The Problem

In 2015, Emily and Alex (co-founders of Duchenne UK) learnt that clinical trials were being turned down in the UK because of a lack of capacity; there simply weren’t enough relevant doctors, nurses, physiotherapists trained to conduct clinical trials for DMD.

The Solution

So, in July 2015, we co-hosted a workshop in Newcastle with Professor Kate Bushby and the global neuromuscular network, Treat-NMD, to resolve this issue of clinical trial capacity. From it, emerged the Newcastle Plan. We held the workshop in July. By September we’d identified four centres of excellence that needed emergency funding to enable them to conduct trials. We collaborated with other UK patients groups in to raise £1.2 million and fund 16 critical posts. Duchenne UK contributed £542,121 to this project.

We also have a 5-year plan to bring more sites on stream. And we’re delighted to be working with NIHR who are considering a pump-priming model where charities fund posts, and are reimbursed when money comes through from industry for those trials.

This is the kind of innovation – putting patients at the heart by responding to what they are saying are the critical issues – that will deliver for patients and for science - and and for the NHS.

We presented the results of our plan to the European Conference on Rare Diseases and Orphan Products in Edinburgh, May 2016. You can view the poster here  

Many congratulations on your fundraising efforts – the extra staffing positions will make a huge difference to the ability of some of the centres being able to participate in clinical trials. A great example of collaboration, to deliver options for participating in clinical research for DMD! 

Christine Medhurst

Senior Investigator Site Development Lead, Pfizer Limited

The posts being funded are:

NEWCASTLE

Clinical Research Associate

Research Physiotherapist

Clinical Research Nurse

Clerical Assistant

ALDERHEY

Speciality Doctor

Research Physiotherapist

Clinical Trial Co-ordinator

GREAT ORMOND STREET (in collaboration with the GOSH charity and the NHS Trust)

Clinical Research Fellow (2)

Physiotherapy Assistant

Research Physiotherapist (2)

Clinical Trial Co-ordinator

Data Manger - fully funded by GOSH

Professor of Neuromuscular

Senior Lecturer - Neuromuscular

The UK charities that contributed towards the total £1.2m funding were:

Duchenne UK, Muscular Dystrophy UK, Harrison's Fund, Duchenne Now, Action Duchenne & Alex's Wish.