Cross-Border Access to Rare Disease Clinical Trials: A Patient-Centric Approach to Feasibility and Execution

Rare disease clinical trials operate under a strong set of constraints: extremely limited patient populations, globally dispersed expertise, small number of clinical trial sites involved and a patient journey often marked by delayed diagnosis, high burden of care, and limited treatment options.

To reach and maintain sufficient sample sizes, sponsors increasingly rely on cross-border access trial models. But once trials extend across geographies, they introduce significant operational, regulatory, and personal challenges—from travel logistics and language barriers to financial burden and caregiver dependency, particularly in pediatric populations. Specific considerations require accommodate the language diversity of the participants, specially in Europe with 24 official languages and in other jurisdictions with diverse population of patients enrolling in the clinical trials.

As a result, unless these trials are designed around the real-world needs and constraints of patients and caregivers, enrollment might stall and retention might fail.

In this webinar, we examine how sponsors can design and operationalize cross-border rare disease trials that are both patient-centric and feasible. We will address critical questions: how to enroll patients globally and how to ensure they can realistically participate and remain in the study through completion. 

Learning Outcomes

1. How to evaluate patient needs for cross-border access participation beyond clinical criteria
2. How to identify and overcome key barriers impacting both enrollment and retention in rare disease trials
3. How to apply practical strategies to design patient-centric, cross-border trial models