The availability of Orkambi® (containing lumacaftor and ivacaftor) through the Pharmaceutical Benefits Scheme for patients with cystic fibrosis

Page last updated: 04 June 2018

Orkambi® (containing lumacaftor with ivacaftor) is currently not available on the Pharmaceutical Benefits Scheme (PBS) for the treatment of any condition, including cystic fibrosis (CF).

Under legislation passed by the Australian Parliament a new medicine cannot be listed on the PBS unless the Pharmaceutical Benefits Advisory Committee (PBAC) – an independent, expert advisory body – makes a recommendation in favour of listing.

When considering a medicine proposed for listing, the PBAC is required by that legislation to give consideration to the effectiveness and cost of the medicine, including by comparing the effectiveness and cost with that of alternative treatments.

The Australian Government and the Minister for Health do not interfere with the decision making process of the PBAC.

The PBAC has assessed this medicine in the same way it does all submissions for listing medicines on the PBS. At its July 2017 meeting, the PBAC was unable to recommend a third submission to list Orkambi for the treatment of certain CF patients.

The PBAC agreed that there is a clinical place for Orkambi at a price commensurate with its clinical benefits. However, the evidence presented did not support the clinical claims made by the sponsor of the medicine, Vertex Pharmaceuticals (Vertex), that Orkambi slows the rate of decline in lung function. Further, the price requested by Vertex was disproportionate to the benefit demonstrated for the medicine.

More information about the July 2017 PBAC consideration of Orkambi is available on the PBS website.

Vertex has now made another submission to the PBAC to list Orkambi on the PBS for the treatment of certain CF patients aged 12 years and over. Vertex has also made a new submission to the PBAC to list Orkambi on the PBS for the treatment of certain CF patients aged between 6 years and 11 years. These two submissions will be considered by the Committee at its July 2018 meeting. More information is available on the PBS website.

The PBAC process allows interested parties to provide comments on, and input to, submissions being considered by the PBAC. The PBS website link above includes a PBAC online form that can be used to provide this input. The closing date for the receipt of input for the July 2018 meeting is 6 June 2018.

The PBAC outcomes for the July 2018 meeting will be made public on the PBS website on 17 August 2018.

The Australian Government has a policy to list every medicine on the PBS following a recommendation from the PBAC to do so. Should Orkambi be recommended for listing at the July 2018 meeting it will be listed on the PBS.

Meanwhile, there are other options for gaining access to Orkambi. The patient’s treating doctor may wish to contact Vertex to find out whether Orkambi could be supplied at a reduced cost through expanded access or compassionate access programs. Decisions regarding such programs are at the sole discretion of the sponsor.

The contact details for Vertex can be found on its website.

Subsidising clinical treatments is not the responsibility of the Australian Government alone. The Australian Government provides significant funding to the states and territories to assist with the costs of operating public hospitals.

The treating doctor may wish to consider making an application, on behalf of their patient, to the drug and therapeutics committee of a local public hospital with the cost of treatment.