Presentation (CTI)

OBJECTIVES: SU is defined as uncertainty associated with fundamental choices rather than about parameters’ values of MBEA. The French HAS review MBEAs submitted by companies, depending on key features such as SU, which can result in reservations. Here, we aimed to assess how HAS identify and qualify SU.
METHODS: All publicly-available opinions by HAS relying on MBEA from February 2014 to April 2025 for first or extension of reimbursement were included. SU (explicit wording available in the MBEA) was categorized as per Bojke et al criteria (‘Comparator’, ‘Event’, ‘Health state’, ‘Statistical tool’, ‘Data’, ‘Other’); and presence of reservation resulting from SU was recorded. Analyses were performed overall and by period according to HAS guidelines update date (before/after 07/31/2020). Medians were reported with interquartile range, relevant statistical tests were performed.
RESULTS: We reviewed a total of 212 opinions (112 before guideline update/ 110 after), of which 127 (59%) supported a first reimbursement, with anticancer therapies representing 55% of all submissions. Of these, 197 (92.9%) reported a total of 725 SUs (331 before guidelines update/ 394 after). SUs pertained to issues on Comparators (n=90), Events (n=105), Health states (n=110), Statistical tools (n=121), Data (n=167), or Other (n=132). There was a median number of 3 [2-5] SUs per opinion. It increased with guidelines update (3 [2-4] before, 4 [3-5] after, p<0,0002), mainly within the ‘Other’ category (37 SUs before / 95 after), especially in relation to a concern pertaining to treatment waning (16 SUs before / 52 after). SUs resulted in 571 reservations, with a prevalence increasing from 72% to 85% between the two periods (p<0,00006).
CONCLUSIONS: SU is deemed highly prevalent by HAS in MBEAs, which results in a growing number of reservations. Updating Bojke et al.’s categories would be relevant to better qualify SU and new types of SU such as treatment waning.