PBAC green light for cystic fibrosis therapy welcomed, yet reimbursement delays persist

Pharma News: The Pharmaceutical Benefits Advisory Committee (PBAC) has given a green light to Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for children with cystic fibrosis (CF) ages 2-5 years who have at least one F508del mutation in the CFTR gene.

Data from a 24-week open-label, two-part phase 3 study evaluated the efficacy and safety of Trikafta in this age group. Results showed patients experienced clinically meaningful reductions in sweat chloride concentration and lung clearance index.

Jo Armstrong CEO Cystic Fibrosis Australia, said “This is fantastic news because we know that there is a significant benefit to commencing therapies early to prevent irreparable damage.

“The next hurdle is knowing when Trikafta will be subsidised by the Pharmaceutical Benefits Scheme (PBS) so that it can be accessed by those in the 2 to 5-year age group. We hope that there is no delay and we call on all stakeholders to please accelerate this listing, do not delay access for these little ones.”

“With this recommendation approximately 130 children living with cystic fibrosis ages 2–5 years would have access to a medicine that treats the underlying cause of their disease for the first time. We are committed to working with the Department of Health and Ageing to ensure Trikafta can be made available to Australian children as quickly as possible,” Vertex said in a statement.

Ms Armstrong also drew attention to the ongoing delays in the PBS listing of Kalydeco, saying “It felt like a Christmas miracle when we had the news of that announcement in December 2023 for children 4 months and older, including additional gene mutations.

“Now more than four months later people are still waiting to access it. The benefit of recommending Kalydeco has not (yet) been realised because the people who need it cannot access it! This must change. We need this listed on the PBS urgently. We are continuing to liaise with all stakeholders in the hope of minimising the delays between PBAC recommendations and PBS listings. Currently, it is taking too long.”

The Department of Health’s latest report on the PBS processes showed a slower average time from PBAC minutes to PBS listing between the current and previous periods, despite ongoing reforms.

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