Archives: Agenda

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• Considering ways to boost recruitment through investigator meetings and recruitment workshops
• Communicating effectively and working together with sites to assist in recruitment
• Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

• Clinical study design and key outcomes in sporadic ALS patients
• Mechanistic insights: targeting mitochondrial dysfunction as a therapeutic strategy
• Lessons learned and implications for future rare disease trials

• Creating vendor selection criteria and processes
• Establishing shared goals and KPIs to foster trust and alignment
• Managing expectations on timelines, budgets and deliverables
• Evaluating vendor capability in finding and supporting rare disease patients
• Monitoring vendor performance, governance and communication plans

• Think ahead and move beyond protocol approval to consider downstream data dissemination in peer-reviewed publications, at congresses, for medical education and MSL scientific exchange.
• Understand how protocol design and statistical planning determine the data available for scientific communications by maximizing the value of limited datasets to avoid missed opportunities.
• Identify opportunities during study design to generate data that support meaningful Scientific Communications
• Explore how early collaboration between Clinical Development and Scientific Communications can reduce future data gaps and maximize the scientific value of limited rare disease datasets.

• Sharing insights on how to use AI to bring drugs to rare disease patients
• Running through step by step processes including pre IND, FDA work, organising and conducting trial
• Discussing challenges, successes and real-world experience

Despite advances in therapies, decentralized trials, digital tools, and recruitment, many patients never reach a point where clinical trial participation is a realistic option because they must navigate complex healthcare systems with little guidance. This burden affects all patients and caregivers, but is often amplified in underserved and historically underrepresented communities, contributing to trial populations that do not reflect those most affected by disease. Meanwhile, many frontline and allied healthcare professionals interact with patients throughout this journey but receive little training in healthcare navigation literacy or practical coaching approaches.

This interactive session highlights healthcare navigation literacy as an upstream workforce opportunity to improve preparedness for specialty care and clinical research. Participants will explore how equipping patient-facing professionals with simple navigation-coaching strategies can help patients ask informed questions, access resources, and recognize research opportunities when appropriate. Attendees will leave with actionable, workflow-friendly strategies to strengthen patient engagement and support more equitable pathways to clinical research.