Access to treatments

Duchenne UK's general election manifesto

The upcoming General Election on 4 July 2024 is a chance to reflect on our health system, and to ask what we want to see a future government do in a new parliament. Dozens of MPs won’t be standing for re-election, and there will be many new faces in Westminster eager to tackle the big issues. What would we ask for, if we had the chance to sit down with the next Prime Minister on their first day in Downing Street?


Firstly, we would tell the Prime Minister to trust what patients say, and to let patients and their communities speak for themselves. Too often mere lip service is paid to the goal of ‘putting the patient first’, and listening exercises can be shelved or ignored. It is also easy for those in charge to fall into the trap that there is a single patient voice, which represents every person and community which suffers and grieves. In this manifesto, we suggest ways a new Prime Minister could hear from many patient perspectives, and bring what patients say, in their own words, into government.

Secondly, we would urge the Prime Minister to be prepared to invest in the nation’s health with new and promising medicines. While every party is interested in the NHS and our health system, what they talk about is beds, waiting lists and staff. This is very important, but we think a key part of the puzzle is being missed: medicines. The UK falls behind the pack when it comes to medicine spend. Our fantastic NHS workers can only do so much if they have to rely on old treatments. British patients should not be at the back of the queue for the newest, most promising treatments.

Thirdly, we would talk to the new Prime Minister about the modern reality of getting a new treatment for a disease like DMD approved for use in the NHS. NICE has recently reformed how they assess medicines, putting a greater emphasis on how rare the disease is, and giving more flexibility if they are rare. Shockingly, diseases like DMD – which has only around 2,500 patients in the UK – are too common to be considered for that flexibility. But what makes DMD tricky is not because it’s rare, it’s because it’s complex. We urgently need those in power to cultivate a system which can account for complexity.

And finally, after talking to the new Prime Minister about how they could include the patient voice across government, how they should address the glaring lack of investment in new medicines, and how they could create a health system which can recognise complexity, we would turn to the standard of care patients receive. Patients and clinicians are experts in their field, and by working together – like we have we DMD Care UK – the best standards of care can be found. But even when that happens, those recommendations aren’t always adopted universally. We would urge a new Prime Minister to make encouraging best practice in the NHS one of their priorities.

Read on if you’d like to know more about how we think our four priorities could be achieved.


For years, discussions on health matters have been dominated by the aspiration to ‘put the patient voice’ at the heart of everything. This was welcome when the perspective of patients was mostly excluded from the debate, but if we truly want to make the patient voice central, we need to turn this paradigm on its head.

Rather than bringing the ‘patient voice’ in as a nice-to-have addition, with the views of the health system, clinicians, companies and all the other stakeholders baked in from the start, the views of patients should be put on an equal footing, with an equal weight.

This will not happen overnight, and requires a sea change in how we think about health and seek solutions to the challenges our society faces. A big problem is that currently the patient voice is fragmented and divided across the health system, with no clear leadership or advocate for their interests.

The Government is represented by Ministers in the Department for Health and Social Care. Industry has large and influential trade bodies such as the Association of the British Pharmaceutical Industry (ABPI) and British Generic Manufacturers Association (BGMA). Our health system has CEOs in the NHS and regulators like NICE. But patients do not have a single figure or institution which can represent all, and those which come close exist outside the health system – where the people in power can choose not to engage with them.

That is why we’re calling for the establishment of a Patients’ Commissioner, which we believe will be an affordable and effective way of starting this sea change through a method which has a successful precedent.

In the justice system, victims of crime had a similar problem as patients do in the health system. The system was meant to serve them, but they were small, individual voices drowned out by larger players. In 2010, the first Commissioner for Victims and Witnesses was appointed by the Justice Secretary. The Commissioner can make proposals directly to the Justice Secretary, make recommendations to authorities within their remit who must respond within 56 days, and lay a report before Parliament. They have a platform to contribute to legislation and an ability to intervene with those in power in formal ways that anyone outside the system cannot.

We believe a Patients’ Commissioner, like the existing Victims’ Commissioner, could do that for patients in the health system, fulfilling a different role to the existing Patient Safety Commissioner – who has a background as a GP, not a patient, and whose role came about to promote patient safety following the pelvic mesh scandal.

Beyond a Patients’ Commissioner, we’re also calling on the next Government to bring the Minister for Disabled People into Cabinet. This is a role which is often overlooked – and was even scrapped recently between December 2023 and April 2024. They are meant to have cross-government responsibility for disabled people, but they are placed in the Department for Work and Pensions, and are answerable to the Work and Pensions Secretary. This will inform, and we believe limit, their priorities for disabled people.

Bringing the Minister for Disabled People into cabinet would empower the person who holds that office to advocate for disabled people across government (not just in the Department for Work and Pensions) and bring the priorities of patients and disabled people to the highest levels of decision-making in the country. There is precedence for this: the Minister for Veterans’ Affairs attends cabinet, representing the interests of veterans across government, and signalling the importance the Prime Minister puts on the welfare of veterans. Disabled people deserve the same.

With this increased empowerment, we also believe the Minister for Disabled People needs greater support. That’s why we want the Office for Disability Issues (ODI) to be restored, with a remit to support the Minister and the Patients’ Commissioner, and help them implement the UN Convention on the Rights of Persons with Disabilities across government. The ODI was closed in 2019 and turned into the Disability Unit, which in turn is one part of the Equalities Hub. We believe this area deserves more attention and resources, and civil servants working on it need a greater capacity to support the Minister for Disabled People.

We believe these asks, based around the principle of letting patients speak for themselves, will be good first steps by the next government to improve the lives of disabled people and people living with rare diseases, and bring their experiences into the heart of government.


Much of the political debate is rightly focused on the NHS, and acute issues such as waiting lists and A&E delays. But an area which is completely overlooked is the shocking lack of investment in medicines.

The UK spends the least in the G7 (7 of the richest countries) on medicines, and we are below average in the OECD (38 developed nations). For every £100 in GDP, the UK spends 81p on pharmaceuticals, compared to £1.94 spent by Germany, and £1.66 spent by France.

You get what you pay for, and if our country is not prepared to pay for new and innovative treatments, then British patients will continue to be at the back of the queue. What makes it even more disappointing is that many of these treatments are trialled and researched here in the UK, where we have a fantastic life sciences industry. British patients play their part in developing these treatments, but then they’re made widely available in other countries who are prepared to invest in medicines first.

Duchenne UK is also calling on the next government to invest in mental health. It is appalling that families can be hit with a diagnosis for a rare disease like DMD, and receive no counselling or psychological support. This support should not only be made available at diagnosis, but also for when patients progress to each new stage of the disease.


Medicines are made available on the NHS by first being assessed by a regulator called NICE. NICE serves an important purpose we applaud: it ensures medicines are not only clinically effective, but cost-effective too, so public money is spent wisely. But we are concerned changes NICE made in 2022 to how medicines are assessed have raised the bar so high that any DMD treatment will struggle to be approved.

There are two routes a drug can be assessed by NICE. The first, called a Single Technology Appraisal (STA), is the standard way treatments are assessed for the general population. The second path allows for more flexibility – and is more forgiving on cost-effectiveness – and is called a Highly Specialised Technology (HST) evaluation.

DMD treatments used to qualify for HST evaluations as it is a rare disease, but the 2022 changes mean DMD is too common to qualify any more. Forcing treatments for a complex, life-changing disease like DMD – which has a significant paediatric population – down the same route as medicines for the general population is not appropriate. How can we expect the STA committee to be versed in such a broad range of conditions, and how can a price point deemed cost-effective for millions of people also make sense for our community of 2,500?

We understand why NICE wanted to clarify what can qualify for HST. But not only are NICE’s rarity criteria too restrictive, it doesn’t reflect what makes a treatment hard to assess. The prevalence of a disease isn’t the main barrier to assessing it, it’s the complexity of the disease, and we want NICE to reform their criteria for HST to reflect this.


The UK is fortunate to have some of the best DMD experts in the world, but not everyone can access this world-class care. There are variations, and some members of our community have been let down by their healthcare professionals.

To address this, Duchenne UK established the DMD Care UK project, an initiative to ensure everyone with DMD can access the best care, no matter where they live. It brought together expert clinicians and patients to agree and publish DMD standards of care. These recommendations are based on the internationally recognised care standards published in The Lancet Neurology, but tailored to the British health system, and reflecting the input of British patients.

DMD Care UK has already published recommendations in areas such as bone & endocrine, cardiac, and respiratory care. Each set of recommendations has been published in a journal, and endorsed by a relevant professional body – for example the cardiac care recommendations were published in BMJ Open Heart, and were endorsed by the British Cardiovascular Society.

It may seem obvious that our health system would want to adopt internationally recommended standards of care, which have been adapted by world leading experts to work in the NHS, for a rare disease the NHS may not have the resources to focus on in such detail themselves. But our experience has been far from that.

We are calling on the Government to create a mechanism for best practice for a given disease to be disseminated to and adopted by clinicians throughout the NHS. This could be through a NICE accreditation, guideline or quality standard, or an NHSE service specification.

We have the tools and guidance clinicians need to make the best decisions for our community, and we urge government and the health system to start a dialogue with us on how we can not only secure these changes for DMD, but do it in a way that the mechanism can be used by any rare disease area, and allows patients recourse if the care they are given doesn’t meet the recommended standards.

Published on 27 June 2024

Share this article