Hope on the Horizon: New Zealand Considers Expanding Access to Life-Extending Cystic Fibrosis Drug
New Zealand families are cautiously optimistic as the government considers expanding access to trikafta, a “miracle” drug offering a new lease on life for children battling cystic fibrosis (CF).
Two-year-old Luca Rooney is one of more than 50 young children in New Zealand who could benefit from the expanded access. Luca’s parents, Liam and Courtney Rooney, learned of their son’s diagnosis at just two weeks old, facing the grim reality that without treatment, he might not live beyond his thirties due to organ damage.
Understanding Cystic Fibrosis
Cystic fibrosis is a genetic condition that causes a buildup of thick, sticky mucus in vital organs, particularly the lungs and pancreas. This mucus traps bacteria, leading to frequent and severe infections. While there is currently no cure, advancements in treatment have significantly improved the quality of life for those living with CF.
Trikafta and the OFI List
Trikafta, a modulator drug, addresses the underlying cause of CF in many patients. It has been approved for use in individuals aged six years and older in New Zealand, and is now under consideration for funding for children aged two to five. Inclusion on the OFI (Options for Investment) list doesn’t guarantee funding, but signals that Pharmac, New Zealand’s pharmaceutical management agency, will prioritize the drug for funding consideration as budget allows. The ranking of drugs on the OFI list is not publicly available.
A Financial Burden for Families
The financial implications of accessing Trikafta independently are staggering. The annual cost of roughly $400,000 is prohibitive for most families, forcing them to wait for potential public funding.The Rooneys face the agonizing possibility that Luca could become too ill, or require a lung transplant, before he reaches the age of six, possibly disqualifying him from future Trikafta eligibility.
This uncertainty has led to a highly protective lifestyle for Luca, including avoiding daycare, undergoing twice-daily physiotherapy, using a nebuliser – a type of inhaler – most days, taking vitamins three times a week, and meticulously avoiding exposure to potentially harmful bacteria found in water. “So anytime he’s playing anywhere, we’re basically standing over him just to make sure that he’s not anywhere near any water that could affect him,” Rooney explained.
Despite these precautions, Luca has spent much of his short life in and out of the hospital, and has been on antibiotics for half of his two years.
The Potential of Early Intervention
Starship respiratory specialist Dr. Julian Vyas emphasized the importance of early intervention. “We know even by a year or two of age children have evidence of damage and evidence of harm to their lungs on CT scans,” he stated. “So the rationale is, if we can intervene sooner, by starting the Trikafta sooner, we can maintain wellbeing for longer, and there’s a number of benefits from that. It’s the idea of nipping things in the bud.”
Dr. vyas also highlighted the significant burden placed on parents,who often take on the roles of nurse,doctor,and physiotherapist to care for their children. “It’s an ongoing thing, it’s not you need it for two weeks, job done. This is for the foreseeable future,” he said. “A number of parents have said to me, it kinda shifts that dynamic from being mum or dad, into being a nurse, doctor, physio all rolled into one.”
Advocacy and Hope
Simone Brown, head of Cystic Fibrosis NZ (CFNZ), called the inclusion of Trikafta for two to five-year-olds on the OFI list “an importent milestone.” She added, “starting this medicine from two years of age is vital to prevent deterioration, preserve lung function, and protect children with cystic fibrosis from significant decline.” CFNZ continues to advocate for broader access to the drug, having already witnessed it’s transformative effects on hundreds of New zealanders living with CF.
The Rooneys, along with other families, remain hopeful that the government will prioritize funding for Trikafta, offering a brighter future for Luca and other young children battling this challenging condition. “It would mean everything to us,” Rooney said.
