Colchester mum fears for Cystic Fibrosis medicine withdrawal

Lizzie Phillips is facing an “agonising wait to see if her young daughter’s hope of a normal life will be taken away” as health bosses consider withdrawing medicine to treat the condition.

The National Institute for Health and Care Excellence last week published draft guidance it would not recommend three of the cystic fibrosis drug treatments for use on new patients.

Despite the institute recognising the major benefits of modulator drugs Trikafta, known as Kaftrio in the UK, Symkevi and Orkambi, they were seen as not cost-effective.

If passed, young children or babies who are yet to receive these therapies, made by pharmaceutical company Vertex, would not be guaranteed them under NHS care.

Colchester mum Lizzie Phillips now faces an “agonising wait” to see if her daughter, Alice Andrews, four, will have access to the “life-changing” treatment.

Family - Colchester mum Lizzie Phillips with her daughter Alice Andrews (Image: Public)

With Kaftrio not available to children under six, Lizzie was hoping to get Alice, who currently takes Orkambi, on the treatment when she was old enough to qualify.

However, after the recommendation to withdraw what some have dubbed the “miracle medication”, Lizzie is concerned for her daughter’s future.

“This treatment reduces the number of other meds needed and the amount of hospital stays too”, she said.

“Alice moving onto Kaftrio would reduce coughs and colds, physiotherapy and more.

“She is on 12 different meds which equates to about 30 tablets a day.

“But one of the biggest things is doubling life expectancy.

“Babies born with CF have an expectancy of mid 40s, but taking Kaftrio is expected to almost double that with no other health complications.”

Now, with a four-week consultation in place, Lizzie says she faces an “agonising wait to see if her daughters hope of a normal life will be taken away from her”.

Smiles - Alice Andrews, four, was born with Cystic Fibrosis (Image: Public)

“This drug would take away so much worry and stress”, she added.

“We can give her the childhood she deserves to have.

“Alice’s dad and I work full time, so one of us would have to give up our career to help manage her day-to-day care.

“If it was passed, Alice would be able to stay on Orkambi as she is protected, but they are not sure if she could move on to Kaftrio.

“To take away the access to these drugs would be life-changing.”

What have NICE said?

The National Institute for Health and Care Excellence (NICE) said it wants to "ensure taxpayers are getting their value for money".

It also added the consultation is to ensure the agency has all "relevant information to accurately capture the value of these effective medicines".

Helen Knight, director of medicines evaluation at NICE, said: "We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money after interim access where further data was collected.

“The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions.

“This is to ensure that we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision."

Ms Knight continued: "We are continuing to work collaboratively with the company, NHS England and other stakeholders including the Cystic Fibrosis Trust to deliver the best outcome both for people with cystic fibrosis and for the wider NHS.  

"Existing patients and new patients who are started on treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome."