Table 3

Summary results of comparing treatment effects between central and local assessments

Summary/subgroup Objective response rate (ORR)Disease control rateProgression-free survival
Study
(n)
Patient
(n)
OR (95% CI)P values*I2†‡Study
(n)
Patient
(n)
OR (95% CI)P values*I2†‡Study
(n)
Patient (n)HR (95% CI)P values*I2†‡
Summary2917 9491.02 (0.97 to 1.07)0.420%1595900.97 (0.92 to 1.03)0.320%7243 6951.01 (0.99 to 1.02)0.320%
Funding source
 Pharmaceutical2817 7641.02 (0.97 to 1.07)0.430%1494050.97 (0.92 to 1.03)0.330%6941 7491.01 (0.99 to 1.02)0.380%
 Academic11851.09 (0.61 to 1.95)0.7611850.98 (0.67 to 1.42)0.91319461.03 (0.95 to 1.11)0.50
Mask
 Open label2314 6161.03 (0.98 to 1.09)0.230%1277770.96 (0.91 to 1.02)0.230%3620 4031.01 (0.99 to 1.04)0.130%
 Single blind11851.09 (0.61 to 1.95)0.7611850.98 (0.67 to 1.42)0.91
 Double blind531480.95 (0.83 to 1.08)0.41216281.02 (0.89 to 1.17)0.773623 2921.00 (0.98 to 1.02)0.93
Region
 Global2315 3841.01 (0.95 to 1.06)0.8124%1286360.96 (0.91 to 1.02)0.2037%6138 7141.01 (0.99 to 1.02)0.370%
 Intracontinental625651.08 (0.97 to 1.21)0.1739541.11 (0.90 to 1.36)0.341149811.01 (0.97 to 1.05)0.68
Trial design
 Superiority2715 7871.02 (0.97 to 1.08)0.480%1595900.97 (0.92 to 1.03)0.326941 5701.01 (0.99 to 1.02)0.34
 Other§221621.03 (0.91 to 1.15)0.67321251.01 (0.95 to 1.07)0.79
Number of enrolled
patients
 >5421713 2601.00 (0.94 to 1.06)0.9411%1077110.97 (0.91 to 1.03)0.260%3730 4931.01 (0.99 to 1.03)0.400%
 <5421246891.06 (0.97 to 1.16)0.18518791.01 (0.88 to 1.15)0.933513 2021.01 (0.98 to 1.04)0.60
Tumour type
 Breast cancer640281.08 (0.97 to 1.21)0.180%534350.97 (0.88 to 1.07)0.580%1510 4101.00 (0.97 to 1.03)0.880%
 NSCLC851721.01 (0.93 to 1.11)0.80320631.01 (0.89 to 1.13)0.911382751.02 (0.99 to 1.06)0.19
 Renal cell carcinoma639171.01 (0.91 to 1.12)0.81319510.95 (0.84 to 1.08)0.431167201.00 (0.96 to 1.04)0.87
 Ovarian cancer319850.98 (0.85 to 1.12)0.7218290.92 (0.78 to 1.08)0.31645361.03 (0.99 to 1.09)0.17
 Melanoma24351.35 (0.91 to 2.00)0.1411850.98 (0.67 to 1.42)0.91414901.02 (0.92 to 1.12)0.74
 Others¶424120.99 (0.87 to 1.12)0.83211271.00 (0.84 to 1.19)1.002312 2641.00 (0.97 to 1.03)0.94
Response assessment criteria
 RECIST2717 0881.02 (0.97 to 1.07)0.550%1494050.97 (0.92 to 1.03)0.330%6840 9481.01 (0.99 to 1.02)0.410%
 WHO28611.11 (0.90 to 1.37)0.3311850.98 (0.67 to 1.42)0.91322021.02 (0.97 to 1.08)0.44
 Not given15451.00 (0.88 to 1.33)0.94
Primary endpoint
 Central assessed1711 1511.04 (0.98 to 1.10)0.2347%1061860.97 (0.90 to 1.04)0.370%4024 5361.01 (0.99 to 1.03)0.440%
 Others**744650.93 (0.83 to 1.04)0.19426800.98 (0.88 to 1.08)0.65959921.02 (0.98 to 1.07)0.26
 Local assessed523331.08 (0.94 to 1.24)0.2717240.99 (0.79 to 1.24)0.952313 1671.00 (0.97 to 1.03)0.96
Primary endpoint
outcome
 Positive1911 8111.04 (0.98 to 1.11)0.240%954840.99 (0.91 to 1.06)0.700%5028 9301.00 (0.99 to 1.02)0.630%
 Indeterminate11851.09 (0.61 to 1.95)0.7611850.98 (0.67 to 1.42)0.9119210.98 (0.89 to 1.07)0.65
 Negative959530.99 (0.92 to 1.07)0.86539210.95 (0.88 to 1.04)0.292113 8441.02 (0.99 to 1.04)0.24
Statistical inferences between central and local assessments
 Consistent1810 7261.02 (0.96 to 1.09)0.530%850940.95 (0.88 to 1.03)0.220%5632 6761.00 (0.99 to 1.02)0.690%
 Inconsistent1172231.02 (0.95 to 1.10)0.60744960.99 (0.92 to 1.08)0.891611 0191.02 (0.99 to 1.05)0.20
  • *P value for the comparison between central and local assessments.

  • †I2 in summary outcome was for heterogeneity of data synthesis.

  • ‡I2 in subgroup was for subgroup difference, representing the interaction effects between the elements of each subgroup factor.

  • §Four non-inferiority, one hybrid design combing superiority and non-inferiority.

  • ¶Four gastrointestinal stromal tumour, three pancreatic tumour, three sarcoma, three medullary thyroid cancer, two glioblastoma, two prostate cancer, two neuroendocrine tumour, one colorectal adenocarcinoma, one gastric cancer, one head and neck cancer and one hepatocellular carcinoma.

  • **Nine overall survival and one unknown-assessed ORR.

  • RECIST, Response Evaluation Criteria in Solid Tumors.