Vertex’ cystic fibrosis (CF) drug treatment Kalydeco (ivacaftor) should be listed on Australia’s Pharmaceutical Benefits Scheme (PBS), but if patients do not improve on the drug they should be taken off it and the company should make full refund to the government, experts have recommended.

The recommendation, by the Pharmaceutical Benefits Advisory Committee (PBAC), has been condemned by Vertex and also by patient groups. The drug costs A$300,000 per year for each of Australia’s approximately 200 CF patients.

Vertex says it is unable to agree to all the conditions which PBAC has attached to its recommendation and has urged the government to reject them. The conditions being sought “will prevent patients who could benefit from Kalydeco from being able to get it, or from remaining on treatment even when demonstrating improvement,” said Simon Bedson, senior vice president and international general manager at Vertex. 

The Committee’s decision “ demonstrates the difficulties of assessing medicines for people with rare diseases who generally have few or no treatment options and shows a need for more flexibility and a more appropriate process,” he commented.

“We believe that physicians are in the best position to make treatment decisions based on what is most relevant to individual CF patients,” Mr Bedson added.

The firm points out that the submission for Kalydeco whch it presented to the PBAC in March is consistent with what was offered to and accepted by governments throughout the world.

“Unlike any other country, Australia is seeking to impose strict eligibility and discontinuation criteria to limit the number of patients who could benefit or are showing benefit from this medicine,” said Mr Bedson.

The recommendation was also criticised by advocacy group Cystic Fibrosis Australia, whose chief executive, Michelle Skinner, pointed out that PBAC had initially recommended Kalydeco for PBS listing almost six months ago. She urged the Minister of Health, Peter Hutton, to intervene and “assist PBAC and Vertex in reaching an amicable agreement.”

PBAC has made a similar “pay for performance” recommendation relating to the listing of Alexion’s Soliris (eculizumab) for the treatment of atypical haemolytic uremic syndrome (aHUS), which affects around 60 Australians.

And according to local reports, the “pay for performance” model for listing has only been used once before by Australia’s PBS.