Are You Kidding Me?!?! The Federal Government in Australia is crazy if they don’t think ERT works!
The Federal Government has twice refused to subsidise Myozyme, which treats the deadly and extremely rare Pompe disease, under the Government’s life-saving drugs program.
Drug company Genzyme, which has spent $36 million supplying the drug free of charge to patients for three years, said it could no longer afford to provide it under a charity access program if a third attempt to gain a subsidy fails in November. “I can’t sustain the charitable funding beyond this year,” Genzyme managing director Dan Brown said.
A senior member of the medical profession described the threat to cease the charitable funding as “blackmail” by the drug company.
The Government said the drug company had failed to produce evidence the medicine would save lives.
Patients with Pompe disease are missing an enzyme that breaks down glucose in the body. Fats accumulate in their muscles and they usually die of cardiac or respiratory failure.
Myozyme, delivered by infusion every two weeks, replaces these missing enzymes.
The Government funds the medicine for babies born with the disease because they will die within 12-14 months without the enzyme.
But children and adults who develop the disease later in life experience a less rapid form – the progression can be so slow in some people that they will outlive it.
Some also have limited benefits from the drug and only a few people achieve remission or dramatic benefit.
The Government already funds three high-cost medicines to treat other enzyme diseases under the life-saving drugs program. But in May it ruled such drugs must substantially expand a patient’s life.