Access to miracle drug trikafta a 'lifeline' for cystic fibrosis patients with no other hope

Ed Lee, pictured with wife Kalyn Ponti, says taking trikafta for cystic fibrosis has changed his life. Photo / Supplied

A handful of cystic fibrosis patients have been thrown a lifeline with a drug company offering to provide their "miracle drug" for free to Kiwis who have exhausted all other options.

For nearly 18 months Cystic Fibrosis NZ has been campaigning for trikafta to be approved for use by Medsafe and funded by Pharmac.

It is already being used in a number of countries including the UK, Republic of Ireland, United States, Denmark, Germany and Switzerland with patients hailing it as life-saving.

Drug manufacturer Vertex last month applied to Medsafe for approval for the drug's use in New Zealand and plans to put an application in to Pharmac for funding "very soon".

But, in the meantime, Vertex has agreed to provide a "managed access programme" which would provide free access for the most critically ill patients who had exhausted all other medicinal options.

Cystic Fibrosis NZ chief executive Jane Bollard said they were thankful for the decision which was a step in the right direction.

"It does provide a lifeline for the sickest people with cystic fibrosis," she said. "These people don't have the luxury of time while they wait for it to be assessed by Medsafe and Pharmac."

Trikafta is the first drug to treat the cause of cystic fibrosis rather than simply address the symptoms. Trials had proven it slowed the progression of the terminal genetic disease which causes the body to produce thick, sticky mucus, damaging the lungs,
digestive system, liver and other parts of the body.

"It's pretty miraculous," Bollard said. "It may save someone from having a lung transplant."

At a cost of hundreds of thousands of dollars a year, only a few Kiwis had been able to pay for the drug themselves but it was making a difference for those who had, she said.

"It's really changed their lives."

Trikafta increases lung function and weight gain, reduces the burden of treatment,
improves quality of life, and increases life expectancy.

Cystic fibrosis sufferer Ed Lee decided to bite the bullet and self-fund the drug a year and a half ago.

"It's completely changed my life," he said. "This drug has alleviated every single symptom of cystic fibrosis for me. Within hours of taking trikafta suddenly all the mucus inside your lungs comes out and in three to four days you're no longer coughing."

Lee said since starting trikafta his lung function had increased from 28 per cent to 50 per cent and he now ran 5km a day.

"Trikafta for cystic fibrosis is like insulin for a diabetic. Without it we are going to suffer and die. We need this."

As for the managed access programme, Lee said it would provide hope for families who before had none.

"This is absolutely a game-changer for certain people who have no other chance in life. People who are about to have a lung transplant, this thing gives them hope that they don't have to go through that horrific, really dangerous operation."

Lee, a financial adviser, said before trikafta he did not expect to make it to 65 so had never joined Kiwisaver but he had recently signed up. He and his wife were also talking about starting a family.

"It really gives cystic fibrosis sufferers hope that they can have a normal life - a healthy life."

Under the managed access programme clinicians would have to apply on behalf of their patients and only a small number would be selected but Bollard saw it as a sign Vertex was confident it would get approval and funding in the country.

Despite the good news, Bollard said the organisation would continue to push for access for the 90 per cent of patients who would benefit from the drug.

A petition to get all cystic fibrosis patients the drug already had more than 35,700 signatures and would be presented at Parliament in September.

Medsafe had made the drug a priority, meaning it would have its initial evaluation sooner than usual and Pharmac had agreed to consider funding at the same time it was going through Medsafe which was a rare move, Bollard said.