Rare diseases are diseases that affect 6.5 to 10 out of every 10,000 people; however, there are nearly 11,000 known rare diseases.1,2 In total, more than 400 million people suffer from rare diseases worldwide. Rare Disease Clinical TrialsFor many patients with rare diseases, clinical trials are often the best and only treatment option. Specifically, patient recruitment is one of the biggest bottlenecks for rare disease clinical trials, which is made challenging due to the small patient pool for any given rare disease. Cross-border CollaborationConcerted global efforts are essential to accelerate orphan drug development and reduce costs involved in rare disease research. Founder and Chairman Dr. Harsha Rajasimha, who lost a child to a rare disease in 2012, has been a rare disease advocate for more than 10 years.

Source: Economic Times March 07, 2023 01:21 UTC