Trikafta’s second PBAC deferral disappoints CF advocates

Cystic fibrosis

By Michael Woodhead

22 Jun 2021

A decision on PBS listing for Trikafta (elexacaftor/tezacaftor/ivacaftor) for treatment of cystic fibrosis has been deferred for a second time by the Pharmaceutical Benefits Advisory Committee (PBAC).

At a May intracycle meeting the PBAC again deferred on making a recommendation based on revised submission from manufacturer Vertex for Trikafta to be listed for the treatment of CF patients aged 12 years and older who have at least one F508del mutation in the CFTR gene.

The PBAC said the sponsor’s revised proposal did not adequately address the parameters outlined at the previous PBAC meeting. At its March 2021 meeting the PBAC had concluded that Trikafta would provide a significant benefit for some patients, “in particular, patients who are homozygous for the F508del mutation in the CFTR gene and patients who are heterozygous for the F508del mutation in the CFTR gene with a minimal function mutation

However it said there was uncertainty about the safety and effectiveness of Trikafta beyond 48 weeks and suggested this could be managed with a Managed Access Program (MAP), as with other CFTR modulators.

In its response to the revised proposal the PBAC said it had agreed to further increase the budget for the listing by 80%, but the submission still did not meet the required pricing and financial estimates.

Therefore, it deferred completion of its consideration to allow the sponsor to provide further information.

In its latest communique, advocacy group Cystic Fibrosis Australia said it had been supported by Professor Peter Wark in a meeting with the PBAC and industry representatives in Canberra to try resolve the key issues hampering a positive recommendation for Trikafta, namely long term data; patient numbers and the details of Managed Access Program (MAP).

“Professor Peter Wark spoke about data and research comparing Trikafta to other modulators and he also shared information on international health outcomes. It was clear to all that Trikafta will change lives for the better and extend lives so that people with CF can have a fulfilling future,” it said.

Cystic Fibrosis Australia (CFA), said it would commit to use Australian Cystic Fibrosis Data Registry (ACFDR) to provide regular information on patient numbers and Trikafta onboarding and discontinuations.

Patient representatives also asked for immediate compassionate access once a PBAC positive recommendation was given “and reminded the group that even delays of ‘days’ not ‘months’ negatively impacted people’s health and life expectancy.

CFA said it hoped the sponsore, Vertex would resubmit a PBS listing application to the July PBAC meeting “and to be creative and innovative when considering MAP criteria. Vertex needs to make this work. We will wait and see if our pleas are heard and acknowledged.”

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