Debatable Issues in Effectiveness and Efficiency Studies of New Drugs

Modern drug approvals are increasingly granted through accelerated pathways, often based on surrogate endpoints rather than long-term outcomes. While this speeds up patient access, it leaves substantial uncertainty about a new drug’s real-world performance. As a result, there is a growing need for robust post-launch evidence to inform outcome-based agreements and clinical practice.The evaluation of new drugs now relies on a complex mix of traditional trials, real-world data, and economic modeling. This convergence raises important, and sometimes controversial, questions about how we define and measure both effectiveness and efficiency. This Research Topic aims to explore, clarify, and critically discuss these key debatable issues, examining both their methodological foundations and their policy implications.On the effectiveness side, a central theme is the relationship between pragmatic clinical trials and Real World Evidence (RWE) studies. Pragmatic trials maintain randomization but are designed to reflect routine clinical practice, while RWE studies use observational data from sources like registries or electronic health records. These approaches create well-known tensions between internal and external validity and introduce challenges around bias, confounding, and data quality. We particularly welcome contributions that compare these designs, define when each is most appropriate, and propose ways to integrate their findings for regulatory, reimbursement, and health technology assessment (HTA) decisions.On the efficiency side, this Research Topic invites debate on core methodological choices in economic evaluation. One key question is whether probabilistic analysis should be the standard base case in economic modeling, rather than just a sensitivity analysis. This choice has major implications for how uncertainty is communicated and used in pricing decisions. Furthermore, the choice between cost-effectiveness analysis (CEA) and cost-utility analysis (CUA) remains contested. We encourage submissions examining the methodological trade-offs, normative assumptions, and policy consequences of these different approaches.We welcome original research, methodological papers, conceptual and policy analyses, and systematic reviews addressing any of these themes. Dr. Rubio Terrés is the founder and director of HEALTH VALUE, a consultancy firm providing health economics, evidence generation, and market access support to pharmaceutical companies. Topic editors: Abdulmuminu Isah, Huey Yi Chong Source: https://www.frontiersin.org/research-topics/78538/debatable-issues-in-effectiveness-and-efficiency-studies-of-new-drugs