Statisticians at the Forefront of Health Technology Assessment: Aligning Regulatory and HTA Evidence Through Transdisciplinary Collaboration

Le résumé fourni par la source

Health technology assessment (HTA) is a transdisciplinary and collaborative process evaluating the clinical, economic, and societal impact of new medical interventions, thereby informing payer decisions. The European Union HTA Regulation (HTAR) and its Joint Clinical Assessment (JCA) procedure mandate the early submission of comprehensive evidence packages by health technology developers, amplifying the need for coordinated statistical expertise throughout the development lifecycle.This review, prepared by a scientific working group of the American Statistical Association’s Biopharmaceutical Section, delineates how statisticians can enhance their impact across the HTA lifecycle. It examines the evolving evidence-generation landscape, emphasizing the benefits of early collaboration between clinical development and market access teams.The article provides guidance on the conduct of robust indirect treatment comparisons and discusses best practices for the collection and analysis of patient-reported outcome (PRO) data. Within the domain of economic modelling, we detail the role of statisticians across a range of activities, and in the realm of artificial intelligence (AI), the potential applications of automation and generative and agentic AI are explored.Through technical expertise and leadership, statisticians can collaborate with cross-functional teams, ensuring that HTA decisions are grounded in rigorous, patient-centered, and timely evidence — facilitating equitable and efficient access to innovative therapies.

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