Contribution
It is a real pleasure to respond to this moving debate. I know that many people with Duchenne muscular dystrophy and their families will have wanted to tune in to hear what was said this afternoon. I thank Members who have contributed in different ways to the debate. In particular, I thank my hon. Friend the Member for Stockton North (Chris McDonald), who secured this debate and advocated so powerfully for these families. He really brought to life the experiences of Benjamin, Jack and Eli, and I commend him for doing so.
I first acknowledge the profound impact that this debilitating disease has on those living with it, and their families, and the urgent need for new and effective treatments. As has been said, my right hon. Friend the Secretary of State for Health and Social Care heard at first hand from people affected by this condition earlier this year, when he attended an event hosted by Duchenne UK. He met many young patients and listened to what they said about the challenges that they face. As we all know, meeting families and individuals from our constituencies leaves a long-lasting effect on us, and it is important that we continue to meet them.
Timely and equitable access to innovative medicines for the treatment of DMD and other rare diseases mentioned today is of the utmost importance. The National Institute for Health and Care Excellence is the independent body responsible for assessing whether new licensed medicines can be recommended for routine use in the NHS, based on a thorough assessment of their clinical effectiveness and cost-effectiveness. Through this process, many thousands of patients, including those with rare diseases, have been able to benefit from effective new treatments at prices that represent value to the NHS. NICE has been able to recommend two medicines for the treatment of DMD: ataluren, recommended in 2023, and vamorolone, which was recommended in January this year and is now available on the NHS to around 1,700 eligible patients, in line with NICE’s recommendations.
As my hon. Friend has said, NICE is appraising givinostat, and the first NICE committee meeting is scheduled for July this year. If the medicine is recommended, the NHS in England will be legally required to fund it. I am aware that a small number of patients in the UK have been receiving treatment with this drug through a company-led early access programme, established by the pharmaceutical company Italfarmaco, as we have heard. It is important to note that participation in these programmes is decided at NHS trust level, and although the drug is free to patients taking part in it and to the NHS, NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP.
NHS England has published guidance on free-of-charge medicine schemes, such as the givinostat EAP, providing advice on financial, administrative and clinical risks. NHS England cannot, however, centrally direct NHS trusts to participate in company sponsored EAPs like this one, or in any other private activity. To issue any form of national direction around participation in EAPs would both pre-empt and undermine the role of NICE, whose purpose is to advise the NHS on whether particular treatments should be made routinely available on the NHS.
Even when there is an agreement that a company will continue to provide a drug free of charge in the event of a negative decision by NICE, participating trusts remain liable to cover the significant costs of delivering that service, including the cost of the clinical resources and staff time needed. That would be outside their funding allocation and in addition to paying for any subsequent NICE-recommended treatments that they would be mandated to fund.