NHS Rejects Funding for Life-Changing Drugs for Myasthenia Gravis Despite Dramatic Patient Improvements
London – The National Health Service has declined to fund two recently approved drugs, efgartigimod and zilucoplan, for patients with myasthenia gravis (MG), a rare autoimmune condition causing severe muscle weakness, despite evidence of significant improvements in patients’ quality of life. The decision has sparked frustration among patients and pharmaceutical companies, even as both manufacturers have agreed to continue supplying the medications to those already receiving them through early access schemes.
Andy Stephenson,45,of Doncaster,a former soldier and father of four,experienced 31 episodes of respiratory arrest over two decades due to MG. He received efgartigimod through an early access program and saw a rapid improvement in his condition. “Even things like making a bed, I really enjoy it,” Stephenson said. “I know it sounds really bizarre, but being able to do that shows that I’m doing all right.”
Similarly,a young woman identified only as Brown,who previously experienced every symptom of MG and was unable to sit exams,is now studying for A-levels in maths,further maths,and economics,aiming for a career in finance or statistics. “The change has been very dramatic,” she stated. “I was one of the worst cases of MG… And now I’m doing so well on this medication. I don’t think anyone could really believe it.”
Both drugs are considered “next-generation” treatments proven to be effective against MG. Though, their high cost presents a barrier to NHS funding.Zilucoplan ranges from £190,000 to £370,000 annually, depending on patient weight, while efgartigimod costs approximately £200,000 per year.
The NHS was offered a confidential discount by both Argenx (efgartigimod) and the manufacturer of zilucoplan, but a spokesperson for the National Institute for Health and Care Excellence (NICE) explained, “For both efgartigimod and zilucoplan, the most likely cost-effectiveness estimates are substantially above what nice considers an acceptable use of NHS resources.”
NICE acknowledged the disappointment caused by the decision, stating, “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… Every pound of the NHS budget can only be spent once.”
Argenx stated it is “continuing to liaise with the NHS, as well as the clinical and patient community, to examine if there may be an choice path to securing routine access to this medicine.” The drugs remain available privately, though the cost is prohibitive for most.
