AI for impact – transforming clinical trial study design and execution

Harnessing the convergence of high-fidelity data with advanced AI is now essential for de-risking clinical trials and accelerating therapies to patients. What if the distance between a draft protocol and a validated study execution plan was reduced to a single, unified simulation?

Join Medidata’s Eleanor McLaurin for a deep dive into the future of holistic trial design and planning. Learn how advanced modelling and simulation capabilities within a central workspace can revolutionise trial operations. This session will illustrate Medidata’s strategy for optimizing protocol design, predicting trial outcomes, and achieving greater clinical and operational success in the next generation of drug development.

 

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