R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 12

Abstract

In this latest update we highlight the final results from the RCT-DUPLICATE initiative, the publication of guidance from Haute Autorité de Santé (HAS), the joint viewpoint from the Institute for Quality and Efficiency in HealthCare (IQWIG) and the Belgian HealthCare Knowledge Center, and a position from the European Organization for Research and Treatment of Cancer (EORTC). Finally, we discuss how the NICE RWE framework has been implemented to allow consideration of RWE external control arms.

EU HTA Joint Clinical Assessment: are patients with rare disease going to lose out?

“…Further, negative controls and quantitative bias analysis can be used to quantify and correct for any potential residual confounding [ 6 , 8 ]. These frameworks are already having an impact on decision making, with NICE's framework allowing for the acceptance for an RWE external control arm [ 9 ].…”

RWE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 16

“…On the theme of protocol development, in a recent editorial, Wang and Schneeweiss [ 10 ] discuss the importance of carrying out data checks prior to registering a protocol for a RWE study seeking to answer a causal question, based on their learnings from being key members of the RCT-Duplicate initiative [ 11 , 12 ]. They argue that without these evaluations, studies often prove unfeasible due to poor data quality, suitability or reliability.…”