Cystic fibrosis girl denied life-saving drug

DEVASTATING NEWS: Sinead Brown's family says she could die within three years.
DEVASTATING NEWS: Sinead Brown's family says she could die within three years.

Government funding for a drug that could save the life of a 12-year-old Dunedin girl with cystic fibrosis has been denied, with her family believing it is because of the cost.

The decision is a "death sentence" for Sinead Brown, her mum Andrea Neame says.

Kalydeco was cleared for use in New Zealand in December, but carries a price tag of $360,000 a year.

The drug has raised the quality of life for some cystic fibrosis patients and is being used in the US, Australia and parts of Europe.

Sinead was born with severe respiratory and nutritional problems that cause her to stay in hospital for weeks at a time.

Without the drug it was likely Sinead would die in the next two or three years, Neame said.

The news was "devastating" for the family who have been waiting for Pharmac's decision for six months.

In a written statement, Pharmac director of operations Sarah Fitt said she could not comment on individual cases but sympathised with Sinead and her family.

"In making the decision, Pharmac looked very closely at the patient's individual situation and health need, and took advice from a number of clinical experts," Fitt said.

"We have explained the decision personally to the patient's treating clinician, and we have offered to meet with the patient's family."

The family is still waiting on a letter formally explaining why Kalydeco was denied but expects it to come down to cost.

Without government funding there is a possibility the company that manufactures the drug, Vertex, will provide it to Sinead on compassionate grounds.

Neame is waiting to hear back from it.

While she continues to fight for access to Kalydeco, Sinead's condition continues to worsen.

"She's pretty sick. Her lung function is about 31 per cent at the moment ... I'm pretty sure it's the lowest it's ever been," Neame said.

In September Sinead had an operation that wrapped her stomach around her oesophagus so food could not aspirate into her lungs as it had been doing.

Since the painful procedure Sinead had not been herself, Neame said.

"She's actually been refusing hospital admission at the moment and that's been really difficult for the doctors."

Sinead was normally a happy child but lately she had gone through times when she did not want to see or talk to anyone, Neame said.

"I think she feels like everybody's letting her down right now."

Kate Russell, chief executive of the Cystic Fibrosis Association of New Zealand, said she was "very sad" to hear Sinead's application for funding had been denied.

"Obviously I want to know what the grounds are that they've turned it down on," Russell said.

"I think we can anticipate the cost is obviously a very big barrier, but I want to know the medical and scientific justification for turning her down."

There was no doubt in her mind that Kalydeco would vastly improve Sinead's quality of life, Russell said.

Kalydeco was targeted for cystic fibrosis patients with the same gene mutation as Sinead. About 4 per cent of patients had this mutation which equated to 26 people in New Zealand, she said.

Part of the reason the cost of the drug was so high was because it could help only a small number of people, but Vertex had to cover the costs of research and development, Russell said.

"I know we're in a rationed system here, we've only got so much we can spend on our healthcare system ... I guess I would just always ask people what they would do if it was their child."

Pharmac's decision could not be appealed unless there was new information or it was felt it had incorrectly followed process, she said.

"As far as we're concerned it's not over. We will be looking very hard at why this has been turned down and we will continue to fight on behalf of this young lady."

Fairfax Media