DMD Hub Gene Therapy Stakeholder Meeting

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Date and Time

Location

Location

The Core

Bath Lane

Newcastle upon Tyne

NE4 5TF

United Kingdom

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Event description
Engaging the key stakeholders in the DMD field to urgently discuss and address the issues we face in bringing Gene Therapy trials to the UK.

About this Event

The aim of this meeting is to bring together the key stakeholders in the DMD field to urgently discuss and address the issues we face in bringing Gene Therapy trials to sites in the UK for the benefit of the Duchenne Community.

Participants will include key opinion leaders, pharmacy representatives and staff who will be delivering Gene Therapy trials at UK sites, patient representatives, industry representatives with experience of running gene therapy trials, industry representatives planning DMD trials in the UK, commissioners and representatives from existing initiatives addressing the issues that are currently being faced (such as The Cell and Gene Therapy Catapult, NA-ATTC, NIHR and TREAT-NMD).

You are invited to attend and actively contribute as the day is planned to be a mix of presentations and interactive discussion.

Agenda Outline

Welcome and Aims of the Day

Session 1: Shared vision and hopes for Gene Therapy in the UK

Clinical Perspective - DMD

DMD Patient Perspective

Industry Perspective

Break

Session 2: Overview of the issues: what are the barriers in delivering the next generation of medicine in the UK?

What can we learn from other diseases? Experiences from the Avexis SMA Gene Therapy trial

What issues are Industry facing? Perspectives, plans and experiences in DMD Gene Therapy trials

Local site-specific and Genetic Modification Safety Committee common issues - Pharmacy

Panel discussion: Industry and Sites

Lunch

Session 3: DMD-Hub sites: NHS Institutional Readiness

Institutional Readiness – feedback from DMD Hub survey

Institutional Readiness in the NHS

Panel discussion: NHS

Session 4: Sustainable Access

Health Economics and Reimbursement Barriers

Navigating the pathway to access, a commissioners perspective (NHSE)

Navigating the pathway to access, a commissioners perspective (NICE)

Panel discussion: Access

Break

Session 5: Existing initiatives to help address the issues – and areas for collaboration

The Cell and Gene Therapy Catapult

Northern Alliance Advanced Therapies Treatment Centre

TREAT-NMD Post-Marketing Surveillance Registry

Session 6: Prioritisation of barriers – Industry, NHS, Regulators and Patients

Discussion Session: What are the top 3 barriers in the UK?

Session 7: DMD Hub call to action

Proposed models of delivery

Where can the DMD Hub add value? Education, reimbursement, commissioning, central coordination, collaboration

Panel discussion

Session 8: Summary and Next Steps

Panel discussion – solutions and next steps

Date and Time

Location

The Core

Bath Lane

Newcastle upon Tyne

NE4 5TF

United Kingdom

View Map

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