Clinical Research: Phase 1 - Phase 4

Operational & Regulatory Factors in Cross-Border Enrollment in Rare Disorders

Researchers are more focused than ever on finding treatments and cures for rare diseases, encouraged by a regulatory environment that is increasingly welcoming to innovation and collaboration. But even under these favorable conditions, the potential success of orphan drug development hinges on finding patients and getting them to trial sites.

Patients for rare disease trials can be exceedingly hard to find, and because of variations in regulations, hospital capabilities, standard of care, and/or financial reasons, it’s often not practical to have trial sites in each of the countries where patients live. This webinar examines challenges and solutions in cross-border trial enrollment, including:

  • Working with sponsors and investigators as well as patients, their caregivers, and local physicians to populate trials
  • Understanding the regulatory landscape for cross-border enrollment including submission requirements and informed consent as well as the requirements for visa and insurance
  • Ensuring anonymity of patient records, either by working with local healthcare providers or relying on information provided by the patient or caregiver
  • Paving the way for uneventful patient and caregiver travel by minimizing out-of-pocket costs and providing support for visa interviews, travel booking, local accommodations, and more
  • Carefully attending to the details of living in another country to maximize patient retention

Premier Research’s rare disease experience spans more than 190 rare disease trials over the past five years. Our connections with patient advocates help us locate hard-to-find patients, even among extremely small populations.