One mother's fight for 'life-changing' narcolepsy drug

 abc.net.au  6/20/2017 7:19:18 PM   medical reporter Sophie Scott and the National Reporting Team's Meredith Griffiths

Posted June 21, 2017 05:19:18

Queensland mother Melissa Jose wanted to do whatever she could to help her son Lucas.

Key points:

  • Mum fought for access to breakthrough drug
  • Xyrem costs up to $1,800 a month
  • Patient groups hope "life-changing drug' will soon be cheaper

He has the debilitating sleep disorder narcolepsy, which means he sleeps 20 hours a day.

"He was sleeping his life away and he was only 18. He had his whole future ahead of him," she said.

She read about a drug available overseas called Xyrem which had been shown to work to significantly help patients with narcolepsy.

But Melissa faced major hurdles getting the medication for him.

She took on the medical system, successfully lobbying the Therapeutic Goods Administration (TGA), to reschedule Xyrem to be a controlled drug.

That meant it could be used but only on the order of an authorised medical practitioner.

Then she had to lobby to get the drug distributor UCB to import Xyrem into Australia.

"The drug company had no intention of importing it because they don't believe there is a market for it in Australia because narcolepsy affects so few Australians," she said.

"You're talking 12,000 people maximum in Australia have narcolepsy and that's not a huge figure for a company that's about making profits."

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She convinced the distributor to bring the drug to Australia.

Melissa and her extended family are shelling out $1,600 a month to pay for the drug.

But the effect of the medication on Lucas has been profound.

"Now he can drive and go to university — which he couldn't do before this drug — so the investment is paying off and will make him a more full-functioning member of society," she said.

Hope drug will be cheaper

Xyrem is currently classified as a controlled drug by the TGA.

It costs $600 a bottle, which can add up to $1,800 a month, depending on the dose.

What is Narcolepsy?

  • Narcolepsy is a neurological disorder characterised by excessive daytime sleepiness.
  • Many also have a symptom called cataplexy where strong emotions make them become paralysed.
  • The Australasian Sleep Association says narcolepsy is difficult to treat. Some patients remain excessively sleepy even on the maximum dose of approved medications. Others react badly to the anti-depressants used to control their cataplexy.
  • As such the Sleep Association recommends the use of the sodium oxybate, marketed as Xyrem, which has been approved as a treatment for narcolepsy in the United States, Canada and Europe since 2005.

A contentious solution

  • Sodium oxybate is a contentious substance.
  • It is also known as GHB and in the 1990s, was designated as a prohibited substances by the TGA because of concerns it was being abused by bodybuilders, nightclubbers and is a potential date rape drug.
  • As it began to be more widely used to treat narcolepsy overseas, in 2014 the TGA rescheduled sodium oxybate to be a controlled drug, meaning it could be used but only on the order of an authorised medical practitioner.

At the moment, patients have to pay for it themselves, but if Xyrem does become a registered prescription medication, people could claim some of the expense back on private health insurance.

Narcolepsy Australia said UCB told them it did not want to apply to have Xyrem registered as a prescription medication because the application process costs as much as $400,000.

But there is new hope — From July 1, 2017 the TGA will change the way it defines a rare condition.

Previously, conditions had to affect fewer than 2,000 Australians to be considered "rare".

But from July 1, 2017, the definition will be broadened so a rare condition will be classified as an illness that affects 12,000 people.

That would include narcolepsy, which is thought to affect 12,000 Australians.

Xyrem could then fall under the TGA's Orphan Drug Program, meaning the drug company could register the medication at no cost.

"We're hoping that UCB Pharmaceuticals who distribute Xyrem in Australia will actually lodge an application to register it as a prescription medication in Australia," she said.

"That's the first step that we need to happen to make Xyrem more affordable and available for everyone."

Being registered would allow Narcolepsy Australia to apply to have Xyrem publicly funded on the Pharmaceutical Benefit Scheme.

Topics: sleep-disorders, diseases-and-disorders, sleep, health, australia, qld

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