19 February 2025 - Conducting high quality health technology assessments requires high quality evidence. With evolving regulatory standards for faster approval of new pharmaceutical products, health technology practitioners often find that the evidence base available to inform their work is lacking.
This review article provides case examples of how health technology assessors have grappled with this tension, from the US and European perspective, including experiences with new therapies for large populations, such as Alzheimer's disease, and gene therapies for ultra rare conditions.