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Table 1 Trial variables associated with subsequent Food and Drug Administration drug approval

From: Food and Drug Administration approvals in phase 3 Cancer clinical trials

Trial Variables

FDA Approval (N = 225)

No FDA Approval (N = 565)

Univariate Analysisf

Multiple Binary Logistic Regressionf

N (%)

N (%)

P

aOR [95%CI]

P

Primary Endpoint

 Overall Survival

65 (28.9)

162 (28.7)

0.01

–

 

 Quality of Life

2 (0.9)

41 (7.3)

 

0.4 [0.1–3.6]

0.41

 Other Endpointsa

158 (70.2)

362 (64.1)

 

1.4 [0.9–2.1]

0.17

  Progression-Free Survival

96 (42.7)

138 (24.4)

   

  Disease-Free Survival

8 (3.6)

39 (6.9)

   

  Event-Free Survival

2 (0.9)

10 (1.8)

   

  Complete Response

4 (1.8)

9 (1.6)

   

  Safety/Toxicity

1 (0.4)

28 (5.0)

   

Industry-Fundingb

219 (97.3)

387 (68.5)

< 0.001

5.9 [2.1–16.7]

0.01

Cooperative-Group-Supportb

21 (9.3)

214 (37.9)

< 0.001

0.5 [0.2–0.9]

0.02

Line of Therapy

 Advanced/Metastatic First-Line

67 (29.8)

174 (30.8)

< 0.001

–

 

 Advanced/Metastatic Second-Line

67 (29.8)

104 (18.4)

 

1.0 [0.7–1.7]

0.85

 Localized Solid

22 (9.8)

119 (21.1)

 

0.8 [0.4–1.6]

0.53

 Hematological First-Line

35 (15.6)

67 (11.9)

 

2.5 [1.3–4.8]

0.01

 Hematological Relapsed/Refractory

26 (11.6)

23 (4.1)

 

2.4 [1.2–4.8]

0.02

 Mixed Stages

8 (3.6)

78 (13.8)

 

1.8 [0.5–7.2]

0.40

Disease Site

 Breast

35 (15.6)

112 (19.8)

0.07

  

 Gastrointestinal

24 (10.7)

74 (13.1)

   

 Genitourinary

30 (13.3)

65 (11.5)

   

 Head and Neck

7 (3.1)

21 (3.7)

   

 Hematologic

60 (26.7)

95 (16.8)

   

 Lungs

29 (12.9)

85 (15.0)

   

Modalityc

 Systemic Therapyd

212 (94.2)

409 (72.4)

< 0.001

–

 

 Radiation Therapy

0 (0)

23 (4.1)

 

0 [0-NA]

0.99

 Surgery

0 (0)

8 (1.4)

 

0 [0-NA]

0.99

 Supportive Caree

13 (5.8)

123 (21.8)

 

0.2 [0.1–0.7]

0.01

Patient Accrual Met

204 (91.9)

421 (76.5)

< 0.001

2.6 [1.4–4.7]

0.01

Total Patients Enrolled – Median [IQR]

572 [366–866]

uOR: 1.0

449 [230–772]

0.97

  

Pre-Hoc Power – Median [IQR]

90 [80–90]

uOR: 1.1

83 [80–90]

< 0.001

1.1 [1.05–1.14]

< 0.001

  1. Abbreviation: uOR unadjusted Odds Ratio, aOR adjusted Odds Ratio, CI confidence interval, IQR interquartile range, FDA Food and Drug Administration
  2. a The most common primary endpoints used in phase 3 clinical trials other than overall survival and quality of life metrics were noted
  3. b Industry funding and cooperative group sponsorship were considered independent variables as certain trials were both industry-funded and performed through a multi-institutional cooperative group
  4. c Modality addressed the primary intervention as part of the randomization
  5. d Systemic therapy trials, including chemotherapy, targeted systemic agents, immunotherapy, and others, accounted for most trials by modality; they used systemic therapies to improve disease-related outcomes (eg, overall survival, disease-free survival)
  6. e Supportive care trials were those where the intervention aimed to reduce disease- or treatment-related toxic effects as the primary endpoint
  7. f Pearson’s Chi-squared and univariate binary logistic regression test were used in univariate analyses to assess the association between individual trial variables and subsequent FDA drug approval. Trial variables that had a two-sided P-value less than 0.05 were subsequently included in multiple binary logistic regression modelling
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BMC Cancer

ISSN: 1471-2407

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