Strength of clinical evidence supporting the United States Food and Drug Administration Accelerated Approvals from 2015 to 2022

Zhang, Xiaofang; Peck, Carl C.; Wang, Yaning; Szucs, Thomas D.; Sun, Wan; Bai, Xue; Chen, Siyu; Wang, Fengzhi; Wu, Yangfeng

doi:10.1186/s12916-024-03800-6

Table 2 Study features of pre-approval pivotal studies during 2015 to 2022

From: Strength of clinical evidence supporting the United States Food and Drug Administration Accelerated Approvals from 2015 to 2022

	Number of trials	Features of pre-approval pivotal studies, %
		Study type^a			Study endpoint			Study phase^a			Number of participants^b
		Randomized controlled trial	Single-arm study	Observational study	Clinical endpoint	Surrogate endpoint	Other endpoint	PhI	PhII	PhIII	Number of participants^b
All	155	21.3	77.4	1.3	0	93.5	6.5	21.6	64.7	13.7	92
2015–16	20	40.0	55.0	5.0	0	85.0	15.0	26.3	52.6	21.1	106
2017–18	41	24.4	75.6	0	0	90.2	9.8	12.2	78.1	9.8	104
2019–20	58	8.6	91.4	0	0	98.3	1.7	31.0	62.1	6.9	58.5
2021–22	36	27.8	69.4	2.8	0	94.4	5.6	14.3	60.0	25.7	106
P for trend 2015–20)			0.007			0.026			0.090		0.014
P (2019–22)			0.012			0.307			0.007		0.004
Oncology indications	128	12.5	87.5	0	0	97.7	2.3	20.3	73.4	6.3	96
2015–16	13	30.8	69.2	0	0	100	0	15.4	76.9	7.7	133
2017–18	36	13.9	86.1	0	0	94.4	5.6	8.3	88.9	2.8	106.5
2019–20	51	5.9	94.1	0	0	100	0	33.3	62.8	3.9	62
2021–22	28	14.3	85.7	0	0	96.4	3.6	14.3	71.4	14.3	106
P for trend(2015–20)			0.016			0.443			0.034		0.001
P (2019–22)			0.212			0.177			0.025		0.011
Non-oncology indications	27	63.0	29.6	7.4	0	74.1	25.9	28.0	20.0	52.0	45
2015–16	7	57.1	28.6	14.3	0	57.1	42.9	50.0	0	50.0	32
2017–18	5	100	0	0	0	60.0	40.0	40.0	0	60.0	94
2019–20	7	28.6	71.4	0	0	85.7	14.3	14.3	57.1	28.6	25
2021–22	8	75.0	12.5	12.5	0	87.5	12.5	14.3	14.3	71.4	83
P for trend (2015–20)			0.148			0.263			0.784		0.859
P (2019–22)			0.037			0.922			0.282		0.096
Novel drugs	70	18.6	81.4	0	0	100	0	2.9	77.1	20.0	104
2015–16	12	33.3	66.7	0	0	100	0	0	66.7	33.3	135.5
2017–18	11	18.2	81.8	0	0	100	0	0	81.8	18.2	104
2019–20	26	7.7	92.3	0	0	100	0	0	92.3	7.7	89.5
2021–22	21	23.8	76.2	0	0	100	0	9.5	61.9	28.6	108
P for trend (2015–20)			0.049			-			0.049		0.038
P (2019–22)			0.127			-			0.387		0.042
Non-novel drugs	85	23.5	74.1	2.4	0	88.2	11.8	37.4	54.2	8.4	64
2015–16	8	50.0	37.5	12.5	0	62.5	37.5	71.4	28.6	0	45.5
2017–18	30	26.7	73.3	0	0	86.7	13.3	16.7	76.7	6.7	106.5
2019–20	32	9.4	90.6	0	0	96.9	3.1	56.3	37.5	6.3	44
2021–22	15	33.3	60.0	6.7	0	86.7	13.3	21.4	57.1	21.4	66
P for trend (2015–20)			0.005			0.009			0.475		0.157
P (2019–22)			0.032			0.187			0.021		0.066
NDA/BLAs	86	27.9	69.8	2.3	0	89.5	10.5	14.3	66.7	19.0	102
2015–16	16	43.8	50.0	6.3	0	81.3	18.8	20.0	53.3	26.7	106
2017–18	17	41.2	58.8	0	0	76.5	23.5	23.5	58.8	17.7	124
2019–20	27	11.1	88.9	0	0	96.3	3.7	3.7	88.9	7.4	87
2021–22	26	26.9	69.2	3.9	0	96.2	3.9	16.0	56.0	28.0	106
P for trend (2015–20)			0.009			0.114			0.952		0.387
P (2019–22)			0.126			0.979			0.564		0.039
sNDA/sBLAs	69	13.0	87.0	0	0	98.6	1.5	30.4	62.3	7.3	63
2015–16	4	25.0	75.0	0	0	100	0	50.0	50.0	0	85
2017–18	24	12.5	87.5	0	0	100	0	4.2	91.7	4.2	95
2019–20	31	6.5	93.6	0	0	100	0	54.8	38.7	6.5	44
2021–22	10	30.0	70.0	0	0	90.0	10.0	10.0	70.0	20.0	89
P for trend (2015–20)			0.229			-			0.050		0.006
P (2019–22)			0.051			0.078			0.014		0.136

^aObservational studies were not included in analysis
^bMedian

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