Archives: Agenda

• 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

• 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

• The new era of pharmaceutical compounds
• Regulatory landscape: From traditional IMPs to ATMPs
• Clinical operations: Managing an ATMP study, main challenges

• Hear about a pragmatic, data-driven framework developed and piloted at HDP to transform CRO selection from an ad-hoc, relationship-based process into an objective and reproducible model
• Move beyond defaulting to global CROs and instead identify the optimal partner profile for each trial type and region.
• Quantify performance, cost, and quality trade-offs using structured assessment tools and scoring matrices.
• Integrate input from clinical, regulatory, and finance stakeholders to ensure balanced decision-making.
• Embed e-clinical and operational system compatibility checks into the evaluation process to prevent integration failures downstream.
• Align sponsor and CRO incentives early—so that success metrics are shared, measurable, and sustainable.
• Faster onboarding, clearer accountability, and partnerships that foster trust rather than friction

• The Clinical Trial Bottleneck and how AI can break this cycle.
• AI as a Full-Lifecycle Catalyst: From protocol design to regulatory submission.
• Recruitment Reinvented: How AI-driven models and molecular matching slash screening times and cut screen failures.
• Smarter Trial Design
• Ethics in the Algorithm Era: Bias mitigation, federated learning for privacy, and transparency frameworks ensuring compliance and trust.
• Blueprint for Implementation: A pragmatic 4-phase roadmap – Foundation, Integration, Scale, Leadership – designed for operational success.
• Future Horizons: Digital twins, foundation models, and quantum computing shaping the next decade of oncology research.

• Changing how researchers select potential patients for better success rates
• How to improve drug efficacy and patient safety before it’s too late
• Back to square one: Differentiating with simpler, patient-centric protocols and optimised study design
• Avoiding disappointing results by implementing the right technology tools to advance your trial
• Lessons learned to mitigate trial failures

Moderator: Sumeet Ambarkhane, Chief Medical Officer, Pathios Therapeutics

• Evolving Regulatory Environment: Significant regulatory reforms across Latin American countries aiming to align with international standards (e.g., ICH guidelines)
• Opportunities Arising from Regulatory Changes: Faster approval timelines enabling quicker trial initiation
• Role of Vendors in Fostering Opportunities: Need for vendors to adapt to local regulatory nuances and evolving compliance requirements
• Turning to Latin America for a genetically diverse population, valuable for global clinical research, but also understanding the need for culturally sensitive patient engagement strategies to improve recruitment and retention