Expected advances in rare disease therapeutics and the role of data in innovation

Spurred by regulatory incentives to develop treatments for rare and orphan diseases, the space has attracted enormous attention and is beginning to bear fruit. Nevertheless, the process of identifying, developing and delivering medicines to patients remains fraught with challenges. This panel discussion examines the dynamic role data might play in rare disease and personalized medicine and determine what companies need to be doing to address this complex and evolving field.

This webinar will answer questions such as:

• What challenges do developers of medicines for rare diseases face in recruitment for clinical trials and gathering data to expedite a path to approval?
• Is there still an appetite for rare disease treatments among investors?
• The skills required for commercializing products are very different to those of an R&D company. How can rare disease companies make the transition?
• How is the Inflation Reduction Act impacting the prospects of companies in this space?
• How can rare disease drug makers secure equitable market access and ensure that these life-saving medicines are affordable to patients?
• What is the role of real-world data in getting treatments for rare and orphan diseases to market?
• What breakthroughs are on the cusp for rare and orphan diseases that make you hopeful for patients?