Cystic fibrosis drug treatment funding in the media

19 September 2017

Pharmac’s recent decision not to fund a revolutionary drug for cystic fibrosis (CF) patients was the focus of an article in the Sunday Star Times last weekend.

NZORD Chief Executive Dr Collette Bromhead says that Pharmac's contestable rare disease fund in 2015 was a good start, however decisions on funding were still made on value for money, rather than value for patients.

In 2012 Vertex Pharmaceuticals released a revolutionary drug called Kalydeco that treats the underlying causes of CF. It does this by addressing one of the main malfunctions of the CF transmembrane conductance regulator (CFTR) protein. Kalydeco binds to the CFTR and enables it to start efficiently shuttling chloride ions back and forth.

Unfortunately, the drug is only effective for around four percent of CF patients, being those who carry the rare G551D gene mutation. One such patient is 12-year-old New Zealander Tylah Davidson, whose parents are desperate to get him this life changing medication. At over $300,000 per year, without funding from Pharmac the medication is out of reach for this family.

Why is the cost so high? The benefits of the drug are limited to a small patient population but the cost of discovery and development is no less than for other drugs. However, in the US, Australia and many other countries the cost is still considered cheaper than having to pay for extended hospital stays and other stopgap measures that CF patients undergo without Kalydeco.

Tylah's family are now hoping for a change of government as Labour have pledged a $20million fund towards rare diseases over four years.