The rare disease space has ~10,000 documented rare diseases across the globe, 80% of which have a genetic origin, and unfortunately, 95% of these indications lack an approved intervention. Often, treatment for these conditions involves gene therapies or Antisense oligonucleotides, which lend well to cross-border enrollment. This is especially true in rare diseases where those impacted are likely to be limited and geographically dispersed. Cross-border enrollment involves a patient from a given country with no treatment opportunities to enroll in a clinical trial located in another country. This process is highly complex and requires a careful strategic approach for successful operations.
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