In the summer of 2017, Duchenne UK launched its first patient survey, to better understand patient views on accessing clinical trials and research. The survey was entitled, “A questionnaire to understand Duchenne patient and caregiver perspectives on Duchenne clinical trials”.

The survey is part of our community engagement work to better understand what patients and caregivers want from care, research and clinical trials.

The aim is to help the Duchenne community to better understand the current clinical trial recruitment process, and to see where we can make improvements to create a fair and equitable trial recruitment process. The survey also aligns with Duchenne UK’s broader mission and the DMD Hub to ensure that all patients with Duchenne have access to clinical trial opportunities.

Sejal Thakrar present the survey results here:

Of the 217 responses received, the survey highlighted a number of challenges facing patients who want to access clinical trials: 

• 91% of patients surveyed said they DID want to take part in research.
• 50% of those who wanted to, couldn’t because they didn’t meet the inclusion criteria
• 68% of patients had never been given an explanation of how to access research opportunities. Instead they use social media to find out about studies. Patients were most likely to get onto a trial through persistent emailing of clinicians and trial co-ordinators.
• 29% had actively tried to enrol on a trial but were not able to. The survey highlighted the sense of desperation felt by patients and caregivers who were unable to access research: “absolutely devastated”, hopeless and a feeling that we are missing out. Disappointed with an unfair system.
• More than 50% said they would travel more than 5 hours to access a clinical trial. However, the cost of participation and burden of travel were also highlighted as the main barriers to taking part in research.
• 71% of those surveyed were registered on Action Duchenne’s registry. 77% of those never or rarely updated their information and 90% did not receive reminders to update details.

Duchenne UK is committed to addressing many of the concerns raised by patients in this survey, especially around the issues of communications and trial recruitment, through the setting up of the DMD Hub.

The DMD Hub is a partnership between Duchenne UK and the two centres of excellence in the UK, Newcastle and Great Ormond Street. Its aim is to address the lack of clinical trial capacity in the UK and is working with key stakeholders to build on existing expertise and create a network of clinical trial sites, which fits into Duchenne UK’s broader mission that all patients with Duchenne are given the opportunity to take part in research.

In particular the Hub will work to improve communication, increase the opportunity for patients to take part in research by increasing the  number of sites able to take on interventional trials, and develop the DMD Hub website with a patient portal on up to date clinical trial information.

Duchenne UK is delighted that Emma Heslop is in post as Hub Manager.

Thank you to all those who participated in helping to put together the survey and all those who took the time to complete the survey. ?

This survey was designed by Alex Johnson from Duchenne UK and Sejal Thakrar from Smile with Shiv in consultation with our fellow Duchenne Charities, Action Duchenne, Alex’s Wish, Duchenne Now, DMD Pathfinders, The Duchenne Research Fund, Harrison’s Fund and Muscular Dystrophy UK, patients and caregivers from across the Duchenne community, Emma Heslop from the Duchenne Hub, Industry and healthcare professionals.

The results have been analysed and Sejal Thakrar has created a survey report which you can download here.

ENDS   -

NOTES TO EDITORS

About Duchenne Muscular Dystrophy (DMD)

• There are around 2,500 boys affected by DMD in the UK and around 300,000 worldwide. It is classified as a rare disease
• Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood, almost exclusively affecting boys
• Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. There is no treatment or cure

About Duchenne UK

• Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD). Duchenne UK has raised more than £5.5million in five years, and spent or committed £5million to DMD research projects – more than any other organization
• Duchenne UK is leading the drive towards the efficient repurposing of existing medicines to bring new treatments for DMD
• Duchenne UK believes that every child with Duchenne should be given the opportunity to access a clinical trial. Duchenne UK created the DMD Hub in 2016 to expand and develop the infrastructure for DMD trials in the UK and accelerate access to clinical studies for those who are interested in participating
• The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell

Visit duchenneuk.org for more information or follow @DuchenneUK on Twitter.

Contact or more information and interview requests:

Molly Hunt, Communications Manager: [email protected]