One of the UK’s oldest Morquio sufferers has spoken of her hope that health bosses will today “really listen” to the evidence as they prepare to decide whether to approve a drug she says has transformed her life.
Angela Paton, 41, of Kippax, is due to speak today at the next crucial meeting of health regulator NICE before it publishes its final decision in October on whether to recommend the pioneering new drug Vimizim for those with the rare and incurable Morquio Syndrome.
The condition - which currently affects 88 people in England - limits growth and mobility and shortens life expectancy to an average age of 25. But those on the clinical trials say they found Vimizim gave them a better quality of life - and Angela credits the drug with allowing her to continue a full-time job.
NICE’s meeting today will discuss feedback from a public consultation and hear evidence from experts and two patients, including Angela.
Speaking beforehand, she told YEP: “It feels a little bit like we’re being put on trial - having to justify why our lives are worth saving.
“I am feeling a huge weight of responsibility in anticipation of the meeting. This is not just my life at stake but that of all 88 people who are currently living with Morquio in England. I just hope that the NICE committee will really listen to what I and the other patient and clinical experts have to say and will see how refusing Vimizim for funding is not only cruel but is completely and utterly immoral.”
Draft guidance published by the committee in June indicated they may not approve Vimizim - which Angela fears is largely due to cost, a basis she says is “wholly inappropriate” for an ultra-rare disease.
As previously reported by the YEP, NHS England recently announced they were waiting for NICE’s final recommendations before deciding whether to fund the drug - leaving patients with no access to treatment and facing an uncertain future with potentially irreversible consequences.
A crowdfunding campaign has been launched to try to raise the £4m needed to pay for four months of Vimizim. To donate visit www.crowdfunder.co.uk/stop-rare-being-unfair-fund-the-gap.