'˜I want to look in my daughter's eyes and tell her we tried everything we could' '“ Portsmouth mums launch desperate battle to get life-changing medicine for daughters on NHS

Gemma Weir and Michelle Frank, from Paulsgrove, in Portsmouth, have set up a petition to get treatment drug Orkambi available for free.

The drug can be used to treat people with cystic fibrosis, an inherited disease which causes the lungs and the digestive system to be clogged up with mucus.

Both Gemma, 34, and Michelle, 32, have young daughters with the condition who have to do a rigorous daily treatment regime of taking tablets, doing physiotherapy, using a nebuliser (a way to deliver medication) and occasionally, having intravenous antibiotics.

They want to see Orkambi added to the list of treatments available on the NHS and have got the 100,000 signatures needed on their petition which they can now deliver to parliament where it will be debated.

Currently, the National Institute for Health and Care Excellence (Nice) has not recommended the drug to be on the NHS due to its cost of £104,000 per patient per year.

Orkambi is state-funded in other countries such as Ireland, Holland, Germany, America and Greece.

Until recently, the European Commission said it should only be used for people aged 12 or over.

But earlier this year, the EU approved it for those six to 11.

Michelle, whose daughter Emma aged six has cystic fibrosis, said: ‘Orkambi won’t cure cystic fibrosis but it will halt it.

‘It means my daughter will be able to live a practically normal life and it won’t be case of maybe she will get to be 30 but that she will get to 30.

‘It is life-changing and will help people with cystic fibrosis have a normal life span.

‘Emma could be taking the drug now and it is so frustrating that it is available in Ireland but not over here.

‘Enough is enough, we need to have these drugs available to people who desperately need them.

‘I want to be able to look in my daughter’s eyes and tell her that we fought and we tried everything we could to get it.

‘I want to tell her that we were successful and we got it.’

Michelle, of Macaulay Avenue, and Gemma, of Coleridge Road, acknowledge the drug is expensive and would cost the NHS millions of pounds a year but said a price should not be put on someone’s life.

‘It is expensive but that is not my concern,’ Michelle said.

‘The NHS spends billions to treat conditions related to smoking and drinking, those are life-style choices – but cystic fibrosis is not a life choice.’

Gemma and Michelle set up the petition less than three weeks ago and already have more than 111,000 signatures.

The pair said Orkambi being licensed for younger children ‘lit the fire in their bellies’ to get it available on the NHS.

Gemma, whose four-year-old daughter Ivy has cystic fibrosis, said: ‘Because both our children are only young, even if Orkambi had been available they wouldn’t be able to take it.

‘But a couple weeks ago it was licensed for children aged six and up. That means Emma would be able to take it and Ivy could as soon as she turns six.

‘Knowing Emma is eligible but it isn’t available lit the fire in our bellies to get something done. We set up the petition and told everyone about it and to share it.’

In 17 days the petition has got 111,000 signatures and Michelle said Portsmouth has been really supportive.

‘Until a couple days ago, most of the signatures had come from people in Portsmouth,’ she said.

‘Northern Ireland might have overtaken the city but we are so grateful to everyone who has signed and is sharing it. ‘We have people at work and friends watching the count go up and it is amazing.

‘The more signatures we get the better and the city has been great in supporting us.

‘Often people don’t understand how serious cystic fibrosis is because it isn’t a visible illness.

‘Doing this campaign and petition means we have been able to raise awareness and how people understand.’

A NICE spokeswoman said: ‘NICE issued guidance in July 2016 which did not recommend Orkambi to treat cystic fibrosis.

‘The decision was based on the evidence and price proposal provided by Vertex at the time of the appraisal.’

She added the guidance could be reviewed sooner than its due in 2019 if there is new evidence or ‘if the company puts forward a proposal that demonstrates their drug is cost-effective’.

To sign the petition visit petition.parliament.uk/petitions/209455