Archives: Agenda
Are you listening? How to retain, not just engage, stakeholders in clinical trials and people as partners
- Effective communication with target patients/families/care professionals and RETAINING them
- How inclusion of AI looks like in this communication, trust building, meeting needs and retaining engagement, from:
- Care researchers’ perspective
- Care Professionals’ perspective, and most importantly,
- Caregivers’ perspective (lived experience and real-world-evidence) in the context of “rare diseases”
Registration and refreshments
Cold Is Easy. Control Is Not. How smart logistics improve PCM benefits in pharmaceutical shipments
- PCMs provide predictable thermal buffering
- When and why temperature control fails in pharma logistics
- Dry ice is powerful – but not trivial
- How to overcome these failures best – (where) can AI help?
- Logistics tools improving PCM benefits
Navigating the future of clinical trial logistics
Insights from the DHL logistics trend radar
- Key logistics trends impacting life sciences & healthcare
- Customer insights on adoption and priorities: LTR ECHO
- What’s next in the DHL logistics trend radar
CLOSING PANEL DISCUSSION: How AI is powering a new era of cancer drug discovery and development
- Moving from basic functionalities to more meaningful impact and examples of this in practice
- When AI is progressing so quickly, how will regulation evolve to match this speed?
- When is AI not the answer?
- How AI has revolutionised innovative ways of collecting data, biosimulation, and early disease diagnosis for oncology trials
Moderator: Asiyah Nawab, Senior Healthcare Analyst (Immunology Team), GlobalData
THIS SESSION WILL BE IN THE ONCOLOGY CONFERENCE ROOM
Afternoon refreshments, networking and prize draw
Clinical trials in rare immunology disorders
- Overview of rare diseases in Immunology
- Pharma focus on rare diseases in Immunology for drug development – where do industry interests lie?
- Challenges in rare disease trials in immunology
- Case Study – Sarepta’s Elevidys in Duchenne Muscular Dystrophy (DMD)
The role of patient research NGOs in drug development – illustrated by the Children’s Tumor Foundation’s leadership role in the EU PEARL project
- Patient driven infrastructure – NF patient registry, NF Preclinical Hub, NF Clinic Network, NF Data Portal – to accelerate trials and insights
- CTF served as IMI research partner on EU PEARL NF1; CTF Europe launched to expand platform trials
- EU PEARL NF1: adaptive platform basket across PN, cNF, LGG/OPG; observational entry → randomization; add/drop arms, early go/ no go signals
- Addresses EMA orphan challenges via clear estimands and central oversight; efficient, de risked paediatric evidence
- NGO impact: contributed to first NF approvals; 60+ US trials; now scaling European execution