Accelerating drug development
Duchenne UK and the DMD Hub Gene Therapy Meeting: Summary Report
20th December 2019
Summary ReportIntroduction: Gene therapy offers a potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy. A number of companies are now testing their approaches in the clinic. Currently these trials are taking place in the US. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be brought to the UK. The UK has a wealth of knowledge and experience in running DMD trials and gene therapy trials in other indications. However, challenges do exist.The meeting was important and timely, and looked in valuable detail at some of the hurdles and barriers that exist, and ways to overcome them so that we can make the UK fully ready for gene therapy trials in Duchenne Muscular Dystrophy. On the 14th November 2019, the DMD Hub team brought together 100 stakeholders, including leading UK neuromuscular centres, patient advocacy groups, national and local pharmacy representatives, colleagues from NIHR, industry representatives with experience of running gene therapy trials in neuromuscular disease, industry representatives currently setting up gene therapy trials in DMD, NICE and NHSE representatives as well as government funded organisations that are working to facilitate gene therapy. Currently, there are several trials underway in the US, some of which are planning to come to the UK in 2020. The meeting was aimed at ensuring the UK is aware of and as prepared as possible for the barriers.For more information on gene therapy, please click here: https://www.duchenneuk.org/gene-therapy-overview.There will be a more detailed report to follow in 2020 but this gives a short summary of the meeting and key actions moving forward, including providing an educational programme for hospital staff and patients, as well as engaging with approval bodies early in the process.The day was split into several sections:
- The shared vision for gene therapy in the UK
- The barriers to its delivery
- The state of readiness in the UK, particularly in DMD Hub Sites
- The pathway to accessing the therapy from the NHS
- The potential avenues for collaboration
- Coordinating recruitment of patients by agreeing a model for fair and equitable recruitment.
- Developing a model for delivering DMD Gene therapy trials, particularly how to balance between gene therapy and other ongoing trials.
- Creating guidance documents and linking existing resources through the DMD Hub Toolkit.
- Collaborating with existing initiatives like Catapult, NAATTCC and UK Research Directors.
- Engaging with commissioners much earlier in the process to speed up approval processes.
- Providing education on gene therapy to both staff and patients.
- Improving understanding of patient preferences.
- Addressing issues and barriers faced by industry, such as delayed approval processes.
Published on 20 December 2019Share this articleCategories Accelerating drug development DMD research
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