Two boys, one horrific disease, different wonder drug funding

A YOUNG boy dying of a rare and horrifying genetic disease faces losing access to a potentially ground-breaking drug on Monday, even though another boy with the same illness — who is a patient at the same Melbourne hospital — gets it for free.

EXCLUSIVE: A YOUNG boy dying of a rare and horrifying genetic disease faces losing access to a potentially ground-breaking drug even though another boy with the same illness gets it for free.

The Royal Children’s Hospital, where both boys are patients, is ­demanding that the federal government address the ethical and financial ­dilemma posed by the inequity in how the two would be treated.

The experimental medication, which is worth $900,000 a year, has yet to be approved for full use in Australia. The RCH is treating both boys with the drug, which eases patients’ suffering and prolongs life.

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One boy will continue to get the drug for free under a private deal.

But a temporary agreement with the medication’s manufacturer to ­provide it to the other boy is due to ­expire in three days.

This prompted the RCH to write to federal Health Minister Greg Hunt over its concerns at the arrangement behind the differing treatment.

In a statement to the Herald Sun on Thursday, the RCH said: “The Royal Children’s Hospital is currently treating two patients to alleviate the symptoms of a particular rare genetic condition. The cost of one patient’s treatment is funded under a privately negotiated agreement between two other parties. The cost of the other ­patient’s treatment is currently ­unfunded from July 16.

“The RCH has made representations to government authorities ­regarding the issue of equity of access to high-cost drugs.

“The RCH position is that all children are entitled to equal access to treatment, under the same conditions and for the same time frame. The RCH is strongly committed to equity for all children and this forms the basis of all RCH considerations.”

The Royal Children's Hospital is ­demanding that the federal government address the ethical and financial ­dilemma posed by the inequity in how the two boys would be treated. Picture: Fiona Hamilton

The two boys are believed to be the only children in Australia who would benefit from the medication.

Mr Hunt’s office was scrambling to secure new measures guaranteeing continuing access to the medication for the boy who now stands to lose it.

Mr Hunt’s spokesman said: “The Minister is deeply engaged on supporting patients and their families battling this horrible disease.

“We will work with the pharmaceutical company and the Royal Children’s Hospital to ensure continuous access for the family, while an application is being considered for listing on the Pharmaceutical Benefits Scheme or the Life Saving Drugs Program.”

The family of one boy moved overseas last year so he could get the drug during a clinical trial. When the treatment significantly improved his condition, the manufacturer agreed to continue to provide the family with the drug, as long as they remained overseas.

The hospital has written to federal Health Minister Greg Hunt over its concerns at the arrangement behind the differing treatment. Picture: AAP/Mick Tsikas

The Herald Sun understands that, when the family wanted to return to Melbourne, arrangements were brokered that would allow the boy to continue to get the drug in Australia. He was referred to the RCH.

However, senior RCH staff shocked by the arrangement said they would treat the boy only if the drug were made available to all patients with the terminal illness — including a child then being treated at the RCH.

The Herald Sun is not suggesting any wrongdoing by the families, neither of whom are fully aware of the differing access to the drug.

The Herald Sun understands the manufacturer eventually agreed to provide the drug to the second boy free for six months. But the hospital or his family would then need to pay $30,000-plus a fortnight.

The Pharmaceutical Benefits Advisory Committee recently began assessing whether the drug should be subsidised on the PBS or referred to the Life Saving Drugs Program, under which it could be provided free of charge to all patients.

The Herald Sun has chosen not to identify the condition so as to protect the families’ identities.

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