This summer, Katie Brown packed a bag and hopped on a flight to Australia.
For most 24-year-olds, five weeks travelling down under would be an unforgettable adventure. For her, it was little short of miraculous.
Brown, from Garsington in Oxfordshire, was diagnosed at the age of 12 with myasthenia gravis (MG), a rare autoimmune condition that attacks the muscles. By the time she was 19, she was completely bed-bound and required her mother to wash and dress her.
Finally, though, came a treatment that turned her life around. She was put on an early access scheme for a breakthrough drug called zilucoplan. A daily injection, which she gives herself at home, stops her immune system attacking her body. The impact was immediate. “The day after I started on it I was able to sit up in bed, I ran downstairs and danced around the kitchen,” she said. “Suddenly every muscle worked.”
This treatment, however, has been denied to other patients with MG.
The drugs watchdog Nice has rejected it for use on the NHS on cost grounds. Not only that, but another breakthrough treatment for MG, efgartigimod, has also been turned down. A third treatment for the condition, rozanolixizumab, was given a draft rejection last year, and campaigners hold little hope that decision will be reversed when Nice delivers its final ruling.
The companies behind two other drugs, eculizumab and ravulizumab, have decided to terminate their Nice applications at the appraisal stage.
Muscular Dystrophy UK, a charity that supports patients with muscle-wasting and weakening conditions, has described these decisions, which deny patients access to a remarkable surge of new treatments, as “devastating” for those living with MG. The condition affects about 4,500 people in Britain.
Charlotte Roy, research communications manager at the charity, says the Nice methodology is too rigid to deal with rare diseases.
“We’re seeing time and time again that rare diseases do not fit into this cookie-cutter economic modelling,” she said. “These conditions are really complex. The drugs are going to be more expensive because there’s not as many patients.”
Nice has come under more criticism in recent weeks for the way it restricts what NHS will pay for medicines, fuelling a wider row between the pharmaceutical industry and the government on drugs pricing.
The US drugs giant Merck said on Wednesday that it was scrapping a £1 billion expansion of its British operations, blaming the “overall undervaluation of innovative medicines and vaccines by successive UK governments”.
Roy said pricing rows cut off innovation. “We’re pushing the UK to become this innovative powerhouse for new research. But the more that treatments like these are rejected, the more chance that companies are not going to come to us.”
Nico Reynders, general manager of UCB UK and Ireland, which makes zilucoplan, said: “No new treatments for this devastating condition have been launched in the UK for more than a decade, despite several targeted treatments for MG receiving marketing authorisation. Science has evolved, but our healthcare system has not kept pace, and we are facing a reality where patients in the UK may not have access to treatments discovered here and readily available to patients across Europe and the rest of the world.”
‘Even things like making a bed, I really enjoy it’
Andy Stephenson
MICHAEL POWELL FOR THE SUNDAY TIMES
Andy Stephenson, 45, from Doncaster, was given access to efgartigimod through an early access scheme similar to the one that allowed Brown to get zilucoplan.
The father of four and former soldier had been rushed to hospital in respiratory arrest 31 times in two decades, his muscles having stopped working to the point that he could no longer breathe. After getting access to efgartigimod, however, his condition rapidly improved.
He now takes great joy from the little things in life. “Even things like making a bed, I really enjoy it,” he said. “I know it sounds really bizarre, but being able to do that shows that I’m doing all right.”
That routine pleasure would not be possible without the medicine.
“These are next-generation drugs that have been proven to work,” he added. “I do understand that there’s got to be a limit on cost, but the decision to reject them is a real shame.”
Argenx, which makes efgartigimod, said: “We are continuing to liaise with the NHS, as well as the clinical and patient community, to examine if there may be an alternative path to securing routine access to this medicine.”
Both Stephenson and Brown have been able to continue taking the two treatments after their manufacturers agreed to continue to provide the medicines to patients already on the early access scheme.
The drugs are also available privately, but few will be able to afford them. Zilucoplan costs between £190,000 and £370,000 a year, depending on a patient’s body weight, and efgartigimod roughly £200,000 a year.
The NHS was offered a confidential discount on both drugs, but a Nice spokesman said: “For both efgartigimod and zilucoplan, the most likely cost-effectiveness estimates are substantially above what Nice considers an acceptable use of NHS resources.
“We know our decision not to recommend efgartigimod and zilucoplan was disappointing for people living with this debilitating condition and those who care for them. We are very conscious of the responsibility we bear in making our decisions. Every pound of the NHS budget can only be spent once.”
Brown, meanwhile, is counting her blessings. Having been unable to sit exams when she was younger, she is studying for her A-levels — maths, further maths and economics — with an eye on a career in finance or statistics.
“The change has been very dramatic,” she said. “I was one of the worst cases of MG. I had every single symptom. And now I’m doing so well on this medication. I don’t think anyone could really believe it.”
Source link
-
-
-
-
-
