Earlier this year, the Duchenne Children’s Trust and Joining Jack identified a chronic lack of capacity to conduct clinical trials in the UK. It’s an issue that we felt was absolutely crucial, especially now. Because treatments for DMD feel tantalisingly close. The drug development pipeline is exciting. More than 20 drugs now in development with 28 studies now ongoing in the UK. So imagine our horror when we learned that the UK is now turning away these potentially life changing clinical trials, because we lack the capacity – the relevant doctors, nurses, physiotherapists trained to conduct clinical trials for DMD.

So, we approached Treat-NMD, and asked them to host a workshop to get a measure of the current situation. The meeting was held in Newcastle in July, and our founder, Emily Crossley, made the opening presentation on behalf of all patient organisations in the UK. The event was extremely well attended, with biotech, drug companies, clinicians, trial nurses, physiotherapists and the NIHR.

Overall, the meeting concluded that the UK is a true centre of excellence for DMD trials, and must continue to remain one of the key “go to” countries for clinical trials in DMD. A huge willingness was clearly demonstrated from everyone to increase capacity and maintain and improve quality. You can read the full report here.

Three phases of development were discussed, to be taken forward by a working group derived from the meeting participants. Emily Crossley suggested that we adopt a one, two and five year plan with the ultimate aim being to to ensure that all patients with DMD, children and adults, have access to clinical research opportunities. 

A smaller group will form who will oversee how the plan takes shape over its lifetime. This group will look at a summary of the current situation, will consider a map of resources and look to produce a publication which will carry more weight in the long term. 

The patient organisations will be meeting in September to keep up momentum and discuss next steps.