50 NEWS REVIEW
50 NEWS REVIEW
19 APRIL 2024 ausdoc . com . au
The wonder drug that costs $ 250,000 a year
CFTR modulators , says Professor Selvadurai .
“ That ’ s the reason why we are very excited about what it implies for the longterm future .
“ Historically , when I was training as a fellow , the prognosis of children with cystic fibrosis was around 30 years … and now there are some good modelling studies that show children born now who access CFTR modulators will lead a normal life span .”
He points to one study — carried out by Vertex and the department of respiratory medicine at Glasgow Royal Infirmary in Scotland — that estimated the survival and lifetime clinical benefits of Trikafta versus other CFTR modulator combinations .
It found that young people aged 12-17 who were treated with the drug could on
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This transformation , Professor Selvadurai stresses , carries complexities .
“ I tell you , some of our teenagers can be a little mischievous with their other therapies [ but ] their adherence with the modulators is generally outstanding — they ’ re not dropping Trikafta , because they ’ ve figured out
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“ But they ’ ve also figured out they can drop various other stuff , including physiotherapy — God help us !”
But clinicians have also needed to adapt how they manage children because they are preparing them for a full , not a partial , life .
“ Previously , we would encourage a high-fat diet and high nutrition , for example , because the more the better — and now we ’ re looking at the
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‘ This drug gives them back their childhood .’
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quality of nutrition that we ’ re giving them .
“ We know there is a risk of cardiovascular disease , because they are going to be living to 70 or 80 , and we don ’ t want to be giving them that .”
Access for all ?
There is still a battle for the children who cannot access the therapy .
For some , it simply will not work on genetic grounds .
As made clear above ,
Trikafta only works for those with the most common delta F508 mutation .
This means 18 of Professor Selvadurai ’ s patients do not benefit .
For the others , age is the barrier . Trikafta is currently only available in Australia for children older than six .
The TGA has recently approved the use of the drug for children aged 2-5 — and this month it is back on the PBAC meeting agenda for whether taxpayers should fund it .
There are 313 children with cystic fibrosis in this age group , according to the Australian Cystic Fibrosis Data Registry , and Professor Selvadurai says 90 % would be eligible for Trikafta .
But a PBS listing could still take around six months .
“ The difficulty is , what kind of outcome can you measure in the 2-5 age group that is robust and objective enough to convince the powers that be to prescribe it ?” says Professor Selvadurai .
“ We can ’ t reliably measure lung function in that age group … We are pretty much stuck with nutrition and sweat tests .”
He explains that , clinically , children of this age are often healthy — they have not been hit by the “ vagaries of cystic fibrosis yet , but there is a need to futureproof against inflammation that could lead to poorer longer-term outcomes ”.
“ Given the opportunity , we would like to get in there earlier so that we prevent inflammation , which we presume will reduce the chances of developing longterm scarring of their lungs .”
He also believes that to take the pathway of NICE and recommend against the drugs would be short-sighted .
“ You are risking not only individuals and their life span , quality of life , but also from an NHS point of view … it will end up costing them much more .
“ Various modelling has shown it will end up costing more … in terms of service delivery , access to medical , allied health and nursing care .
“ There ’ s a lot of data to support that — as ridiculously expensive as it seems to us — this is actually cost-effective .”
He adds Trikafta is a treatment that respiratory physicians have dreamt of “ for years and years and years ”.
“ It ’ s amazing to have this access to something that is tangibly better for the children , to give them hope . This drug is giving them back their childhood , their future .
“ It ’ s enormously rewarding . It ’ s the highlight of my career so far .”