Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability

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 United States and European perspective, including experiences with new therapies for large populations, such as Alzheimer's disease, and gene therapies for ultra-rare conditions. The article concludes by offering some potential policy solutions that can meet the goals of robust evidence generation, patient access, and system affordability, including reimbursement with evidence development, outcomes-based contracts, and other types of managed entry agreements.