Presentation (CTI)

Problem Statement: In an ever-changing environment, health technology assessment (HTA) agencies must adapt their methods to inform decision-makers regarding fair and rapid access to innovative therapies, even in the face of low levels of evidence. In this regard, the Institut national d’excellence en santé et services sociaux (INESSS) introduced a new type of reimbursement recommendation in 2018. This mechanism allows exceptional access to innovative therapies in contexts where clinical data are limited or immature, with a requirement for re-evaluation when deemed appropriate by INESSS. This case study examines the methodology used by INESSS for two CAR T-cell therapies (Kymriah and Yescarta) and the challenges faced when re-evaluating their budget impact analysis (BIA).
Description: A reanalysis of the initial BIA, conducted in 2019 using an epidemiological approach, was performed. The listing of these therapies, in addition to the follow-up requirements inherent to this new reimbursement mechanism, enabled certified centers to collect real-world data (RWD) over the years. This facilitated the conduct of a retrospective BIA with up-to-date data, which was also corroborated by sales data from the IQVIA Canadian Drugstore and Hospital Purchase Audit database. Overall, the initial estimations for Yescarta were accurate, but the market uptake was slower than anticipated. In contrast, the initial estimations for Kymriah were significantly overestimated. Indeed, various discrepancies emerged when comparing the two analyses, each based on a different approach, allowing INESSS to assess the differences and collaborate with Quebec clinicians to elucidate the underlying reasons.
Lessons Learned: In the absence of more robust data, the epidemiological approach is often considered the method of choice to conduct BIAs and inform decision-makers. However, this approach is subject to uncertainty and partly based on assumptions derived from clinicians’ input. A retrospective analysis using RWD provides more accurate estimates and should be favored in a re-evaluation context whenever possible.
Stakeholder Perspective: Government