Table 1.
Characteristics of the phase III RCTs included in the analysis
| Number | Percent | |
|---|---|---|
| Total | 81 | 100 |
| Year of primary publication | ||
| 2012 | 14 | 17.3 |
| 2013 | 8 | 9.9 |
| 2014 | 6 | 7.4 |
| 2015 | 12 | 14.8 |
| 2016 | 4 | 4.9 |
| 2017 | 9 | 11.1 |
| 2018 | 7 | 8.6 |
| 2019 | 5 | 6.2 |
| 2020 | 6 | 7.4 |
| 2021 | 10 | 12.3 |
| Journal of primary publication | ||
| Annals of Oncology | 6 | 7.4 |
| British Journal of Cancer | 1 | 1.2 |
| Cancer | 1 | 1.2 |
| Cancer Cell | 1 | 1.2 |
| Clinical Lung Cancer | 1 | 1.2 |
| European Journal of Cancer | 1 | 1.2 |
| JAMA | 1 | 1.2 |
| JAMA Oncology | 4 | 4.9 |
| Journal of Clinical Oncology | 13 | 16.1 |
| Journal of Thoracic Oncology | 6 | 7.4 |
| Lancet | 4 | 4.9 |
| Lancet Healthy Longevity | 1 | 1.2 |
| Lancet Oncology | 21 | 25.9 |
| Lung Cancer | 5 | 6.2 |
| New England Journal of Medicine | 15 | 18.5 |
| Class of therapy investigateda | ||
| Immunotherapy | 16 | 19.8 |
| Target therapy | 50 | 61.7 |
| EGFR inhibitors | 24 | 29.6 |
| ALK inhibitors | 8 | 9.9 |
| Othersb | 18 | 22.2 |
| Chemotherapy | 17 | 21.0 |
| Control arm: placebo | ||
| Yes | 26 | 32.1 |
| No | 55 | 67.9 |
| Primary tumorc | ||
| Non-specified histology | 37 | 45.7 |
| NSCC | 16 | 19.8 |
| SCC | 7 | 8.6 |
| Non-specified histology—EGFR mutations | 6 | 7.4 |
| NSCC—EGFR mutations | 6 | 7.4 |
| Non-specified histology—ALK rearrangements | 5 | 6.2 |
| NSCC—ALK rearrangements | 3 | 3.7 |
| Non-specified histology—KRAS mutations | 1 | 1.2 |
| Funding | ||
| Profit | 65 | 80.2 |
| Non-profit | 16 | 19.8 |
| Study design | ||
| Superiority | 75 | 92.6 |
| Non-inferiority | 6 | 7.4 |
| Masking | ||
| Open-label | 26 | 32.1 |
| Blinded | 55 | 67.9 |
| Results of the trial | ||
| Positive | 57 | 70.4 |
| Negative | 24 | 29.6 |
| Countries involved in the trial | ||
| Two or more | 55 | 67.9 |
| Single country | 26 | 32.1 |
| Primary endpointd | ||
| OS | 40 | 49.4 |
| PFS | 48 | 59.3 |
| ORR | 1 | 1.2 |
| Safety | 1 | 1.2 |
| QOL | 1 | 1.2 |
| QoL tool usede | ||
| EORTC (QLQ-C30/QLQ-LC13) | 48 | 59.3 |
| FACT (various versions) | 13 | 16.1 |
| EuroQoL (various versions) | 37 | 45.7 |
| LCSS | 17 | 21.0 |
| SILC | 2 | 2.5 |
| SQLI | 1 | 1.2 |
| NSCLC-SAQ | 1 | 1.2 |
| QoL results reported in primary publicationf | ||
| Yes | 56 | 69.1 |
| No | 25 | 30.9 |
aCategories are not mutually exclusive. Two trials included a combination of chemotherapy plus bevacizumab in the experimental arm, compared to standard chemotherapy
b“Others” included the following: 2 trials included a combination of chemotherapy plus bevacizumab in the experimental arm, 2 trials tested bevacizumab, 2 ramucirumab, 2 sunitinib, 2 nintedanib, 1 apatinib, 1 fruquintinib, 1 aflibercept, 1 selumetinib, 1 anlotinib, 1 veliparib, 1 vandetanib, and 1 nytroglicerin
cThe presence of EGFR mutations, ALK rearrangements, or KRAS mutations was mandatory to enroll patients in the trials reported in the table
dCategories are not mutually exclusive. In 9 trials, co-primary endpoints were OS and PFS. In 1, PFS and safety
eCategories are not mutually exclusive
fAmong 56 trials reporting QoL data in primary publications, 10 also reported QoL analysis in secondary manuscripts. Of the 25 trials non-reporting QoL results in primary publciations, 20/25 trials published QoL results in secondary manuscripts, while 5/25 RCTs disclosed QoL data at international conferences