Cross-border enrollment of rare disease patients

Considerations for planning & conducting global rare disease clinical trials

The patient pool for rare disease clinical trials is often small and widely dispersed. Likewise, clinical sites with specialised experience are rare. These issues can be mitigated by enrolling patients who are not residents of the country where the trial is being conducted.

We have seen a recent increase in rare disease drug development activity, partly due to Orphan Drug legislation approved in the US, Japan, Singapore, Australia, and the European Union. Additionally, many organisations are attracted to rare disease research due 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.

As the need for trial participants with rare diseases increases, cross-border enrollment could be the key to enrolling and retaining the required number of participants.

Read the whitepaper to learn:

  • the means to deliver eligible and appropriate study participants to rare disease clinical trials, regardless of national boundaries
  • why cross-border enrollment is a powerful tool for executing efficient and high-quality clinical studies and expediting the development of rare disease treatments