydeco to everyone with an eligible diagnosis.

In 2023, Pharmac announced it would fund Trikafta for children with cystic fibrosis 6 years or older, leaving families of younger children having to decide if they could afford funding the costly medicine themselves or even consider moving to Australia to access the medicine.

Rare Disorders NZ was pleased to see the Associate Minister of Health David Seymour recognise the lifechanging difference it will make to families affected by cystic fibrosis in his statement today, and that he is well aware of the difficult decisions families are faced with when their medicine is not funded.

“We cannot overstate the difference it makes to families impacted by rare disorders when they can access a medicine proven to be effective for their condition. Not only does it keep the affected person healthier for longer and less dependent on the already overstretched health system, but it also reduces the impact on the wider family. They no longer have to consider uprooting their whole family, or contemplate taking out a second mortgage to access the medicine ” says Chris Higgins, Chief Executive of Rare Disorders NZ.

“Rare disorder medicines tend to struggle to compete for funding under the current assessment pathway for medicines, and Rare Disorders NZ has long been calling for equitable access to rare disorder medicines through a separate assessment pathway.”

“We are pleased Minister Seymour acknowledges the value in Pharmac working with patient groups and the importance in making the system work better for the people it serves. Those in the rare disorder community waiting for one or more of the 18 rare disorder medicines on Pharmac’s Options for Investment list will be heartened to hear this from the Minister.” says Higgins.

Rare Disorders NZ will continue to work to strengthen Pharmac’s relationship with rare disorder patient groups and advocate for a separate assessment pathway for rare disorder medicines.