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Table 2 Results of univariable and multivariable logistic regression analyses

From: Randomized controlled trial data for successful new drug application for rare diseases in the United States

Factor

Single-arm trial

(n = 82)

Randomized controlled trial

(n = 151)

Total

(n = 233)

Univariable logistic regression analysis

Multivariable logistic regression analysiss

Odds ratio

95% CI

p value

Odds ratio

95% CI

p value

Prevalence

         

 < 1/100,000

13 (15.9%)

26 (17.2%)

39

Reference

  

Reference

  

 ≥ 1/100,000

69 (84.1%)

125 (82.8%)

194

0.91

0.44–1.88

0.7899

1.31

0.52–3.34

0.561

Severity of the disease outcome

         

 High mortality

67 (81.7%)

67 (44.4%)

134

Reference

  

Reference

  

 Others

15 (18.3%)

84 (55.6%)

99

5.60

2.94–10.68

< 0.0001*

5.63

2.64-12.00

< 0.0001*

Drug usage

         

 Mono therapy

78 (95.1%)

128 (84.8%)

206

Reference

  

Reference

  

 Combination therapy

4 (4.9%)

23 (15.2%)

27

3.50

1.17–10.51

0.0253*

2.95

1.80-18.57

0.0032*

Target age segment

         

 Children with or without adult

24 (29.3%)

55 (36.4%)

79

Reference

  

Reference

  

 Adult only

58 (70.7%)

96 (63.6%)

154

0.72

0.40–1.29

0.2714

1.72

0.75–3.97

0.2027

Primary efficacy endpoint

         

 Pharmacodynamic/Response biomarker

69 (84.1%)

67 (44.4%)

136

Reference

  

Reference

  

 Clinical outcome

13 (15.9%)

84 (55.6%)

97

6.65

3.39–13.06

< 0.0001*

5.57

2.57–12.06

0.0001*

Therapeutic clasiffication

         

 Oncology

63 (76.8%)

46 (30.5%)

109

Reference

     

 Nononcology

19 (23.2%)

105 (69.5%)

124

7.57

4.08–14.06

< 0.0001*

 

N/A

 
  1. *P value < 0.05