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Table 3 Registration, publication, and interpretation concordance of clinical trials supporting FDA new drug approvals in cardiovascular disease and diabetes between 2005 and 2014, stratified by study and drug characteristics, pre FDAAA

From: Association of the FDA Amendment Act with trial registration, publication, and outcome reporting

 

Number of studies (%)

Registered, n (%)

Odds ratio (95% CI), p value

Published, n (%)a

Odds ratio (95% CI), p value

Concordant interpretation, n (%)a

Odds ratio (95% CI), p value

Overall

183 (100)

151 (83)

 

169 (92)

 

152 (90)

 

Pre FDAAA

 Therapeutic categoryb

  Cardiovascular disease

52 (50)

39 (61)

1.67 (0.72 to 3.89), 0.29

47 (90)

1.43 (0.42 to 4.83), 0.76

40 (85)

1.20 (0.40 to 3.64), 0.78

  Diabetes

53 (50)

34 (64)

 

46 (87)

 

38 (83)

 

 FDA review pathwayc

  Priority review

9 (9)

8 (89)

3.82 (0.46 to 31.85), 0.27

9 (100)

–, 1.00

6 (67)

0.47 (0.11 to 2.08), 0.38

  Standard review

96 (91)

65 (71)

 

89 (93)

 

72 (81)

 

 Orphan statusc

  Yes

2 (2)

2 (100)

–, 1.00

2 (100)

–, 1.00

2 (100)

–, 1.00

  No

103 (98)

71 (69)

 

91 (88)

 

76 (84)

 

 Trial designationd

  Pivotal

63 (60)

50 (79)

3.17 (1.34 to 7.52), 0.01

57 (90)

1.58 (0.47 to 5.29), 0.54

51 (89)

2.83 (0.91 to 8.80), 0.08

  Nonpivotal

42 (40)

23 (55)

 

36 (86)

 

27 (75)

 

 Trial phase

  Phase 2

22 (21)

12 (57)

0.43 (0.16 to 1.14), 0.11

17 (77)

0.31 (0.09 to 1.11), 0.12

17 (100)

–, 0.06

  Phase 3

83 (79)

61 (73)

 

76 (92)

 

61 (80)

 

 Randomized design

  Yes

102 (97)

71 (70)

1.15 (0.01 to 13.1), 0.67

92 (90)

18.4 (1.52 to 221.40), 0.04

77 (84)

–, 1.00

  No

3 (3)

2 (67)

 

1 (33)

 

1 (100)

 

 Double blinded

  Yes

87 (83)

66 (76)

4.94 (1.70 to 14.36), 0.003

82 (94)

10.4 (2.82 to 36.64), 0.0007

73 (89)

9.73 (2.46 to 38.46), 0.002

  No

18 (17)

7 (39)

 

11 (61)

 

5 (45)

 

 Active comparator

  Yes

43 (41)

31 (72)

1.23 (0.52 to 2.89), 0.67

37 (86)

0.66 (0.20 to 2.21), 0.54

28 (76)

0.37 (0.12 to 1.16), 0.09

  No

62 (59)

42 (68)

 

56 (90)

 

50 (89)

 

 Trial duration

  ≤12 weeks

52 (50)

37 (71)

1.16 (0.51 to 2.67), 0.88

46 (88)

0.98 (0.29 to 3.26), 0.61

41 (89)

2.21 (0.69 to 7.09), 0.26

  >12 weeks

53 (50)

36 (68)

 

47 (89)

 

37 (80)

 

 Number of patients in trial

  <500

59 (56)

36 (61)

0.28 (0.15 to 0.93), 0.03

48 (81)

0.19 (0.04 to 0.92), 0.01

41 (85)

1.26 (0.42 to 3.83), 0.78

  ≥500

46 (44)

37 (78)

 

45 (98)

 

37 (82)

 

 Jadad score

  <3

3 (3)

2 (67)

0.87 (0.07 to 9.99), 1.00

1 (33)

0.05 (0.01 to 0.65), 0.03

1 (100)

–, 1.00

  ≥3

102 (97)

71 (70)

 

92 (90)

 

77 (84)

 
  1. CI confidence interval, FDA US Food and Drug Administration, FDAA FDA Amendment Act, – too few events to calculate odds ratio
  2. a“Published” includes publication in the peer-reviewed literature. The “Concordant interpretation” column uses “Published” as the denominator to determine percentages
  3. bAs defined by the World Health Organization’s Anatomic Therapeutic Classification system (http://www.whocc.no/atc_ddd_index/), contextualized for clinical relevance
  4. cFDA designation
  5. dA trial was considered pivotal if it was the only trial included in the summary or if it was explicitly designated as pivotal; all other trials were nonpivotal
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ISSN: 1745-6215