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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