Fit-for-Purpose Data in Value Assessment in Japan: How Best to Leverage Real-World Evidence (RWE) and Qualitative Value Elements to Optimize Decision Analysis

ISSUE: Value assessment is a key component of decision-making on newly innovative medicines in Japan with considerable potential impact on the healthcare ecosystem. Innovative technologies are quickly evolving to better treat complex and targeted conditions, resulting in the generation of new types of clinical trial designs, including non-RCT designs, and evidence with novel or intermediate endpoints. For example, in oncology and rare diseases, including regenerative medicines, it is not always possible or ethical to conduct randomized controlled trials (RCT). Therefore, we need to rely on evidence from non-traditional trial designs, novel endpoints, and evaluative methods such as indirect treatment comparisons (ITCs) and real-world evidence to inform value assessment. How then, should these data and methods be used and balanced with qualitative decision-analysis in the Japan HTA system to ensure value recognition and thus reduce the drug lag and loss of new medicines to realize optimized innovative care for patients?

OVERVIEW: This panel will critically evaluate how RWE should be appropriately used in the value assessment of innovative medicines, leveraging the Japanese system as a specific example. Dr. Demia will begin the conversation by describing the aims of the Japan HTA approach, and how it currently utilizes RWE and novel evidence to evaluate innovative medicines where there are limited RCT data. Christoph Glaetzer will then provide the developer’s perspective on the R&D process, the considerations that drive decision-making for clinical trial design and approaches to evidence generation. Finally, Dr. Igarashi will provide an HTA perspective on how best to use RWE and HTA evaluation methods to support clinical evidence assessment in Japan. This group will debate pragmatic approaches to achieving an optimized format for future HTA regarding healthcare sustainability for rapidly aging population.