The United Kingdom is considering a new regulatory pathway that could affect how future treatments for myotonic dystrophy are reviewed and accessed. The Medicines and Healthcare products Regulatory Agency, or MHRA, is seeking public feedback by July 30, 2026 on its draft Rare Disease Therapies Regulatory Framework consultation, which could allow some promising treatments to be reviewed as evidence is still being collected, rather than waiting until all evidence is complete. For the DM community, this is an important opportunity to explain why flexibility around the proposed rarity threshold, safety monitoring, and meaningful patient input must be part of future treatment access decisions.
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What Is Being Proposed?
The draft Rare Disease Therapies Regulatory Framework would create a new pathway called an Investigational Marketing Authorisation, or IMA. This could allow controlled early access to a treatment while additional clinical and real-world evidence is collected and safety continues to be monitored over time.
For rare and complex conditions, this kind of approach may help address challenges that can make traditional treatment development difficult, including smaller patient populations, limited natural history data, and barriers to conducting standard clinical trials.
MHRA’s original GOV.UK announcement, “Landmark new plans bring treatments for rare diseases a step closer,” provides additional background on the proposal and specifically mentions myotonic dystrophy.
Why This Matters for DM
Myotonic dystrophy is specifically mentioned in the GOV.UK announcement about this initiative, which recognizes that DM can affect movement, breathing, heart function, and daily life.
Although myotonic dystrophy is specifically mentioned in the initiative, its reported prevalence of up to 1 in 2,100 could be perceived as not fitting within the proposed “around 1 in 50,000” rarity threshold. This is why flexibility, and input from patients and carers, are so important. Myotonic dystrophy is highly variable, with major differences in age of onset, symptoms, severity, progression and treatment-relevant subgroups. In practice, research and future treatment decisions already focus on much smaller groups of patients with distinct clinical needs. The threshold should therefore be applied flexibly, so that people with clear unmet need are not unintentionally left out.
For the DM community, this is one of the most important issues to raise in public comments. A fixed threshold may not fully reflect the reality of myotonic dystrophy or the way future treatments may be studied, reviewed, and used. People living with DM may have different symptoms, ages of onset, levels of severity, rates of progression, and treatment needs. Some future therapies may also be designed for specific groups within the broader DM community, not everyone living with DM at once.
A flexible approach would help ensure that people with significant unmet need are not excluded based only on broad prevalence numbers.
Why Your Feedback Matters
People living with DM, families, and caregivers can help decision-makers understand what myotonic dystrophy is really like and what fair access to future treatments should include.
Community feedback can help explain:
- Why flexibility around the proposed “around 1 in 50,000” threshold is needed for DM.
- How much DM can vary from person to person.
- Why smaller groups within the DM community may need to be considered.
- How early access to new medicines should be managed.
- What ongoing safety monitoring should include.
- What protections are needed if access is delayed, changed, restricted, or withdrawn.
- Why treatment decisions should reflect daily life, family impact, disease burden, and outcomes that are meaningful to patients.
Community input can also help show why MHRA and the National Institute for Health and Care Excellence, or NICE, need to work together to avoid unnecessary delays between regulatory review and real patient access. The proposed pathway could support earlier review, but access for NHS patients would still depend on NICE approval.
How to Comment
The consultation is open to patients, families, caregivers, clinicians, researchers, patient organizations, members of the public, and other stakeholders. You do not need to be a policy expert to provide meaningful feedback.
MDF encourages members of the DM community, especially those in the UK, to review the MHRA consultation landing page and consider completing the public comment survey by July 30, 2026.
