Table 2.

Summary of Characteristics of Pivotal Evidence in Included Submissions

Characteristic of Pivotal Evidence	N (%)—FDA (N = 20)	N (%)—EMA (N = 17)
Single-arm trial(s) submitted as pivotal evidence alongside 1 or more traditionally controlled trials (randomized controlled or open-label parallel assignment trials)	8 (40)	7 (41)
Single-arm trial submitted as sole pivotal efficacy evidence in submission	12 (60)	10 (59)
Pivotal single-arm trial was Phase 2*	8 (40)	5 (29)
Pivotal single-arm trial was Phase 3*	15 (70)	13 (77) +
Single-arm trial and/or external control arm was included in product labeling, package insert, or summary of product characteristics	18 (90)	2 (12)

*Three studies were considered phase 2/3

 + One study was considered phase 2/3