Duchenne Muscular Dystrophy

Debate between Freddie van Mierlo and Karin Smyth

Thursday 12th June 2025 Commons Chamber

[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.

[Freddie van Mierlo]

Will the Minister give way?

[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.

Attention Deficit Hyperactivity Disorder

Debate between Freddie van Mierlo and Karin Smyth

Tuesday 10th June 2025 Westminster Hall

[Karin Smyth]

I will come on to shared care agreements. As the hon. Member says, this is a devolved matter, and I am focusing on support for issues facing ADHD services in England and what we are doing to support trusts to get back on top of waiting lists and improve access to services.

First, NHS England has commissioned an independent ADHD taskforce, which is working to bring together those with lived experience and experts from the NHS, education, charity and justice sectors. The taskforce is developing a better understanding of the challenges affecting those with ADHD, including timely and equal access to services and support. I can confirm today—I know that this will be of interest to many hon. Members—that the taskforce will publish its interim findings shortly, with a final report expected after the summer recess. The interim report will helpfully focus on recommendations that support a needs-based approach, beyond just the health system, in which people can access support based on their needs, not their diagnosis. The report will also set out recommendations for support to be provided beyond medication, and by healthcare professionals other than specialists.

Secondly, NHS England recently published an ADHD data improvement plan to inform future service planning, and on 29 May it published management data on ADHD waiting lists. Thirdly, it has been capturing examples from ICBs that are trialling innovative ways of delivering ADHD services and using that information to support systems to tackle waiting lists and provide support.

Fourthly, as part of the Government’s five long-term missions, we have launched the 10-year plan to deliver the three big shifts that our NHS needs to be fit for the future: from hospital to community, from analogue to digital, and from sickness to prevention. All those shifts are relevant to supporting people in all parts of the country with a range of conditions such as ADHD.

Fifthly, we are supporting innovation. Earlier this year, at a parliamentary event, many of us will have met innovators who are supported by NHS partners. I heard about the QbTest technology that complements the knowledge and skills of clinicians as part of the ADHD assessment process. I understand that 70% of NHS children’s ADHD services already use that technology, and the evidence suggests that it has a positive impact in making the assessment process swifter and simpler.

[Freddie van Mierlo]

Will the Minister comment on the fact that in Oxfordshire there is no commissioned service for the specialist reviews that NICE requires annually as a condition of being on the powerful medication? How can it be that someone can be started on medication but have no route to continue on it because they cannot get their annual review?

[Karin Smyth]

I am not aware of the detail of the pathway in the hon. Member’s ICB. I suggest that that is a matter for him to discuss with the ICB, which will have heard his question about how it is delivering those services on the ground.

Members raised issues around shared care agreements and the difficulties that people with ADHD are experiencing in accessing medication through such agreements, particularly when they have received a diagnosis through a private provider. It is the responsibility of secondary care specialists such as consultants, rather than GPs, to initiate treatment of ADHD. However, sometimes a shared care agreement, in which the GP takes over monthly prescriptions and routine monitoring once the patient is happy with their medication and dosage, can be put in place. The General Medical Council, which regulates and sets standards for doctors in the UK, has issued guidance to help GPs decide whether to accept shared care responsibilities for any condition. NHS clinicians need to be content that any prescriptions or referrals for treatment for any condition are clinically appropriate. All shared care arrangements are voluntary, so even where arrangements are in place, practices can decline shared care requests on clinical or capacity grounds.

If I may, Ms Jardine, I will take the opportunity to update the House on the supply of medicines, which has also been raised by colleagues; I understand that it was raised at business questions recently, too. The Government recognise the difficulties that some people have experienced with accessing ADHD medication due to medicine supplies. We know how worrying and frustrating those shortages are for patients and families. I am pleased to say that we have resolved many of the outstanding issues affecting the supply of lisdexamfetamine, atomoxetine capsules, atomoxetine oral solution and guanfacine prolonged release tablets. However, some specific manufacturers continue to have issues with methylphenidate.

We continue to work with manufacturers to resolve remaining issues. In fact, I met the medicine supply team this morning, as I do very regularly, to make sure we are on top of these issues as much as we can be. The team is working hard to make sure that the situation improves. Where issues remain, we are directing suppliers to secure additional stocks, expedite deliveries where possible, and review plans to support continued growth in demand for the short and long term. We have worked with specialist clinicians during this time to provide comprehensive guidance to healthcare professionals where there is a disruption to supply. We keep the Specialist Pharmacy Service website up to date with the latest availability of ADHD medicines. I commend it to people listening to the debate and to hon. Members. It also provides comprehensive guidance on switching to alternative treatments, supporting clinicians to make informed choices with their patients.

I can assure colleagues that, as the Minister responsible for medicine supply, I will instruct officials to keep a close eye on this issue, so we do not see any of the progress we have made undone. I plan to hold an event, hopefully in Parliament and possibly in the autumn, to keep updating hon. Members on this issue, because I know it is one that concerns us all.

In closing, I want to address the young people who may be watching or tuning into the debate at home. I know it is tough for many neurodivergent kids today. You might be stuck on a waiting list, suffering at school or struggling to find your medicine. We really do care about this. We are trying to get to grips with some of the problems we found when we came into office, and I hope you will start to feel that progress within the next few years.

Oral Answers to Questions

Debate between Freddie van Mierlo and Karin Smyth

Tuesday 6th May 2025 Commons Chamber

[Karin Smyth]

I absolutely join my hon. Friend in recognising the importance of supporting women’s health throughout pregnancy and into parenthood on Maternal Mental Health Day. We are committed to improving the support available, and it will form an important part of our 10-year plan. We are investing £126 million in family hubs and Start for Life services, to support parents from pregnancy to their child’s early childhood, and we will continue to work with her on this.

[Freddie van Mierlo (Henley and Thame) (LD)]

I thank the hon. Member for Aylesbury (Laura Kyrke-Smith) for her question and the Minister for her answer. I am delighted to be forming the all-party parliamentary group for fatherhood. Will the Minister outline the steps that she will take to improve perinatal mental health for fathers?

[Karin Smyth]

I congratulate the hon. Member on taking forward that work. The Minister responsible will be happy to continue to work with him in any way possible to support that work on this important aspect of parenthood.