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