ISSUE: Innovative health technologies, precision medicine and the increasing recognition of real-world evidence (RWE) have transformed the healthcare decision-making process. To follow these trends and to meet a high need for earlier patient access to promising technologies, the clinical trial landscape is rapidly evolving with the rise of single-arm trials. To establish comparative effectiveness of new technologies, decision makers have shown preference for national data to construct external control (EC) arm reflecting local treatment pathways with an emphasis on patient representativeness. However, this preference is not always realistic depending on data availability, sample size and quality that may affect confidence in results and, ultimately, in decision-making. Can the advance in RWE methodological approaches overturn the HTA over-dependency on local data?

OVERVIEW:

Unquestionably, the selection of EC arms to support the comparative effectiveness analyses for health technologies evaluated in single-arm trials has been a major area of criticism during HTA submissions. The moderator will begin by framing the topic, conflicting issues around quality and locality in the selection of the most reliable source for RWE EC, and the impact of under-development HTA guidance. Then, the moderator will present three case-studies in different disease areas (rare diseases, oncology, chronic conditions) to the panel, who will debate, from an industry, academic and HTA perspective (12 min each presentation) the main drivers for RWE EC acceptance and challenges when decision makers are urged to select the least biased RWE source to support reimbursement submissions. How challenging is it to walk the line between RWE quality and locality when selecting the “most appropriate” EC arm? Which factors may shift the decision from one side (quality) to the other (locality) and is this disease-dependent? A 12-minute interactive discussion using online polling system with the audience will be encouraged. Industry, researchers, and payers will benefit.