Why Pompe Patients Down Under are Down and Out

 
 
 
 

 

Are You Kidding Me?!?! The Federal Government in Australia is crazy if they don’t think ERT works!

From the Herald Sun
July 22, 2010
By Sue Dunlevy

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.

Really?

 

10 comments

  1. That’s the same problem with the New Zealand government! I wonder if all of a sudden things would change if a politician’s 20something year old offspring fell ill with Pompe. Bet they would do everything in their power to make sure they were able to get ERT! It really sucks that one human being can sit up there in parliament and say “hey too bad for you but we’ve got better things to spend our money on”! You know, like wars and keeping criminals in jail, cool stuff like that!

    1. I hear ya sister! That is why it is up to us to inform and educate the policy makers.

      We can be heard! We will be heard!

  2. Unbelievable, not not surprising. It’s up to us to educate them. It’s a simple and understandable fact that early trials were not done with adults, so there is more limited trial data available. However, all it takes is someone willing enough to look at the information available to see that it is well substantiated that it does indeed work. If it didn’t, it wouldn’t be in use _all_over_the_world_!!

  3. Quite dissapointing, but unfortunately we may be heading down the same path here in the US. Let’s hope for the best for all the WW pompe patients!

  4. Good God… Check this out:
    “The Government funds the medicine for babies born with the disease because they will die within 12-14 months without the enzyme.”

    AND THEN:
    “The Government said the drug company had failed to produce evidence the medicine would save lives.”

    I call that PPI – Politician’s Purposeful Iliteracy.

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