Archives: Agenda

• Correcting common errors when importing drugs and devices into the United States.
• How to communicate with FDA when your shipments are on hold?
• Understanding the complexity of filing an FDA electronic entry.
• Getting to know your FDA management staff when importing through the West Coast.

During the roundtable discussion session, the conference hall will be divided into four ‘zones’. Delegates can choose which zone they would like to join. Each zone will be led by a table moderator and will focus on a different challenge within clinical data management. After 45 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

ROUNDTABLE 1
Overcoming challenges in patient identification for ultra rare diseases
Kenneth Harvey, Director Clinical Operations, Epic Bio

ROUNDTABLE 2
Exploring strategies for building greater DE&I in rare disease trials
Aditi Kantipuly, MD, MS, Physician, Visiting Scholar University of New Mexico

ROUNDTABLE 3
Crafting recruitment strategies for rare disease trials
Patrick Shores, Senior Director of Sales, Leapcure

• Where rare disease trial sponsors can step in to fill scientific advice gap and teach patients about their condition
• The importance of making patients feel cared for throughout and beyond trial participation
• How rare disease trials differ from other clinical trials when it comes to supporting patients in understanding and managing their disease long term

• Understanding successful protocol design trends and competitive activity in the rare disease space
• Utilizing data analytics and benchmark models for assessing geographic feasibility, timelines, enrollment, and trials cost forecasting
• Improving subject accrual and the site selection process
• Execution phase and beyond – tracking investigators and competitive trials, mitigating potential complications

• Involving patients in trial design to ensure that trial outcomes are relevant and meaningful to patients
• Patient-centric approach: integrating patient feedback and needs into research
• Building a collaborative network

• Analyzing the specific regulatory challenges posed by the IVDR for rare disease studies and the implications for trial design and execution
• Exploring the crucial role of diagnostics in rare disease clinical trials, including their use for patient stratification, biomarker identification, and treatment assessment
• Addressing the need for streamlined processes and collaborative efforts to reduce the burden on test development for diagnostics used in rare disease studies, ensuring efficiency and compliance

• Discussing the impact of eligibility criteria and small patient population
• Exploring innovative approaches to recruit eligible participants, including leveraging digital platforms and social media outreach
• Highlighting the significance of collaboration between researchers, clinicians, patient advocacy groups, and regulatory bodies in overcoming enrollment challenges
• Strategies for navigating the complexities of rare diseases to ensure accurate diagnosis, patient identification, and enrollment
• Maintaining integrity in clinical trials and risks of premature publicity