Parents held a candlelit vigil outside parliament on Monday after MPs debated a petition to make life changing drugs available on the NHS.
Wallingford couple Zoe and Graham Bagley’s three-year-old daughter, Grace, takes a cocktail of medication and uses physiotherapy to treat her incurable cystic fibrosis.
A drug called Orkambi could slow the decline in her lung function by almost half, but was not recommended to the NHS by government health watchdog the National Institute for Health and Care Excellence (NICE) in July 2016 due to its cost.
After an online petition reached more than 113,000 signatures, the issue was raised in parliament as nearly 100 protestors gathered outside in the wintery conditions to lend their voice to the debate.
Zoe, who also met Henley MP John Howell on Friday, said: “It seems like it was a hugely positive debate, 60 MPs attended and lots of them used personal stories from their constituencies.
“There’s still a way to go because there’s probably a lot of wrangling to be done over the price, but it feels like we have got the support now and the momentum is behind us.”
David Ramsden, chief executive of the Cystic Fibrosis Trust, said the debate is a “major milestone” for bringing about access to precision medicines.
On Friday, NHS England put forward a new proposal to Vertex, manufacturers of Orkambi, which was dismissed by the pharmaceutical company.
A Vertex spokesperson said: “We are astonished and dismayed by NHS England’s initial response to our portfolio approach.
“It amounts to a refusal to make any additional funding available for Orkambi or future medicines.
“The scale of parliamentary engagement in this debate echoes and amplifies the CF community’s sense of urgency.
“Every day matters for CF patients and we stand ready and willing to engage in meaningful dialogue about a way forward.”
An NHS England spokesperson said the NHS can only offer treatments which are “both effective for patients and offer good value for taxpayers” and said further progress is “frankly unlikely” unless Vertex reviews its proposed pricing.
A NICE spokesperson said it remains open to a further approach with “new data or a revised price”.