Ivacaftor Stakeholder Meeting
11 August 2014
A stakeholder meeting was held on 31 July 2014 to discuss the Pharmaceutical Benefits
Scheme (PBS) listing of ivacaftor (Kalydeco) for the treatment of cystic fibrosis
in patients aged six years and older who have a G551D mutation in the CFTR (cystic
fibrosis transmembrane conductance regulator) gene.
The meeting’s outcome statement reflects the pay for performance framework recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) in March 2014, under which all eligible patients could be treated with PBS-subsidised ivacaftor but the price paid by the Government varies depending on the magnitude of the benefit gained by patients, in recognition that not all patients respond equally to treatment. Should the pay for performance framework not be successfully negotiated with the sponsor of the medicine, the criteria for response could be used as stopping rules in a manner similar to many other PBS listings.