Resources

HTAi Webinar: The Characteristics of Rare Diseases That Complicate the Assessment of Technology Value

Feb 29, 2024

This HTAi Webinar is hosted by the HTAi Rare Diseases Interest Group. Evaluating the value of rare disease technologies poses challenges due to factors like poor natural history, small populations, limited data, complexity, and high unmet needs. Challenges vary across the spectrum of ultra-rare to more prevalent diseases, with added complexities in different populations, such as pediatrics. This webinar aims to highlight these characteristics, elucidate their role in hindering evidence generation and value assessment, and discuss the resulting difficulties in obtaining approvals. The session will also outline the impact of these challenges and detail the strategies planned by the Rare Disease Interest group to address and overcome them.

Panelists:

  • Elena Nicod – Director, Dolon | Italy
  • Fleur Chandler – Head of Market Access UK and Ireland, Sanofi | United Kingdom
  • Dan Ollendorf – Chief Scientific Officer and Director of Health Technology Assessment Methods and Engagement, ICER | United States of America
  • Karen Facey – Independent | United Kingdom
  • Sheela Upadhyaya – Life Sciences Consultant, Independent | United Kingdom

This webinar was held February 27, 2024.