Children with cystic fibrosis to benefit from latest PBAC recommendation

Pharma News: The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended the extension of Vertex’s Orkambi (lumacaftor/ivacaftor) to patients aged between 1 year and less than 2 years. This announcement brings a ray of hope to the cystic fibrosis community, heralding a pivotal opportunity for young children to access a treatment that holds the potential to improve their quality of life.

Although the PBAC the challenges in securing efficacy data from paediatric patients, it concluded that the assertion of enhanced efficacy over best supportive care for patients aged 1 to less than 2 years was not only biologically plausible but also likely to to be beneficial. Notably, the PBAC underscored that Orkambi is expected to be cost-effective, provided the unit price remains aligned with the current Pharmaceutical Benefits Scheme (PBS) listing for patients aged 2 years and older. The PBAC advised that the “current risk sharing arrangement financial caps should be increased to accommodate patients commencing treatment earlier.”

Jo Armstrong, the CEO of Cystic Fibrosis Australia, remarked on this development as a pivotal milestone, underlining the urgency surrounding these changes. She said “While this development marks an important milestone, we recognise the urgency that surrounds these changes. We call upon both the Australian Government Department of Health and Aged Care and Vertex Pharmaceuticals to work closely and swiftly to ensure that this access is made available without delay. Timely access to treatments is crucial in offering the best possible chances for these children to thrive.

“We extend our deepest gratitude to the medical community, patient advocates, and all those who have played a role in advocating for this expansion of access. Our collective efforts have brought us to this point, and we remain steadfast in our commitment to pursuing further advancements and improvements for the cystic fibrosis community.”

In response to the PBAC recommendation, Vertex Pharmaceuticals expressed their enthusiasm, labelling it a vital initial step toward achieving reimbursed access in Australia. “This is an important first step to achieving reimbursed access in Australia. With this recommendation, approximately 35 children in Australia with two copies of the F508del mutation could have access to a medicine to treat the underlying cause of their disease. We are committed to working with the Department of Health and Ageing to ensure Orkambi can be made available to these Australian children as quickly as possible,” the company said in a statement.

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