CF sufferer puts hope in Labour victory
A $20m boost would help people with rare diseases. Report by Cate Broughton.
The parents of a 12-year-old boy who suffers from cystic fibrosis say a Labour victory is their only hope of getting life-changing drugs for their son.
Wayne and Kim Davidson discovered their son Tylah was born with the life-threatening genetic disease, and have searched for the drugs that could save his life ever since.
A few years ago they discovered Tylah had the specific gene mutation that a drug called Kalydeco targets, which Wayne Davidson described as akin to winning Lotto.
But Pharmac declined a funding application for Kalydeco from supplier Vertex in 2015 ‘‘based on the high cost and poor costeffectiveness’’, director of operations Sarah Fitt said.
The Davidsons considered going to Australia where the drug is publicly funded until they realised they would not be eligible as non-citizens.
Now they hope a Labour Government will deliver on a pledge to set up a $20m fund for rare diseases over four years.
Apart from a pilot for a contestable rare diseases fund in 2015 by Pharmac, the National Government had not supported separate resourcing model for medicines like Kalydeco.
National’s Jonathan Coleman said: ’’Pharmac needs certainty about the benefits these drugs provide, the likelihood of delivering better health gains than other treatments, and that it’s affordable.’’
The Davidsons say this leaves families like theirs with no hope.
‘‘This is our child, and there is a drug available that effectively cures him... and doubles his life span and it’s like... the elusive wonder drug, it is always going to be out of our reach because of Pharmac’s stance,’’ Kim Davidson said.
New Zealand Organisation for Rare Disorders (NORD) Collette Bromhead said the pilot fund was a ‘‘good start’’ but the decisions on funding were still made on value for money, rather than value for patients.
For now, the Davidsons continue with constant vigilance to protect their son from the ravages of the disease.
Daily use of a nebuliser, morning and night physiotherapy and religiously taking medications with food are critical to manage the build up of thick mucous in the lungs and the lack of enzymes in his pancreas.
‘‘The scariest thing for us is his health will decline, not get better,’’ Kim Davidson said.
An estimated 36 people would benefit from Kalydeco in New Zealand, which costs more than $382,000 per year.
A recent test showed Tylah’s lung function had dropped to 60 per cent and increasing fatigue made it hard for him to keep up with school work and sports.
Cystic Fibrosis New Zealand (CFNZ) which represents patients like Tylah advised the Davidsons early this year they would be taking a step back from lobbying for Kalydeco.
Chief executive Jane Bollard said as a small charity, CFNZ lacked the ‘‘resources and information required to carry out a full lobbying campaign, at this point in time.’’
The organisation fully supported public funding of Kalydeco but had decided to spend time gathering information and support from doctors to strengthen future lobbying work, Bollard said.
The change was ‘‘gutting’’ Kim Davidson said. She said the onus was on the Government to ensure access for its citizens.