The Minister for Secondary Care (Karin Smyth)
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.
Chris McDonald
I thank the Minister for her response, to which I am listening very carefully. I appreciate her point that it would be inappropriate for the Department of Health and Social Care to direct what trusts should do, but what we have heard from the trusts is that they would like to issue this drug, but have certain issues and problems. Perhaps it might simply be a matter of the Department giving help and support, and facilitating information-sharing between trusts that have made this work and those that have not. Perhaps it could be more encouragement than direction.
Karin Smyth
I thank my hon. Friend for that constructive suggestion. We need to wait to see how the NICE recommendation goes in July. With this disease and so many others, it is important to share learning and information, and trusts should be encouraged and supported in doing so. We will work with him on that constructive recommendation.
I understand my hon. Friend’s concerns about the fact that non-ambulant patients are not yet able to access givinostat, but it is important to note that the eligibility criteria for participation in the early access programmes have been determined by the pharmaceutical company. NHS trusts that decide to participate in the EAP must only provide treatment in line with the criteria, which state that patients must be ambulant. A clinical trial is being carried out by the pharmaceutical company to evaluate the safety and tolerability of the drug in non-ambulant patients, and to further explore the efficacy of the drug in this population. I know that for the patients and families affected, it will be disappointing to hear that there is no access to the drug for ambulant patients before a NICE decision, or for non-ambulant patients prior to clinical trials being concluded. I want to assure my hon. Friend that we have arrangements in place to support rapid access to new medicines.
Karin Smyth
I will not; I want to finish in the time available, and I think Members want to hear the full response.
Outside of company-led EAPs, there are established routes for patients to get access to new, innovative medicines prior to them being licensed. The early access to medicines scheme, or EAMS, helps give people in the UK with life-threatening or seriously debilitating conditions early access to new medicines that are not yet licensed where there is a clear unmet medical need. EAMS is supported by key partners, including the MHRA, NICE and NHS England, and is a key part of this Government’s commitment to accelerating patient access to innovative, life-changing treatments, in support of the UK’s position as a global leader in life sciences. In fact, since the scheme launched in 2014, over 50 medicines, including for this disease, have benefited from being accessed early through EAMS.
The innovative medicines fund has also made available £340 million of ringfenced funding for the NHS to fund early access to medicines that NICE has recommended with managed access. Through this process, licensed treatments that demonstrate substantial clinical promise but still have significant uncertainty around their clinical and cost-effectiveness can be funded. Further evidence is then collected on the drug for a defined period of time. That is considered by NICE in determining whether the drug can be recommended for routine NHS funding.
The Secretary of State has been clear that if givinostat is recommended by NICE in draft guidance, NHS England should aim to work with the pharmaceutical company to provide early funding through the innovative medicines fund. This could potentially speed up access by up to five months, and the treatment could be funded as soon as this summer, if recommended.
This scope of this debate is wider than just access to new medicines. It is important to note that while rare diseases are individually rare, they are collectively common. One in 17 people will be affected by a rare condition over their lifetime. The UK rare diseases framework outlines four priorities, based on engagement with the rare disease community. They are: helping patients to get a final diagnosis faster, increasing awareness of rare diseases among healthcare professionals, better co-ordination of care, and improving access to specialist care, treatments and drugs. In England, we publish a rare diseases action plan annually. These detail the specific steps we are taking to meet the shared priorities of the framework. I am pleased to highlight the 2025 England action plan, which was published in February this year on Rare Disease Day. One such action is reviewing the effectiveness of early access schemes, such as the early access to medicines scheme, the innovative licensing and access pathway, and the innovative medicines fund. They are all designed to help make innovative treatments available earlier to patients who need them. We are specifically considering how well they support access to treatment for people living with rare diseases like Duchenne.
NHS England, NICE and MHRA will meet annually to continue to discuss progress on these schemes. These meetings will include representatives from patient advocacy groups and from industry, and clinical researchers, and the next one will happen in the summer.
Managing a complex rare condition can be challenging, and it often means interacting with many different specialists and providers of health and social care. It can mean travelling across the country to access highly specialist care from experts. All of that can add up to a significant emotional and physical burden, and it can deepen inequalities. Co-ordination of care can minimise this burden on patients and their carers, and it can ensure that healthcare professionals work together to provide the best possible care, as we have discussed this evening.
In the 2025 action plan, we have introduced a new action to incentivise providers to run clinics for multi-system disorders, in order to reduce the number of appointments and improve co-ordination of care for families. The NHS is also working to include the definition of “co-ordination of care” that is set out in the CONCORD—co-ordinated care of rare diseases—study in all new and revised NHS service specifications for patients with rare diseases.
I recognise how hard it is when patients want access to these new treatments. I also recognise the distress and worry it causes, not only to patients, but their families and friends. Hon. Members have articulated that well on behalf of their constituents this evening. The Government are committed to providing access to the most innovative medicines, but it has to be at a price that provides value for the NHS, and it has to be clinically safe and effective. That is why we are working hard with industry, NICE and MHRA to make that happen. I know that my hon. Friend the Member for Stockton North will continue to work with the Government and providers to make that happen. I am grateful for the opportunity to respond to this debate on such an important issue.
Question put and agreed to.