The COVID-19 pandemic is having significant impact on clinical trials globally. Many trials face suspension or closure due to clinical priorities and the impact of social distancing and quarantine.

Clinical trial patients with a rare condition face great challenges; being part of a clinical trial when living with a rare condition can often be the only lifeline available, offering the chance to access novel therapies that could improve life enormously. During the pandemic, not being able to access novel therapies could have a major negative impact on health, disease progression and mortality. In addition, progress toward an approved therapy should not have to be completely stalled because of a lack of ability to collect relevant data.