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Access to cystic fibrosis medication expanded


Matt Woodley


22/07/2019 2:53:05 PM

Children aged 12–24 months with a rare form of the genetic disorder will soon be eligible to receive subsidised medication, saving their families up to $300,000 per year.

The Pharmaceutical Benefits Scheme website.
A number of new listings have been added to the Pharmaceutical Benefits Scheme.

Ivacaftor (trade name Kalydeco), a medication specifically targeted to treat people with a G551D mutation or other class III gating mutation in the CFTR gene, is designed to lessen the viscosity of mucus in the lungs in order to help patients breathe more freely.
 
It was previously only available through the Pharmaceutical Benefits Scheme (PBS) for patients aged two years and over, but the new listing will help treatment begin as early as possible. The medication will cost up to $40.30 per script, or $6.50 with a concession card.
 
The new listing followed an independent assessment by the Pharmaceutical Benefits Advisory Committee (PBAC), which also recommended the addition or expansion of a number of other medications.
 
While noting that the efficacy data in the 12–24 months age group was limited, the PBAC said it was difficult to obtain clinical data within this setting and considered a benefit in earlier treatment was biologically plausible based on the overall evidence.
 
A new brand of the antibiotic flucloxacillin for the treatment of serious staphylococcal infection and osteomyelitis has also been added. More than 525,000 patients accessed the previously available brand of antibiotic for this condition.
 
Other changes to the PBS include: 

  • A new strength of benzathine benzylpenicillin (trade name Bicillin L-A) for patients requiring treatment of infections due to penicillin-sensitive micro-organisms
    • The new listing is 0.6 million units (517 mg) in 1.17 mL pre-filled syringe, at an equivalent price per unit to the currently listed strength of 1.2 million units (900 mg) in 2.3 mL pre-filled syringe
    • More than 4400 patients are expected to access this listing, who would otherwise pay at least $150 per script.
  • Patients with low levels of testosterone in their body will be able to access a new strength of testosterone (trade name Testaven)
    • Without a PBS subsidy, more than 10,500 patients would otherwise pay around $1400 per year for this treatment.



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