PURPOSE:

Health technology assessment (HTA) bodies make decisions on the added benefit of new products compared to standard of care (SoC). However, in some rare diseases and highly selected populations, SoC may be not established. Although the value of real-world evidence (RWE) for HTAs is well discussed, the selection of appropriate RWE sources and its modelling assumptions when no SoC is established, and how it may impact HTA decisions, is unclear. Recent RWE frameworks can inform some of these steps, but decision uncertainty remains.

DESCRIPTION:

Dr. Dawoud will introduce the HTA challenges when direct comparative clinical data are lacking due to absence of established SoC and how RWE may address this evidence gap; two recent NICE submissions in the same rare disease and target population (10 minutes). Dr. Kent will present the RWE sources submitted by the companies and their justifications. The audience will be asked to take the decision-making perspective and hypothesize HTA critiques on the submitted RWE evidence and its quality (15 minutes). Dr. Hernandez will present the challenges from the industry perspective, the HTA critiques in these case studies, and the recommendations; the audience will be asked to take the industry perspective and consider if and how to address the challenges and HTA suggestions to resolve RWE-related uncertainties (15 minutes). Lastly, Dr. Sarri will present how current RWE frameworks could guide RWE decisions, and will prompt questions to the audience on areas such as unbiased selection, data provenance, suitability, and robust analysis that may increase trust in RWE estimates for SoC (15 minutes). The workshop will conclude with an audience discussion on opportunities and challenges of using RWE frameworks in HTA submissions when SoC is not defined (5 minutes). This workshop will benefit statisticians, epidemiologists, payers and industry.