Presentation (CTI)

OBJECTIVES: To identify the key drivers, including clinical evidence, comparator selection, and other supporting data submitted for oncology therapies reimbursed by HAS in France that achieved an ASMR rating of I or II, with the aim of deriving transferable learnings
METHODS: A list of all HTA assessments in oncology published by the HAS since 2001 was compiled. We then selected assessments that received an ASMR rating of I or II, resulting in a total of 63 assessments. Duplicate assessments for the same drug, or with only minor variations in the patient segment, were excluded. For the shortlisted assessments, information was extracted on trial comparators, commentary on comparator selection, efficacy and safety outcomes, additional evidence such as PROs and ITCs, and other key objections
RESULTS: A total of 44 assessments were included after filtering out 19 assessments based on criteria mentioned above. Of these, 32% received an ASMR I rating, while 68% received ASMR II. Notably, 25% of the assessments did not meet HTA comparator expectations, and 14% were based on single-arm studies. Additionally, 18% of the assessments demonstrated comparable or non-significant efficacy outcomes. Lack of existing treatment options was a key driver for achieving ASMR I/II, accounting for 32% of cases, while another 32% were therapies that demonstrated significant efficacy improvements compared to existing options
CONCLUSIONS: Our analysis indicates that achieving an ASMR I or II rating has become increasingly challenging, reflecting the advancements in the standard of care. While demonstrating superior efficacy has been a key driver, historical assessments show that some therapies achieved these ratings even in the absence of prospective trial data. However, such scenarios are unlikely to be replicable now, and manufacturers must adopt a more rigorous evidence strategy that combines efficacy and safety data with well-designed ITCs and meaningful PROs