Building evidence about the disease and its impact
We are committed to making sure that everyone with Duchenne muscular dystrophy can access effective new treatments as soon as possible.
We know that developing treatments is only part of the challenge. For those treatments to reach patients, they must be approved, priced fairly, and reimbursed by health systems. That process depends on evidence—clear, robust data that shows the value of these treatments for people living with Duchenne.
Our approach
Duchenne is a rare condition, and that makes things harder. When the evidence is strong, decisions are faster and fairer. It means that new treatments can be priced and reimbursed in a way that reflects their value—without unnecessary delays for families who urgently need them.
Because there are fewer patients, there is often less data available to show how treatments work and what difference they make to quality of life. This can slow down decisions about pricing and access.
HERCULES
We’re changing that. Through projects like HERCULES, we work with patient groups, researchers, and industry partners to create tools and resources that strengthen the evidence base for Duchenne. This includes developing better ways to measure the impact of treatments, collecting real-world data, and creating models that help health systems understand the true cost and benefit of care. When the evidence is strong, decisions are faster and fairer. It means that new treatments can be priced and reimbursed in a way that reflects their value—without unnecessary delays for families who urgently need them.
Collaborating with Pharma
When health technology asssessment (HTA) decisions are based on robust, patient-centred evidence, treatments reach the market faster. That benefits families - and it benefits companies by reducing risk and uncertainty. Together, we can make sure innovation translates into access.
Collaborating with HTAs
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.
