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Pharmaceutical Benefits Scheme Orkambi rejection leaves cystic fibrosis patients heartbroken

Fighter: Morgan Gollan is refusing to give up on access to Orkambi. “We will band together and be as loud as we can until our voice is heard. We have been fighting our whole life so giving up is just not an option.” Picture: Marina NeilMORGAN Gollan made an impassioned promise to herlate friend Brooke, shortly before she passed away at the age of16 after a failed double lung transplant.
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Ms Gollan told her friend she would never give up fighting against cystic fibrosis.

“I’ve had 10 close friends with cystic fibrosis pass away and have heard of many more,” she said. “You can see your own mortality. You take on their fight as well and speak up for them, so losing them is not in vain.”

Ms Gollan, 25, also lives with the life-shortening chronic condition, whichaffects the lungs and digestive system.

She will be among a group that will march onCanberra when Parliament returns in February, to ask the government to support their right to access Vertex’s “life changing” new drug, Orkambi.

The drug is available to eligible people in the United States, Austria, Germany and France and targets thecause of cystic fibrosis, not just the symptoms, and has been shown in clinical trials toimprovelung function and reducehospitalisationand antibiotic use.

Ms Gollan saidOrkambi hadthe potential to change 1000 n lives, but costs about US $260,000 without subsidy each year.

The government-appointedPharmaceutical Benefits Advisory Committee has recognised the drug’s “potential clinical value” but announced this month it would notrecommend it for listing on the Pharmaceutical Benefits Scheme “on the basis of unfavourable and uncertain cost effectiveness at the requested price”.

“The PBAC noted that the resubmission did not address the issues previously identified in its consideration of the [first] March 2016 submission,” a government spokesperson said.

“The PBAC particularly noted the continuing uncertainty regarding long-term benefits of treatment on lung function and overall survival.

“The PBAC noted that the estimated net cost…to government was more than $100 million in each of the first five years of listing.”

Vertex said “government timelines” prevented reconsideration before July 2017, “meaning that n patient access is now unlikely until the second half of 2017 or 2018”.

Minister for Health Sussan Ley said she was looking to international collaboration to address the “unsustainable and unaffordable”price being demanded.

Ms Gollan – whoreceivescompassionate access to the drug and has seen her lung capacity grow from 35 per cent to a high of 50 per cent –said people with CF spent their lives wishing for a cure.

“Right now there’s a revolutionary treatment that works but isn’t available due to cost. That’s worse than having no treatments available at all –it’s heartbreaking.”

Rebecca Northam, 20, said her lung capacity had risen from below 40 per cent to a high of 58 per cent since she started taking Orkambi.

Her number of hospital admissions has halved in the past year andshe is attending most of her university lectures, picking up more shifts at her workplace and has started going to the gym.

“So many different aspects of my life are better,” she said.

“It’s scary to think where I would be if I did not have access to it –but there’s so many people in that position.

“It should not be this hard and people should not have fight this much.”

Ms Northam has started a petition that already has more than 37,000 signatures, asking Vertex and the government to work together to make the drug available on the PBS.

Cystic Fibrosis CEO Nettie Burke said patients should “not be caught in the financial crossfire between the government and Vertex”.

“It is time to ask our elected representative in Canberra to support our urgent need for these transformational medicines and call for a debate in Parliament focusing on social and health equity in ,”she said.

“Cystic Fibrosis will be asking why people born with CF, through no fault of their own, are not being supported and given access to life saving drugs. Their disease is not the result of poor lifestyle choices and they have not endangered their health through any precarious behaviour.”