Value assessment and market access of medicines: emerging challenges in HTA

Appraisal outcomes across national Health Technology Assessment (HTA) bodies are shaped by a broad set of considerations: efficacy, safety, disease severity, unmet need, quality of life, patient and carer preferences, price, cost-effectiveness, and budget impact. Frameworks such as the EUnetHTA Core Model, the revised NICE value framework, and the EU HTA Regulation on Joint Clinical Assessment (JCA) have each begun to acknowledge this landscape. Yet whether and how these factors are formally incorporated —or excluded— from value appraisals remains highly inconsistent across healthcare systems and is often poorly documented in published reports, limiting comparability and the transferability of decisions across jurisdictions.This Research Topic aims to consolidate and advance the evidence base on the role these factors play in HTA appraisal processes, also including the use of multi-criteria decision analysis (MCDA), and managed entry agreements. By bringing together empirical, methodological, and policy-oriented work, it seeks to move the field toward a more complete and transparent understanding of value —one that reflects societal and patient priorities without sacrificing the methodological rigor on which credible, reproducible decision-making depends.We welcome original research, systematic and scoping reviews, and methodological contributions addressing: the identification, weighting, and measurement of any factor in national or multinational HTA processes; the role of patient and public involvement in value assessments and its influence on appraisal outcomes; decision-analytic models and MCDA frameworks; the design and evaluation of managed entry agreements; and cross-country comparisons of how these factors appear in real HTA submissions. Studies addressing the transparency, reproducibility, and measurability of these factors are especially encouraged. Case studies from rare diseases, advanced therapy medicinal products (ATMPs), and orphan medicines are also of interest, given the particular challenges these technologies pose to conventional value frameworks. Topic editors: Stefano Capri, Mark J C Nuijten Source: https://www.frontiersin.org/research-topics/84741/value-assessment-and-market-access-of-medicines-emerging-challenges-in-hta