Fourteen-year-old Bruno Cettina is living his best life thanks to a medicine funded by PHARMAC. According to his proud mum, he is a keen sportsman, a musician, an above average student at school and is tackling life with vigour.
Bruno (left) is also one of 35 New Zealanders with cystic fibrosis who have the G551D mutation, a disease that is able to be treated by a medicine called ivacaftor (branded as Kalydeco). Cystic fibrosis is an inherited life-threatening disorder that damages the lungs and digestive system. Bruno also has type 1, cystic fibrosis-related diabetes.
PHARMAC approved funding for ivacaftor early last year and it is being added to the pharmaceutical schedule on 1 March 2021 following Medsafe’s approval.
“Without PHARMAC’s funding of and support, Bruno would be in a whole different ball game”, says Ana Waalkens, Bruno’s mum.
“We understand and appreciate that cystic fibrosis has a significant and distressing impact on individuals and their whānau,” says PHARMAC deputy medical director Dr Pete Murray.
Medsafe approval is an important process in ensuring the safety of a medicine before it is made available for New Zealanders. PHARMAC decided to enable funding for ivacaftor through our exceptional circumstances process, while waiting for approval from Medsafe for both formulations to be granted.
“The small number of patients and their high health need was key to this decision, as was the fact that we could be confident that our exceptional circumstances funding approval process would ensure patients were able to give their informed consent to using an unapproved medicine prior to commencing treatment,” says Dr Murray.
“PHARMAC prefers to fund medicines that have been assessed and approved by Medsafe, however, we need to balance this with making medicines available in a timely manner to meet a high health need.”
Bruno started accessing funded ivacaftor to treat his cystic fibrosis in March last year.
“Bruno’s life and ours has improved significantly because of this medicine,” says Ana. “He is now a good weight; he is fit and healthy and it has changed our outlook for the future which we wouldn’t even have considered before.”
For most of Bruno’s life, he was admitted to Starship Hospital, on average, three times a year. Since starting ivacaftor 11 months ago Bruno has not been admitted to Starship at all. His lung capacity, damaged by the cystic fibrosis, has responded well to ivacaftor, improving by 91% in recent tests. His cystic fibrosis related diabetes is more stable than it has ever been.
“Ivacaftor is an expensive medicine costing $383,225 per person per year, although PHARMAC was able to negotiate a confidential discount on this price,” says Dr Murray. “We often hear people saying that PHARMAC doesn’t fund high-cost medicines. It is a little-known fact that 85% of PHARMAC’s budget is spent on medicines used by just 10% of the patients accessing funded medicines each year. These patients are using high-cost medicines.
And for people like Bruno, it has been a game changer. “Bruno is doing so well,” says Ana. “He is living the dream as all 14-year-old boys should be.”
“We know the huge impact medicines like ivacaftor can have on an individual’s health and wellbeing, and we are thrilled to hear stories like Bruno. This is why we are always working to make more medicine available to New Zealanders,” concludes Dr Murray.