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

Presented by

Hanna Wide Director, Program Delivery Rare Diseases & Pediatrics at Premier Research

About this talk

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 will examine 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. Register to get access to insights you can leverage in your rare disease drug development program.

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Premier Research is a clinical research company, dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments. As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors. Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.