Kemal Atlay THE potent cholesterol-busting drug inclisiran is now subsidised for adults with hypercholesterolaemia who fail to respond to or tolerate statins and ezetimibe .

Inclisiran ( Leqvio ) — a small interfering RNA therapy — was added to the PBS this month as a third-line alternative to proprotein convertase subtilisin / kexin type 9 ( PCSK9 ) inhibitors in patients with non-familial hypercholesterolaemia and familial heterozygous hypercholesterolaemia .

The long-acting drug is delivered via subcutaneous injection every six months — except for the first two doses , which are delivered three months apart .

GP academic Associate Professor Ralph Audehm says the ‘ setand-forget ’ regimen will benefit patients with treatment inertia and adherence problems .

The PBS listing will save patients around $ 1800 per dose .

A full authority PBS listing is available for patients without

previous PSCK9 inhibitor therapy who meet strict clinical criteria , including previous treatment with ezetimibe and a statin , and where prescribed by a specialist physician or in consultation with one .

The authority ( streamlined ) listing covers patients who have previously received PBS-funded PCSK9 inhibitor treatment and those continuing inclisiran therapy .

However , patients cannot receive inclisiran and PCSK9 inhibitor therapy concomitantly , the PBS says .

Professor Audehm says the PBS criteria are strict but they are a sensible “ starting point ”.

No dose adjustment is needed for patients with mild or moderate hepatic impairment , but the impact on those with severe hepatic impairment has not been studied , according to the product information .

The drug is not recommended for patients with end-stage renal disease . Professor Audehm has received funds for consultancy and advisory boards from Novartis .

Associate Professor Ralph Audehm .

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It would also require a lumbar puncture , bone marrow aspiration , and insertion of a portacath to enable IV introduction of medication .

A paediatric oncologist said the girl had genetically-based developmental and epileptic encephalopathy plus level 3 autism spectrum disorder .

She estimated the treatment regimen would involve 58 general anaesthetics to allow doctors to perform the procedures .

The girl ’ s paediatrician , who had treated her since the age of 18 months , said doctors might have to “ shackle ” the girl to administer treatment .

He suggested palliative chemotherapy to prolong her life by 3-4 months .

“[ She ] would not cope with the pain and consequences of intensive chemotherapy or [ bone marrow ] transplantation ,” he told the court .

“ I therefore support and agree with the mother ’ s decision that it is not in [ her daughter ’ s ] best interests to attempt curative chemotherapy .”

The girl ’ s oncologist acknowledged that treatment was more difficult because of the girl ’ s disabilities , but “ that doesn ’ t mean you don ’ t try ”.

In making his decision , Justice Elkaim said the girl ’ s life was not “ entirely without joy ”, as evidenced by her pleasure in attending her special needs school , and chemotherapy would “ bring her back to her previous life ”.

In conclusion , he had “ little alternative ” but to approve the treatment .

“ Not making the orders is effectively a death sentence ,” he said .