AMs set to hold debate on cystic fibrosis drug
ASSEMBLY Members are set to hold a debate on access to life-enhancing drugs for cystic fibrosis patients after more than 5,000 people signed a petition.
Throughout 2017 patients with the progressive condition of the lungs and digestive system and their families called for “precision” medicines like Orkambi to be made routinely available on the Welsh NHS.
But the National Institute for Health and Care Excellence (Nice) says it is currently unable to recommend the drug for NHS use because it is not cost-effective.
Dozens of people, some wearing oxygen masks and waving placards, protested on the steps of the Senedd in June in a bid to make Orkambi free at the point of need.
But for the matter to be automatically debated in the Assembly campaigners needed to submit a 5,000-signature petition by New Year’s Eve.
The petition states: “We call on the National Assembly for Wales to call for a resolution to ongoing negotiations between NHS Wales, the All Wales Medicines Strategy Group, the Welsh Health and Specialised Services Committee and Vertex Pharmaceuticals regarding access to the cystic fibrosis medicine, Orkambi, as a matter of urgency.
“It is essential that a fair and sustainable method of reimbursement is found for Orkambi and for the pipeline of future treatments.
“People in Wales have been waiting too long for this transformative drug. They deserve better.”
And just days before their deadline they managed to achieve the 5,000 target thanks in part to Beth Clarke, 36, from Cardiff, who wrote a brutally honest account of her battle with the condition for the Western Mail.
Rhian Barrance, who launched the petition, said: “It’s great that the petition has hit its target. We owe a huge thanks to the Western Mail for highlighting this to their readers, and everyone who has got behind the petition to help us achieve this goal.”
Lynsey Beswick, public affairs manager at the Cystic Fibrosis Trust, added: “Our aim is to make sure that all people with cystic fibrosis who stand to benefit from these precision medicines get access to them as soon as possible. We are delighted that the public have got behind this petition to help us make sure a debate on this issue will be considered by the Welsh Assembly.”
Cystic fibrosis is an inherited condition in which the lungs and digestive system can become clogged with thick, sticky mucus.
It can severely shorten people’s lives, with many needing life-saving lung transplants when the condition worsens.
Orkambi has been deemed an “extraordinarily important drug” by the Cystic Fibrosis Trust because it treats the underlying cause of the condition.
It is effective in treating the 40% of the UK population of people with cystic fibrosis who have two copies of the F508del mutation.
Campaigners claim the drug would reduce hospital admissions by half and help prevent pulmonary exacerbations, which is a leading cause of death in cystic fibrosis patients.
It is currently only being prescribed to people on compassionate grounds.