a: Sample size re-estimation and enrichment design
b: Fisher exact test was conducted
c: Conditional marketing authorisation, Post-authorisation safety study,
Post-authorisation efficacy study
Table 4 provides additional descriptive statistics by study progression.
Overall, most studies were completed (24/41, 58.54%) before medicines
were approved. Group sequential design dominated the majority of design
types (9/17, 52.94%) in those drug were approved but ongoing studies
continued. In cases in which the drug was approved and studies had been
completed, the proportion of seamless design outweighed group sequential
design and became the most popular design type (9/24, 37.50%). Compared
with the group of products approved with completed studies, the
proportion of orphan drugs (8/17, 47.06%) was largely greater. When
comparing the 2 groups of different study progression, there was a
statistically significant difference in the proportion of antitumor
drugs (P value = 0.0001) as well as those that required CMA,
PASS, or PAES (P value = 0.0230).
Table 4. Overview of the study status of approvals