TABLE 2.

Characteristics of pivotal trials at initial approval and post‐approval confirmatory trials at conversion, per indication

	SMA indications (initial approval, N = 15)		Converted a CMA indications (initial approval, N = 15)		Converted a CMA indications (conversion, N = 14)		Unconverted a CMA indications (initial approval, N = 17)
Number of trials
Median, range	1	1–3	1	1–2	1	1–3	1	1–1
Availability of at least a:
Phase III trial	11	73%	4	27%	11	79%	3	18%
Controlled trial	11	73%	4	27%	11	79%	5	29%
Trial with OS as primary endpoint	4	27%	0	0%	2	14%	0	0%
Trial with PFS/TTP or DFS as primary endpoint	7	47%	4	27%	9	64%	4	24%
Trial with ORR as primary endpoint	4	27%	11	73%	3 b	21%	13 c	76%
Trial in which OS data were collected	15	100%	15	100%	14	100%	17	100%
Trial in which QoL data were collected	14	93%	7	47%	13	93%	10	59%
Total number of patients included in trials
Median, IQR	356	278–1109	196	116–356	414	320–1053	97	74–139

CMA, conditional marketing authorization; DFS, disease‐free survival; IQR, interquartile range; ORR, overall response rate; OS, overall survival; PFS, progression‐free survival; QoL, quality of life; SMA, standard marketing authorization; TTP, time to progression.

^a (Un)converted as at 31 December 2020.

^b One formally defined as ORR with a duration of at least 6 months, i.e., durable response rate.

^c Two formally defined as major cytogenetic response rate and complete response rate, respectively.