OBJECTIVES: The aim of this study is to describe the characteristics of the economic evaluations submitted to HTA in France (CEESP) and UK (NICE) for therapies with non-comparative phase II trials data.

METHODS: All efficiency opinions based on non-comparative phase II data published on HAS and NICE websites from 2013 to 2022 were reviewed. In each opinion, we analyzed the model structure, time horizon, comparators, indirect comparison method, extrapolation method, methodological reservations, key issues, assessment outcomes and evaluation time. A qualitative comparison between the two agencies was then performed for the analyzable criteria.

RESULTS: 24 opinions (12 indications) were included, with 75% concerning onco-hematology therapies. For all the indications, model structures were similar between CEESP and NICE’s opinions. All NICE’s opinions used a lifetime horizon (mean: 60 years), whereas time horizons were shorter in CEESP’s opinions (mean: 8 years). Considered comparators were the same in NICE and CEESP’s opinions. All opinions estimated comparative effectiveness with indirect comparisons and one indication presented a different indirect comparison method between the two agencies. Extrapolation methods differed for three CAR-T indications (12%). 75% of CEESP’s opinions contained at least one major reservation leading to an ICER invalidation. Non-comparative data and methods of indirect comparison accounted respectively for 24% and 11% of CEESP's methodological reservations. NICE’s key issues concern indirect comparison methods for 26% of opinions and analysis population for 21% of opinions. Mean evaluation time was significantly different between the two agencies (429 days for NICE versus 247 for CEESP).

CONCLUSIONS: Except for time horizons, evaluation methods of phase 2 therapies were comparable between NICE and CEESP. Non-comparative data and their integration methods into the economic model represent the greatest challenge for phase 2 therapies. Specificities for CAR-T regarding extrapolation choices could be observed.