The landscape of rare disease clinical trials is evolving rapidly, with pharmaceutical companies and researchers facing unique challenges in trial design, patient recruitment, and data management. These critical issues will take center stage at two concurrent events in Brussels this November - the Clinical Trials in Rare Diseases Europe 2024 and the Clinical Data Management Innovation Europe 2024.
Navigating Regulatory Requirements and Trial Design
Rare disease trials face distinct challenges compared to studies of common conditions, primarily due to limited patient populations. Vinciane Pirard, Scientific Advocacy and Insights expert from Sanofi's Medical Affairs Rare Diseases division, will address the complexities of building comprehensive data packages for regulatory approval and payer acceptance.
A notable panel featuring representatives from MSD, Roche, and Sanofi will explore fundamental questions about clinical trial necessity in rare disease treatment development. The discussion will delve into alternative approaches to traditional placebo-controlled studies, reflecting the evolving landscape of clinical research methodology.
Patient-Centric Approaches and Diversity Initiatives
The conference emphasizes the critical role of patient voices in trial design and execution. Patient advocates from various organizations, including the Leber's Hereditary Optic Neuropathy Society and Rare Disease Diversity Coalition, will discuss strategies for creating more inclusive clinical trials and establishing effective advisory boards.
Elizabeth Thomson, a rare disease clinical trial expert, will present compelling arguments for expanding recruitment beyond traditional research centers in the United States and major European Union countries. This geographic diversification strategy aims to address the current limitations in patient representation and access to experimental treatments.
Technology Integration and Data Management
The parallel data management conference will showcase how leading pharmaceutical companies are leveraging technological innovations. Representatives from Pfizer and AstraZeneca will present insights on artificial intelligence integration in clinical data management, focusing on practical, scalable implementations.
For smaller biotechnology companies, experts from Eli Lilly, GSK, and GlobalData will outline risk-based strategies to optimize data management processes. These approaches are particularly relevant for organizations with limited resources conducting complex rare disease trials.
Real-World Evidence and Innovation
Real-world evidence has become increasingly crucial in rare disease research, where traditional trial designs may not be feasible. The conference will explore innovative trial designs that effectively incorporate this data while meeting regulatory requirements.
Jenifer Waldrop, Executive Director of the Rare Disease Diversity Coalition, will lead discussions on identifying and supporting underrepresented populations in clinical trials, addressing a critical gap in current research practices.
