Rachel Carter Chief of staff at Australian Doctor .

children — less than 10 admissions a year . The reason for this is a medical wonder — but one that requires vast quantities of money to pay for it .

Professor Hiran Selvadurai .

On a per-patient basis , Trikafta is one of the most expensive drugs ever listed on the PBS .

Astonishing changes

Back in March 2021 , the PBAC

went through the cost – benefit number crunching and , after twice deferring the decision ,

impact , the recommendation had to go before National Cabinet , but it was signed off . As a result of the listing — which was last year expanded to include children aged 6-12 —

the costs , amid accusations that the US company responsible for bringing it to market , Vertex Pharmaceuticals , is exploiting its discovery .

In October last year , the

specific cash figure is usually confidential .

But according to NICE , what the NHS was being asked to pay was “ substantially above ” $ 59,000 for each

Selvadurai , the head of the respiratory department at the Children ’ s Hospital at Westmead in Sydney , their

eventually concluded that the combination cystic fibrosis transmembrane regulator ( CFTR ) modulator therapy agent Trikafta ( elexacaftor /

148 of the 210 patients attending Professor Selvadurai ’ s clinic are on Trikafta .

He says the changes have astonished him .

UK health economists , consultants , GPs and pharmacologists who make up the National Institute for Health and Care Excellence ( NICE ) were doing

quality-adjusted life year generated by the treatment , where each year is equivalent to one perfect year of health .

As a rough guide , the Aus-

The ‘ before ’ would include

lives were being broken by

tezacaftor / iva-

“ With many

their own cost – benefit sums .

tralian Federal Government

a dozen or so children lying in beds with severe pulmonary exacerbations , coughing up bacteria-ridden mucus , almost drowning in their own sputum .

Some would become

cystic fibrosis .

“ When you walked into the room and told families that their child has cystic fibrosis , you were looking at their faces and you knew you

caftor ) should be listed on the PBS .

Initially recommended for those aged 12

‘ Trikafta is an enormous game changer .’

of these kids , when the exacerbations came they had to spend the next two weeks in

And they did not work out in the same way .

A final decision has yet to be made , but the committee ’ s draft recommendation was that Trikafta was not worth

would also be very reluctant to pay above this figure for health interventions , although there is no official threshold for PBS listing .

The NICE conclusion was

hypoxic and need the oxygen

were shattering their dreams

or older in April

hospital

with

the money .

that Trikafta was a waste of

masks to prevent brain injury . And all of them would need a long line of IV antibiotics , along

and aspirations . “ You were talking to them with a sledgehammer and you

2022 , the taxpayer cost was some $ 475 million to treat an estimated 1900 patients . This

an IV drip . “ It was exceptionally emotionally draining , but Tri-

Waste of resources ?

As with any taxpayer sub-

precious health resources . Parents of children with the disease described the conclu-

with a regimen of brutal phys-

would see their hearts break .”

was not a lifetime cost but the

kafta has been an enormous

sidy decision , the price at

sion as “ unthinkable ”.

iotherapy and a carefully con-

But there is also the ‘ after ’.

annual cost — $ 250,000 per

game changer .”

which drug companies offer

As here in Australia , at the

trolled diet .

And this is where those beds

patient per year .

But there has been a fierce

to sell their product var-

sight of young suffering , the

In the words of Professor

are rarely occupied by these

Given its budgetary

debate around the world about

ies with the buyer , and the

British media usually takes aim

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