CLOSING PANEL DISCUSSION: Strategies for minimising participant burden to ensure on-time enrolment in rare disease research

  • Where are the main hurdles patients face and how can these be removed?
  • Barriers to clinical trial participation from a patient perspective
  • Informed consent and addressing various levels of health literacy in the patient population: how to improve communication
  • Engaging patients and advocacy groups early on in the clinical trial design process
  • Patient reimbursement: how to relieve financial pressures on patients participating in orphan trials

How to optimize cost-efficiency in rare diseases clinical trials

  • Control costs in rare disease clinical trials by tackling complexity and uncertainty head-on
  • Optimize site and country selection to reduce startup delays and unnecessary costs
  • Streamline vendor management with clear oversight and performance-driven partnerships
  • Leverage operational efficiencies (monitoring strategies, data management, patient engagement)
  • Maintain quality and patient focus while optimizing financial sustainability