Archives: Agenda

PANEL DISCUSSION: Designing Oncology Trials That Work for Every Patient

  • Tackling barriers that limit access and participation for under-represented patient groups.
  • Ensuring cultural relevance, language accessibility and equitable site selection.
  • Embedding PROs and patient insights to shape inclusive eligibility and trial procedures.
  • Measuring what matters: evaluating the impact of patient-centred designs on recruitment and retention.

CASE STUDY: Gene Therapy in Action: Advancing a Clinical Case Study in Childhood Dementia

This session will explore the translational journey of a novel gene therapy targeting childhood dementia, from early research through to clinical trial readiness. Using a real-world case study, it will highlight the scientific, regulatory, and operational challenges involved in bringing a cutting-edge therapy to patients, and what this means for outsourcing partners across the UK & Ireland.

 

  • Overview of the gene therapy approach and mechanism of action
  • Case study: from discovery to clinical trial planning
  • Key challenges in manufacturing, scale-up, and delivery
  • Regulatory considerations and working with UK bodies (e.g. MHRA, HRA)
  • The role of outsourcing partners in enabling trial readiness
  • Timelines, funding pressures, and “race against time” dynamics
  • Future outlook for gene therapies in rare paediatric diseases