Rare Disorders NZ congratulates Kalydeco campaigners
27 January 2020
Rare Disorders NZ (RDNZ) congratulates campaigners after PHARMAC announced their decision to fund a medicine for people living with cystic fibrosis in New Zealand.
“We congratulate Cystic Fibrosis NZ (CFNZ) and especially Kalydeco for Kiwis for their tireless campaigning to ensure access to this life-changing medication,” says RDNZ Chief Executive Lisa Foster.
“RDNZ, and the support groups we represent including CFNZ, are hopeful this decision signals the opportunity for greater access to other life-changing medicines for all New Zealanders living with a rare disorder,” says Lisa.
“It’s time to take rare disorders seriously to ensure equitable health outcomes for everyone. Rare is part of our communities and deserves a fair go.”
Cystic fibrosis is a serious illness that affects lungs and digestion, and is a rare genetic disorder affecting over 500 New Zealanders. PHARMAC have reached a provisional agreement with Vertex Pharmaceutical to fund Kalydeco for the around 30 people who have a specific rare gene type. Kalydeco is the first medication that fixes the underlying cystic fibrosis gene defect, essentially turning off cystic fibrosis.
The agreement is provisional and the next process is one of consultation. The provisional agreement between PHARMAC and Vertex means everyone in New Zealand that can benefit from this medicine will be able to access it. The plan is to have the medicine available to patients from the 1 March 2020.
Rare Disorders NZ (formerly NZORD) is the connector hub and collective voice for patients and families affected by rare disorders. Rare diseases affect 6% of New Zealanders.
There is no acknowledged definition of rare disease in NZ, however RDNZ follows the European Union policy which defines a disorder as rare when it affects fewer than 1 in 2000. More than 6,000 rare disorders collectively affect around 300,000 New Zealanders. Of this number, 50% are children.