Four treatments approved, one uncomfortable truth
The challenge of converting scientific progress into real-world access
Dr Charlotte Roy
Life Sciences Policy and Influencing Manager, Muscular Dystrophy UK
One month. Three conditions. Four treatments. An incredible milestone for the neuromuscular community – one that would have seemed unimaginable just ten years ago.
These treatments represent major advances in science, especially in conditions that worsen over time and where few treatment options exist. But in rare conditions where evidence is limited, translating that scientific progress into real-world access is rarely straightforward.
For years, the rare disease community has described the challenges in how the National Institute for Health and Care Excellence (NICE) and the wider system recognise the value of new treatments.
At Muscular Dystrophy UK, we have seen this first-hand: long, complex appraisals that take years to reach a decision, multiple rounds of committee meetings, and decision-making that struggles to recognise the value of treatments that improve quality of life, reduce symptoms or slow progression. All while people wait for access to treatments that could make a meaningful difference to their lives.
A system under strain
These four recent approvals show that NICE can reach decisions that both enable access for people with rare conditions and work for the NHS. Across all four appraisals, the processes were undeniably difficult, but NICE demonstrated a willingness to work with stakeholders to find a path to a positive outcome.
However, these outcomes were only achieved by pushing the system to its limits.
In each case, reaching a positive decision required pulling on every lever the system had to offer. In some cases, it took three committee meetings; in others, lengthy and restrictive managed access programmes or extended commercial discussions. In one instance, approval was ultimately reached by building on learnings from previous rejections and appeals. But in every case, decisions relied on the use of every available flexibility – every inch of headroom – within the NICE manual.
Above all, it depended on persistence and resilience. Persistence from people living with these conditions and the uncertainty they face, while continuing to push for access; from clinicians working within a stretched NHS, taking time away from busy services to advocate for these treatments; and from patient organisations navigating a complex system.
So, when the rare condition community says the system is challenging, these approvals make that clear. The system isn’t working for rare conditions.
Can the system deliver on the UK’s life sciences ambition?
As the UK looks to meet its ambition of becoming Europe’s leading life sciences hub by 2030, the limitations of the current system cannot be ignored.
Recent policy developments show what is possible. Changes such as the updated cost-effectiveness threshold have played a role in enabling access in one of these appraisals. But even with this progress, the decisions still depended on making full use of the system’s flexibilities. This is not a model that can be relied on at scale.
There is also a risk that upcoming changes could further constrain an already stretched system. The introduction of the new EQ-5D-5L value set, expected this year, may disadvantage treatments that significantly improve quality of life without extending it – outcomes that are particularly important for people living with rare conditions. While NICE has identified mitigations, these again depend on flexibilities that are already under considerable strain.
A breakthrough – and a warning
These four approvals show what is possible when NICE, NHS England, clinicians, industry, people with lived experience and patient organisations come together with a shared commitment to finding a way forward.
But they also highlight the level of effort required to get there.
If the UK government is to deliver on its life sciences ambitions, it needs a system that is better equipped to reflect the realities of rare conditions. One that recognises the true value of treatments – including their impact on people affected, their families and carers, healthcare systems, and wider societal productivity.
And one that does not treat uncertainty as a barrier to access, but works with it to enable timely, equitable access to the science that can change lives.
The challenge now is how this system is built, ensuring that the perspectives of people with lived experience are not just represented, but inform how value and access are defined and applied in practice.
Because for people living with neuromuscular conditions, time does not stand still while decisions are made.
This is a guest blog. Its author is responsible for content within it, which does not necessarily reflect the opinions or positions of BIA.
