Figure 3. Bar chart of year of first marketing authorization of 41 EMA approvals planned with adaptive designs in clinical trials.
All descriptive statistics in the 3 time periods are reported in Table 3, which provides a general review of all approvals. Of the 41 approvals, 32 (78.05%) contained an adaptive design trial as pivotal evidence. Most of these were not orphan drug approvals (27/41, 65.85%) but were NAS drug approvals (32/41, 78.05%), and fewer than half were antitumor drug approvals (19/41, 46.34%). Moreover, 92.68% (38/41) were not approved by accelerated assessment, and 53.66% (22/41) were required to undergo AM. Of the 41 products reviewed, 58.54% (24/41) did not require CMA or post-authorisation safety or efficacy studies. In total, approvals including 15 types of indications according to SOC, neoplasms (7/41, 18.92%), and blood system disorders (8/41, 19.51%), were relatively more prevalent. Among the 3 periods, the proportion of AM was the highest in 2017–2020 with statistical significance (Pvalue < 0.0001).
Table 3. Overview of the European Medicines Agency (EMA) approvals planned with adaptive designs