In the days after Queensland mother Ella Sawyer gave birth, her baby daughter Evie didn’t gain a lot of weight.
But initially, doctors weren’t concerned.
Key points:
- Cystic Fibrosis sufferers are calling for ‘life changing’ drug Trikafta to be added to the PBS
- The medicine currently costs patients nearly $300,000 per year
- Drug company Vertex says it wants to offer government funded access to Trikafta as quickly as possible
“As she was a breastfed baby, doctors were saying to me, ‘You know it’s very hard to tell how much they’re getting,” Ms Sawyer said.
“No one thought there was a problem because she was putting on weight but just at a minimum.
But just six weeks later, despite no known family history, baby Evie was diagnosed with cystic fibrosis — a life-limiting recessive genetic condition.
“My husband and I both didn’t know that we were carriers,” Ms Sawyer said.
“I hadn’t really heard a lot about cystic fibrosis. I’m not sure I even knew what it was.”
Cystic Fibrosis primarily affects the lungs and digestive system because of a malfunction in the exocrine system, which is responsible for producing saliva, sweat, tears and mucous.
Approximately 3,500 Australians live with the disease.
Exorbitant cost of medication
Evie is now 12 and manages her condition with numerous medications, supplements and twice-daily breathing exercises to help clear her chest.
She has increased calorie needs, takes enzymes with every meal and is prone to long-lasting infections.
The one drug that would drastically improve Evie’s lung function is Vertex’s Trikafta.
But it currently costs Australian patients nearly $300,000 a year.
The drug is already available and reimbursed in 17 countries but is not yet subsidised in Australia.
Cystic Fibrosis Australia chief executive Nettie Burke said the lack of access to Trikafta was putting lives at risk.
“I think we do, but we can’t profess that when we can’t get drugs that have been in America for two years.”
‘A whole lot of babies being born’
Ms Burke said recent trials have shown Trikafta to be “life-changing”.
She said some people diagnosed with cystic fibrosis who previously were unable to have children were now starting families thanks to the medication.
“One of the wonderful things we’ve seen from people overseas, or from those on trials, is that there’s a whole lot of babies being born.
“Before, that wasn’t necessarily going to be the case because people were too unwell to have a family, but we’ve seen a big increase in babies.
Ms Burke and families of people with cystic fibrosis are renewing calls for Trikafta to be added to the Pharmaceutical Benefits Scheme (PBS).
They want immediate compassionate access given to all eligible patients while government negotiations continue.
Ms Burke says she fears lives will be lost and families destroyed while protracted price negotiations play out.
“We could save lives by getting access immediately,” Ms Burke said.
‘Deep commitment’ to list medicine
The Department of Health said the federal government had a deep commitment to list all medicines on the PBS that were recommended by independent medical experts on the Pharmaceutical Benefits Advisory Committee (PBAC).
By law, no medicine can be listed without such recommendation.
The PBAC deferred making a recommendation about listing Trikafta on the PBS at its recent meeting in May to allow further engagement with Vertex Pharmaceuticals.
The department said the government would not hesitate to list Trikafta on the PBS if it was recommended by the medical experts and the company agreed to the terms of the listing.
Vertex said it wants to find “flexible and creative solutions” to ensure all 2,200 eligible patients who can benefit from treatment have government-funded access to Trikafta as quickly as possible.
“We know this can be done as Trikafta is now approved and reimbursed in 17 countries, including Denmark, Finland, Germany, Ireland, Israel, Switzerland and the countries within the UK,” a Vertex Australia and New Zealand spokesperson said.
“In the last two weeks, four new agreements were signed with France, Italy, Czech Republic and Austria.”
‘Someone’s life comes down to a price’
The drug company will continue to provides Trikafta free of charge to critically ill patients for whom there is no other medicinal option, subject to specific eligibility criteria and if requested by the person’s doctor.
Fewer than 200 Australians fall into that category and 12-year-old Evie doesn’t qualify.
“It’s now, for us, a bit of a waiting game. It’s almost like a postcode lottery,” Ms Sawyer said.
Ms Sawyer said the lung function of children with cystic fibrosis could drop quickly.
“If Evie were to get the flu or another infection, she could have a big loss of lung function quite quickly whilst we wait for price negotiations to go on,” she said.
“It’s just very frustrating when the quality of someone’s life comes down to a price.”
The next update from the PBAC is due in August.