At yesterday’s Health Questions, I raised the case of Oli Rayner, a cystic fibrosis patient whose condition deteriorated in his 30s. He was given the drug Orkambi – which is currently not routinely available on the NHS for the thousands of cystic fibrosis patients who stand to benefit from it – just to make him well enough to undergo a lung transplant operation.
This follows a debate on NICE appraisals of treatments for rare diseases last week, during which we heard that patients with conditions such as spinal muscular atrophy, PKU and Batten disease are also being denied access to life-saving drugs due to arguments over cost-effectiveness.
It is ludicrous to wait until people are virtually at death’s door before being prepared to give them life-saving drugs. I am pleased that the Secretary of State is taking an interest in this and I hope a solution can be reached very soon.