Agenda Archive

Registration and refreshments

Introducing systems to maintain ambient temperatures as well as cool or cold chain
Assessing the potential risk of a heatwave to pharma products and how to mitigate against this
Clear communication and labelling to minimise risk to products once they have reached the patient

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

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)

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

Incorporating digitalization and new technologies into your clinical trials
Collaborative platforms: improving communication and efficiency among stakeholders
Success stories, transformative cases and key takeaways

The landscape of clinical trials is undergoing a transformative shift as digital technologies continue to permeate every aspect of the healthcare industry
One potential transformation could be the replacement of traditional paper-based relabelling in clinical trials with digital display labels
The integration of digital display labels into clinical trials represents a significant leap forward in the industry. As this technology matures and becomes more widely adopted, it could impact the way clinical trials are conducted and managed especially in the areas of remote monitoring, adaptive trials, and patient engagement
In this session, we will explore the challenges associated with traditional relabelling processes in clinical trials, the advantages of adopting digital display labels, and the potential impact on the future of healthcare research