-What advancements have been made towards AI, RWE and scientific breakthroughs in rare diseases? e.g. Patient behaviour and trial recruitment, data collection, digital health, mRNA & other platforms, wearables devices etc.
-Are these technologies being used and do they add significant value to the healthcare ecosystem. E.g., to improve the diagnosis pathway, access to care, clinician workload and impact on healthcare system for rare diseases
-Are regulators, HTAs and payers supportive of these advancements, does it help with their decision-making process?