Table 3 Characteristics of participants in pivotal trials at the indication level providing the basis of U.S. Food and Drug Administration approval of novel therapeutic agents between 2011 and 2013, stratified by indication-level characteristics

 ITT population

490 (360)

6080 (6708)

1368 (623)

1829 (1698)

1181 (789)

415 (106)

2301 (2013)

2295 (2560)

323 (249)

2664 (3607)

 Median, years (IQR)

57.6 (52.2–62.4)

57.1 (50.7–62.2)

47.1 (37.0-50.4)

43.5 (37.6–52.0)

37.7 (34.8–49.9)

40.6 (24.8–65.1)

44.3 (40.9–47.7)

56.2 (44.4–62.5)

51.5 (39.5–59.5)

54.6 (43.1–61.6)

 Aged ≥65 years, proportion of patients (95 % CI)

36.6 % (27.6–45.6 %)

35.1 % (23.1–47.0 %)

12.0 % (0.0–38.1 %)

11.4 % (0.0–32.8 %)

1.5 % (0.0–100.0 %)

35.3 % (0.0–100.0 %)

1.0 % (n/a)

25.4 % (14.5–36.4 %)

30.0 % (21.7–38.2 %)

28.3 % (20.8–35.7 %)

 Male

56.2 % (46.9–65.8 %)

46.2 % (35.8–56.8 %)

66.2 % (55.5–76.8 %)

31.6 % (0.0–75.3 %)

43.3 % (32.3–54.3 %)

64.9 % (27.0–100.0 %)

37.5 % (0.0–77.8 %)

53.6 % (32.5–53.6 %)

51.3 % (46.2–56.3 %)

49.8 % (42.8–57.9 %)

 Female

43.8 % (34.2–53.4 %)

53.8 % (43.3–64.2 %)

33.9 % (23.2–44.5 %)

68.4 % (24.8–100.0 %)

56.8 % (45.8–67.7 %)

35.1 % (0.0–73.0 %)

62.5 % (22.2–100.0 %)

56.9 % (46.4–67.5 %)

48.8 % (43.7–53.8 %)

50.2 % (43.1–57.2 %)

 White

82.6 % (76.4–88.8 %)

71.2 % (63.6–78.8 %)

71.9 % (52.8–91.1 %)

68.4 % (38.8–98.0 %)

81.7 % (70.6–92.8 %)

74.5 % (45.3–100.0 %)

83.3 % (59.1–100.0 %)

83.1 % (78.2–88.1 %)

78.0 % (70.2–85.8 %)

79.8 % (76.4– 83.2 %)

 Black

4.1 % (2.1–6.2 %)

4.6 % (0.3–8.3 %)

16.7 % (8.8–24.7 %)

5.8 % (0.0–12.9 %)

3.2 % (0.0–6.4 %)

15.0 % (0.0–40.5 %)

10.9 % (0.0–49.5 %)

8.6 % (4.6–12.6 %)

5.4 % (2.8–8.2 %)

8.3 % (5.8–10.9 %)

 Asian

9.0 % (3.0-15.0 %)

17.5 % (9.6–25.3 %)

4.3 % (1.2–7.4 %)

12.4 % (0.0–28.1 %)

9.3 % (0.0–18.4 %)

0.4 % (0.0–1.0 %)

4.3 % (0.0–25.8 %)

2.9 % (1.4–4.4 %)

10.6 % (4.9–16.4 %)

5.9 % (3.8–7.9 %)

 Other^a

4.2 % (2.8–5.7 %)

7.0 % (2.8–11.2 %)

7.0 % (0.0–17.5 %)

13.5 % (2.0–24.9 %)

5.7 % (0.1–10.4 %)

10.1 % (0.0–28.9 %)

1.8 % (0.0–7.8 %)

5.4 % (3.0–7.8 %)

5.9 % (2.9–8.9 %)

6.0 % (4.2–7.7 %)

 Hispanic

7.6 % (1.7–13.5 %)

12.6 % (7.4–17.7 %)

15.3 % (11.6–18.9 %)

35.2 % (n/a)

17.1 % (0.0–43.8 %)

42.3 % (15.4–69.2 %)

6.8 % (n/a)

10.7 % (7.1–14.2 %)

10.9 % (4.1–17.6 %)

14.1 % (10.6–17.5 %)

 Non-Hispanic^a

92.4 % (86.5–98.3 %)

87.4 % (82.3–92.6 %)

84.7 % (81.0–88.4 %)

64.7 % (n/a)

82.7 % (56.6–100.0 %)

57.5 % (30.7–84.3 %)

93.1 % (n/a)

89.3 % (85.8–92.9 %)

89.1 % (82.4–95.9 %)

85.9 % (82.5–89.4 %)