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Breakthrough drug approved in Australia – but PBS listing still needed

Publish Date: 9/2/2017

Deborah Sims fantasises about the day when her haematologist can write her a script for venetoclax and she can stroll downstairs to have the life-saving drug dispensed at the onsite pharmacy.

It will be a far cry from her current situation. Ten return trips to London in 14 months, including a seven month stint living there,  at a personal cost of more than $700,000 in expenses and lost income  – to pick up this very same drug to bring home in her hand luggage.

The breakthrough cancer drug which has the power to ‘melt away’ advanced forms of chronic lymphocytic leukaemia (CLL) has recently been approved by the Therapeutic Goods Administration (TGA) for use in Australia.

It is a significant forward step for Australians like Deborah, who went from being terminally ill to being in complete molecular remission within 12 months of accessing venetoclax through a UK clinical trial.

But until venetoclax is listed on the Pharmaceutical Benefits Scheme (PBS) it can’t be made publicly available.

Repeatedly saying goodbye to her three children to travel more than 10,000 miles, effectively to pick up a script – she leaves on a Tuesday and gets home on a Friday – has been Deborah’s only option to stay alive, she says.

Despite the enormous toll of the traveling, she considers herself “fortunate”.

“I’m one of the very lucky ones who have been able to get it (venetoclax). I know of patients who have died… I just thank my lucky stars I have a British passport,” said Deborah.

“The fact I have gone from being terminally ill, to not having one single detectable cancer cell – within a year of being on this drug – is just amazing.

“The TGA listing is a very important step. Until it gets on the PBS though, I won’t be able to buy it here.”

Ironically, venetoclax was developed in Melbourne.

The Leukaemia Foundation's National Research Program helped to fund early work on the precursor to venetoclax by Dr Kylie Mason and her team at the Walter and Eliza Hall Institute.

Deborah was diagnosed with an incurable form of CLL on December 20, 2011. She was told she could expect to survive a mere five years.

“My daughter was only 2, my boys 7 and 5 – and that wasn’t long enough with them,” she said.

“If it wasn’t for the venetoclax clinical trial, I seriously don’t think I would have still been here.”

Reflecting on this prospect, Deborah knows just how significant the recent TGA approval is for others like her.

“I was more excited about getting the TGA approval than I was about getting on the Bart’s trial,” she said. “This really will change lives. Once on the PBS, it will make it available for everyone who needs it – it’s just such a great drug.”

Deborah’s story has been embraced by the Australian media and highlights the Leukaemia Foundation’s quest to see new life-changing medicines reviewed and made available more quickly to match the rapidly evolving world of anti-cancer therapy.

You can see Deborah’s story on ABC’s Australia Wide and on The Project (Network 10) and read her personal blog at www.abtandme.com

Venetoclax will be marketed as VenclextaTM and is approved for Australians with relapsed or refractory CLL with 17p deletion, a mutation that makes the disease resistant to standard treatment, as well as for people with relapsed or refractory CLL who do not have any other treatment options available.

Almost 80 per cent of all new CLL cases are diagnosed in people over the age of 60. It is rare in people under 40 and occurs more frequently in men than in women.

Venetoclax goes before the Pharmaceutical Benefits Advisory Committee (PBAC) at its March meeting. Historically the delay between TGA approval and PBS listing ranges from 14 to 31 months for cancer drugs but Deborah’s doctors are hopeful that because it is such an important drug it will be listed earlier.

While Deborah’s latest bone marrow biopsy showed no detectable cancer cells using the most sensitive testing now available, she continues to travel back and forth to London.

“In case I relapse, I can’t take the risk of coming off the trial until I can access it (VenclextaTM) in Australia,” she said.

“I have no super. I no longer own a home but I’m alive and looking forward to a future with my children.

You can help Deborah and other families who are facing the difficulties of a blood cancer diagnosis by making a generous donation today.

Learn more about how your donation will assist the Leukaemia Foundation in delivering free practical and emotional support to families in need.

Editor’s note: The Leukaemia Foundation continues to lobby for VenclextaTM to be PBS listed and will keep Lifeblood readers updated on our progress.

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