Vertex: Conditions of PBAC recommendation may limit Australians who could benefit from KALYDECO

The Pharmaceutical Benefits Advisory Committee (PBAC) has again recommended KALYDECO™ (ivacaftor) for listing on the Pharmaceutical Benefits Scheme (PBS). However, Vertex is deeply disappointed in the conditions the PBAC has attached to the recommendation. These conditions fail to reflect the substantial clinical benefits recognised by the PBAC, and would result in limiting the number of eligible Australians who would be able to receive KALYDECO.

“The conditions of the PBAC recommendation 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. “We believe that physicians are in the best position to make treatment decisions based on what is most relevant to individual cystic fibrosis patients.”

Vertex is particularly concerned by the PBAC conditions that exclude the sickest patients from being treated (those with a baseline FEV1, a measure of lung function, of less than 40 percent predicted) and the discontinuation criteria that would result in patients experiencing benefits being taken off treatment. These conditions could disqualify approximately half of current G551D patients in Australia from receiving KALYDECO.

The submission presented by Vertex to the PBAC in March is consistent with what was offered to and accepted by governments throughout the world. Today’s PBAC recommendation 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.

“While we are unable to agree to all the conditions proposed by the PBAC, we remain committed to reaching an agreement with the Australian government that is in the best interest of all patients and similar to the agreements reached around the world,” said Mr. Bedson. “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.”

“We hope the government will reject the unreasonable conditions recommended by the PBAC and list KALYDECO so all eligible Australians can benefit.”

KALYDECO is currently available to all G551D patients ages 6 and older in England, Scotland, Northern Ireland, Wales, the Republic of Ireland, France, Germany, Italy, the Netherlands, Austria, Denmark, Sweden, Norway, Greece and the United States of America.

KALYDECO is the first and only medicine to treat the underlying cause of cystic fibrosis in people ages 6 and older with the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.