OBJECTIVES: Despite increasing demand from regulators and payers to increase the evidence quality in submissions, assessments lacking comparative trial data remain common at the regulatory and HTA level. This research identifies the HTA outcome success across the EU5 (TC, G-BA, NICE, AIFA, AEMPS) following EMA marketing approval using only non-comparative data.

METHODS: Using Prismaccess, EMA approvals from 1^st Jan 2015 to 24^th Apr 2020 were extracted with their EU5 outcomes. Submissions were excluded if the EPAR was not available or the NCT number of phase II or III trials could not be identified. In the case of HTA resubmissions, the latest decision was considered in the analysis. The highest rating was considered if an HTA body assessed several sub-populations.

RESULTS: Between 2015-2019, 49 positive EMA assessments using only single arm trials were identified from 46 different products. At the EMA, 22/49 (44.9%) assessments were for the first indication of a product, 21/49 (42.9%) assessments were for oncological indications, and 21/49 (42.9%) assessments were for orphan products (at the time of EMA assessment). Cancer and orphan therapies proportionally received a more favorable outcome at each of the EU5 HTA bodies than non-cancer and non-orphan products. A majority (10/12; 83.3%) of orphan assessments completed at the G-BA were awarded non-quantifiable added benefit.

CONCLUSIONS: These results show that cancer and orphan products submitted at the EMA with single arm trials tend to receive better HTA outcomes than non-cancer and non-orphan products. This could be due to a greater unmet need in cancer and orphan indications, resulting in greater flexibility from HTA bodies. The large proportion of orphan assessments awarded non-quantifiable added benefit at the G-BA is likely to be influenced by the automatic legal provision of additional benefit when the annual turnover is less than €50m.