Improving Research in Rare Diseases: Answering Key Questions for 2021 and Beyond
The awareness, treatment, and management of rare and orphan disease continues to expand at a global pace. However, with over 6,000 identified rare diseases affecting a population nearing 300 million people, there is still so much unknown. As the pharmaceutical industry looks back on two decades of discovery and innovation, the need for an assessment of current best practices becomes critical to further clinical research, especially in light of the current COVID-19 pandemic. What is on the horizon for rare disease intervention in 2021 and beyond?
This panel discussion will provide insight into the successes, challenges, and expectations of the rare and orphan disease clinical trial arena, answering key questions related to:
• Examining the affect that the COVID-19 pandemic has had on patients suffering from rare diseases, as well as obstacles to the progress of clinical research
• Understanding the need for changes to traditional trial processes in order to improve success rates in rare disease clinical studies
• Identifying market trends in treatment options, patient burdens, and disease diversity as they evolve in the coming years
Speakers:
Marc M. Gas, Ph.D., Head, Nephrology Center of Excellence, Senior Manager of Program Development, Biorasi
Monica Gangal, Vice President Clinical Operations, Eiger Biopharmaceuticals
Bruno Gagnon, Senior Vice President, Development Operations, Eidos Therapeutics
