Australia finally gives cystic fibrosis patients access to Kalydeco

After months of wrangling, Australia has listed Vertex' cystic fibrosis (CF) therapy Kalydeco in the Pharmaceutical Benefits Scheme, finally giving patients access to the new drug.

Negotiations over pricing of Kalydeco (ivacaftor) have been dragging on since the drug was approved in Australia in July 2013, even though the Pharmaceutical Benefits Advisory Committee (PBAC) recommend it should be made available to patients with a form of CF caused by the G551D mutation.

The PBAC baulked at Kaydeco's price tag of A$300,000 a year per patient, however, holding up the drug's addition to the PBS. Now, the government has agreed a pay-for-performance deal with Vertex that should help the 250 eligible CF patients in Australia gain access to the drug but will also double the amount the Government spends annually on treatments for CF.

If the patient does not respond at all the cost of the drug should be repaid by the sponsor, while for partial responders only a portion of the total cost would be subsidised, according to panel recommendations published last year. The specific details of the pay-for-performance arrangement remain confidential.

Frustrated CF patients and their families who had mounted a social media campaign (#yestokalydeco) to vent their displeasure at the delay have taken to Twitter to celebrate the verdict.

Faye Upston tweeted: "ThAnK YoU!!!! For saying #YesToKalydeco, this decision will save hundreds of lives including my own."

"With this treatment many patients can experience an improved quality of life, with an improved lung function, weight and reductions in hospitalisation, including hospital in the home," said Australia's Department of Health in a statement.

It added that it had been able to fund the drug "because of the tireless work of the Cystic Fibrosis Association to develop the criteria to manage the use of Kalydeco in Australia."

"It is because of this work, the government was able to take a decision to fund this drug for patients to provide Australians in need with subsidised access to this important medicine."

Vertex has claimed that it has made Kalydeco available to the Australian healthcare service at the best rate in the world.

The company reported worldwide sales for the CF therapy of $127m in the third quarter of 2014, taking the year-to-date tally to around $340m, and the product is expected to gain momentum should the FDA follow its own advisory committee's advice and approve it for use in CF patients with another mutation called R117H.