Trikafta recommended for limited PBS listing

The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended the listing of Trikafta (elexacaftor/tezacaftor/ivacaftor) for the treatment of cystic fibrosis patients aged 12 years and older who have one F508del mutation and one minimal function mutation in the CFTR gene (F/MF population).

However the PBAC said it could not yet make a recommendation about listing for the broader population of CF people (F508del homozygous and F508del heterozygous with a Residual or Gating mutation), as it was awaiting a response from sponsor Vertex on proposed eligibility parameters

Cystic Fibrosis Australia welcomed the partial PBAC recommendation as “a strong step in the right direction” but called for rapid progression to a full listing for all people over 12 with CF. The group said the Australian Cystic Fibrosis Data Registry (ACFDR) had provided ample data on eligibility (indexed by gene mutation) data to support a listing, and claimed that government and the manufacturer were “simply jockeying for their preferred price”.

ICS speeds recovery in COVID-19

Inhaled budesonide improves time to recovery by about three days in people with COVID-19 in the community who are at higher risk of complications.

A UK study, published in The Lancet, randomised 2,500 older adults (≥65 years or ≥50 years with comorbidities) with COVID-19 to either usual care, usual care plus budesonide or usual care plus other interventions.

It found time to first self-reported recovery was 11.8 days with budesonide versus 14.7 days with usual care alone (HR 1.21).

There was also a high probability of reducing COVID-19-related hospital admissions or deaths by an absolute difference of 2%.

“This trial has provided evidence of a safe and cheap community treatment for COVID-19 that reduces symptom burden and enhances sustained recovery over 28 days, with a high probability of also reducing the need for hospital admission, albeit just below the prespecified superiority threshold in the primary analysis population,” it said.

A Comment article said that on the basis of the data, it seemed reasonable to consider inhaled corticosteroid use in early COVID-19 in patients at risk of complications.

However, the authors noted the study was conducted when vaccination against COVID-19 was uncommon.

TSANZ recommendations for home ventilation in children

The TSANZ and Australasian Sleep Association have issued a joint position paper on long-term invasive and noninvasive ventilatory support for children at home.

The recommendations cover discharge planning, tracheostomy, NIV, BPAP and CPAP, psychological support for carers and family members, translation to adult services, and support for children with life-limiting conditions receiving palliative care.

The position paper also covers equipment requirements and risk management considerations.

The Working Group, chaired by Dr Andrew Tai from the Women’s and Children’s Hospital Adelaide, acknowledged the significant burden of care to parents and families caring for children requiring long-term ventilation and the need for appropriate support and respite.

They also noted disparities in the provision and timing of funding.

“Funding packages for those with high-dependency non-invasive ventilatory support should be based on those for children on tracheostomy ventilation,” they said.

“The working group noted that this group often receive less funding due to not having a tracheostomy, even when their care needs are equivalent.”

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