Health Minister overturned decision by her department and reinstated Christian's elaprase

18/07/2013 01:26

Campaign success for sick boy

Rebecca Trigger, The West AustralianMay 28, 2013, 12:16 pm
Campaign success for sick boy

Michelle Dierkx and 5-year-old Christian. Picture: Steve Ferrier/The West Australian



The Federal Health Minister will review the eligibility criteria of the Life Saving Drugs Program after a little Perth boy's family spent 16 months campaigning for reform.

The family of Christian Dierkx, who suffers from a rare genetic condition, are relieved their son has been put back on the program but say its eligibility guidelines are discriminatory and need to be reformed.

Christian, 5, was accessing the drug Elaprase through the program, a Federally-funded initiative which is designed to supply extremely expensive medicines that are otherwise not available on the Pharmaceutical Benefits Scheme.

He was being treated for Hunter syndrome, also known as Mucopolysaccharidosisa (MPS), a degenerative condition where the body lacks an important enzyme and can lead to stiffness of the joints, aggressive behaviour, hyperactivity and intellectual impairment.

But under the scheme's guidelines, patients must demonstrate stabilisation or improvement of their condition to continue to receive the medication.

Christian's supply of Elaprase - which is shown to slow the debilitating effects of Hunter syndrome - was cut off in January last year after his condition was found to have deteriorated.

After a sustained campaign by his parents - which included countless letters, emails and calls to the Federal Health Minister - the Government late last week reversed its decision and has reinstated Christian's medication.

"We are relieved just to be able to put this whole issue behind us and focus on Christian's development, for him to continue to grow and be a happy and healthy boy," Christian's mother Michelle Dierkx said.

But she said he had sustained permanent damage during almost 16 months without the life-changing drug, including thickening of his features and restricted growth.

"I think Australia needs to have a good look at the way they have the guidelines set up around the way they administer this treatment to Hunter Syndrome sufferers."

His parents have always maintained undiagnosed hearing loss caused Christian to underperform on verbally-administered tests, making him appear to have intellectual impairment caused by his central nervous system becoming affected by his condition.

But they say even if there is central nervous system involvement in cases of Hunter Syndrome, Elaprase is still effective and to withhold treatment is discriminatory, because it treats someone with an intellectual impairment differently - deciding they are less eligible than someone without that disability for publically-funded treatment.

"I think the exclusion criteria should be taken out of the guidelines immediately," Mrs Dierkx said.

"For all the other MPS sufferers out there that have not been able to access this treatment, which I understand is about eight, because it's the only thing that is available to these patients, … if they are not able to access it will make their quality of life not very good."

A spokesman for Federal Health Minister Tanya Plibersek said the Minister made the decision to reinstate Christian's medication based on new and emerging evidence that treatment with Elaprase is likely to improve Christian's airways, lung function and range of movements of joints, and therefore the therapy may prolong his life.

The Minister also asked her department to commence a review of this emerging evidence, he said.