What we do for Health Technology Appraisals (HTAs)
We make HTA decisions faster and fairer—so families don’t face unnecessary delays.
Our approach
At Duchenne UK, we know that developing new treatments for Duchenne muscular dystrophy is only half the battle.
For people with Duchenne to benefit, those treatments must be approved and made available through the NHS. That process is called health technology appraisal (HTA) - and we play an active role in making it happen.
We take part as official stakeholders in HTA processes. This means we submit evidence, share patient perspectives, and speak directly in committee meetings to make sure the voices of families are heard. We explain what life with Duchenne is really like and why access to new treatments matters.
Through HERCULES, we work with people with Duchenne, researchers, and pharmaceutical companies to create tools that strengthen the evidence base for Duchenne.
Impact
Developing models that show the real impact of treatments on patients and families
Data
Collecting data on quality of life and long-term outcomes
Insights
Sharing insights that help health systems understand the true value of care
By doing this, we make HTA decisions faster and fairer—so families don’t face unnecessary delays.
It matters because every day counts for people living with Duchenne.
By being part of the HTA process, we help ensure that new treatments reach patients as quickly as possible.
We’re not just advocating.
We’re shaping the system to work better for rare diseases.
