Blood disorder drug gets funding

PATIENTS who have the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH) will benefit from a multimillion-dollar drug commitment in this month’s Federal Budget.

Eculizumab (Soliris) has been added to the Life Saving Drugs Program at a cost of $135.9 million. This will provide five years’ therapy to around 50 patients with PNH, a disease that causes haemolysis and thrombosis.

The drug is administered intravenously every fortnight, with the annual cost of treatment running to almost $500,000 per patient.

“It’s expensive, yes, but it is definitely not the most expensive drug in the world… Unfortunately that was a misnomer that crept into the debate,” said Jenny Sturrock, a Melbourne patient who campaigned for access to the drug. 

Ms Sturrock, aged 29, said it had extended her prognosis from five years to a “normal life expectancy”.

Around 30 countries now provide