'Morally bankrupt': Government stalls funding for life-saving cystic fibrosis drug for children

A lively almost two-year-old, it's hard to fathom she is expected to die before the age of 37.

Anna has cystic fibrosis, a disease that claims half of sufferers by their mid-20s.

But what is just as difficult for her parents, Penny France and Paul McMullen, is knowing there is a drug available overseas that is meant to improve these statistics, yet is not government-subsidised in Australia for children Anna's age.

Doctors at the Royal Children's Hospital broke the news to the couple after a routine test at five weeks discovered she had the incurable disease.

"It came as a huge shock," Ms France said. "It's something that we have never 100 per cent gotten over, but we learn to deal with it every day."

The condition causes an excess of mucus within sufferers' lungs, airways and digestive systems, leading to recurrent infections and irreversible damage.

Anna takes daily antibiotics and her parents administer 30 minutes of physiotherapy twice a day to shift the life-threatening mucus.

The couple had clung on to hope when told during the appointment about new drug Kalydeco - the first to treat the underlying cause for Anna's gene type and stop mucus build-up, not only increasing life expectancy but drastically improving quality of life.

Dubbed a "game-changer", and already on the Pharmaceutical Benefits Scheme for cystic fibrosis sufferers over the age of six, the doctors said it was likely to be made available for two to five-year-olds by Anna's second birthday this February.

American drug company Vertex is understood to want more than $200,000 per person per year for the drug - a price too high for the couple to pay privately.

There are about 30 children with the same cystic fibrosis gene in the age-bracket, who have families also awaiting the drug's availability.

But the Pharmaceutical Benefits Advisory Committee deferred the decision to make the drug available days before Christmas.

"We felt crushed," Ms France said. "Every single day, the Australian Government and Vertex continue to discuss and negotiate over price, children with cystic fibrosis are becoming more and more sick.

"They are contracting ongoing lung infections, which cause irreversible lung damage, diminish their quality of life, and cut years from their precious lives."

A health department spokeswoman said the committee noted that Vertex had difficulty demonstrating the drug's benefit and the cost-effectiveness of supplying it at an earlier age.

"Clear and reliable scientific evidence is required to show that the medicine works for the intended purpose, is unlikely to cause more harm than good, and that the benefit it offers justifies the money expected to be spent by the Australian community," she said.

"The PBAC and the Department of Health are working with Vertex to consider the outstanding issues raised by the PBAC to resolve this matter as soon as possible."

However, Cystic Fibrosis Australia chief executive Nettie Burke said it was a case of Vertex being "morally bankrupt" and the government not wanting to foot the bill for the Therapeutic Goods Administration-approved drug.

"It's about price. It's obscene," she said.

"It's clinically sound - we need access while Vertex and the government negotiate.

"It's 30 children - that's all we're asking for. Let's not put these little lives at risk"

Ms Burke said the drug, which was subsidised for children aged two to five in the US, Britain, Germany, Austria, Belgium and the Netherlands, turned the lives of sufferers around.

"It allows people to live a life close to normal - they can get married, have children, work . . . little kids can go to school," she said.

Anna's parents want Health Minister Sussan Ley to intervene on the decision.

A spokeswoman for the minister said she couldn't comment as the PBAC was independent of the government.

"The government accepts PBAC recommendations without fear or favour," she said.

"It is entirely appropriate that decisions regarding the listing of medicines are made by independent experts, at arm's length from government."

Meanwhile, Ms France said the family couldn't afford to wait any longer.

"It's really heartbreaking to think our bureaucratic process is holding back us access to a life-changing drug - this is what worries us most."

A protest is being organised at Parliament House in Canberra on February 7.

Vertex were contacted for a response but did not answer Fairfax Media's questions.