Archives: Agenda

• What degree of oversight should you have over third party vendors?
• Risks and benefits of handing third party management to CROs
• Regulatory and quality considerations around outsourcing to third party vendors

Visit our exhibitors’ booths throughout the conference and collect stamps in order to enter our Prize Draw and be in for a chance of winning Apple devices or Amazon vouchers. The Prize Draw will take place in the Exhibition Hall, make sure you don’t miss out!

• An overview of recent US legislation around diversity in clinical trials
• Understanding requirements around DAP approval and submission: what should you include?
• Preparing documentation to demonstrate diversity in your clinical trial
• Experience with DAPs in view of the received FDA feedback

• Outline critical factors biotech investors evaluate before committing their resources, offering actionable insights to help startups stand out in a competitive market.
• Steps for biotech founders to differentiate, IP strategy and to attract funding
• Avoiding missteps in budgeting and resource allocation together with prioritization and careful planning.
• Storytelling about key milestones achieved aligned with operational activities with feasible exit strategy?
• Does the biotech investor bring strategic expertise beyond capital needs?
• Does the biotech have the appropriate team and expertise to execute?

• Optimizing study-start up for improved timelines and site performance
• Integrating patient input into protocol design and the impact of patient motivation assessments
• Case studies: patient-informed protocol design for successful outcomes

This presentation delves into dynamics of enrollment challenges and underscores the significance of fostering strong site relationships in rare diseases, which often prove instrumental in driving successful outcomes. Beginning with a compelling case study, we navigate key drivers influencing sponsor decision-making processes, creating innovative strategies that positively impact the study. Emphasizing the paramount importance of timelines, we discuss how sponsor decisions are strategically molded to align with project deadlines. Through the lens of the “one team, one approach” philosophy, we highlight collaborative strategies that surmounted our enrollment hurdles effectively. Key takeaways offer actionable insights for stakeholders navigating the complex terrain of rare disease research, paving the way for enhanced enrollment outcomes.

• Exploring different funding avenues: how to structure fundraising initiatives
• Working in collaboration with academia: benefits and opportunities
• The importance of building networks and insights from investors in addition to capital
• Lessons learned: analyzing both successes and learnings for future biotech initiatives

• Biopharma’s role in supporting patients with their disease once no longer study participants
• Working with advocacy groups on long term patient engagement
• Where and how can trial sponsors step in to provide long term support for rare disease patients?
• Bridging the scientific advice gap for patients with rare diseases

• Optimizing and de-risking trial design and feasibility: how data-driven modeling and simulation tools are helping sponsors design smarter, more efficient trials from the outset
• Enabling in-stream decision-making during execution: using real-time insights to adapt trial operations dynamically, improving quality, timelines, and decision confidence
• AI’s transformative impact on trial delivery: from reshaping how we plan and run trials to redefining roles and workflows, AI is set to re-engineer every stage of clinical development