Cross-Border Enrollment of Rare Disease Patients

In this white paper, PRA experts discuss the unique challenges drug developers face when planning and conducting global rare disease trials, and the robust potential that cross-border enrollment can have in navigating regulatory boundaries.  

Executive Summary

Clinical trials in rare diseases present unique challenges unseen in trials for more common conditions. Rare disease patient populations are inherently small, and prospective participants are widely dispersed. Likewise, the number of clinical research sites with such specialized experience is limited. These issues can be mitigated by enrolling patients who are not residents of the country in which the trial is being conducted. Cross-border enrollment can be key to enrolling and retaining the required number of participants in a rare disease clinical trial.

Thanks in part to Orphan Drug legislation approved in the US, Japan, Singapore, Australia, and the European Union, we have seen a recent increase in rare disease drug development activity. In addition to financial incentives supporting development of treatments for rare diseases, these previously intractable conditions are targeted by advancement in medicinal therapy such as gene therapy and somatic cell therapies. The aforementioned factors combine to create a regulatory maze that must be appropriately navigated. Understanding and complying with these regulatory considerations as part of patient logistics is critical to timely and successful study completion.

Download Our White Paper

Trials in rare indications require innovative approaches designed to engage these patients in what can be challenging studies for the entire family. Through our experience, PRA has developed an effective planning and execution process. Click here to learn more.


Amy Raymond
Clinical Scientist, Scientific Affairs – Rare Diseases

Joanna Sprague
Manager, Global Regulatory Affairs

Venkata Jaggumantri
Clinical Scientist, Scientific Affairs – Rare Diseases