Fig 1.
Selection of historical controls to estimate effect sizes in single-arms studies.
Table 1.
Regulatory characteristics of FDA approvals for lung cancer from 2006 to 2018.
Table 2.
Characteristics of studies of lung cancer approvals by the FDA from 2006 to 2018.
Table 3.
Characteristics of studies used to support FDA lung cancer approvals (2006 to 2018) made via the accelerated vs regular pathways and BTD vs non-BTD.
Graph 1.
Accelerated approval (red), Scatter plot distribution of each study effect sizes measured with hazard ratio as primary endpoint.
Studies using Overall Survival were represented with the triangle and Studies using Progressive-Free Survival were represented with the Circle. Color RED referred to accelerated approval pathway and the BLACK referred to regular pathway.
Graph 2.
Accelerated approval (red), Scatter plot distribution of each study effect sizes measured with risk ratio calculated from studies using response rate as primary endpoint.
All Studies (circle) were single arms, response rate was the primary endpoint and we calculated the risk ratio estimated by using historical control. Color RED referred to accelerated approval pathway and the BLACK referred to regular pathway.
Graph 3.
Breakthrough therapy designation (blue), Scatter plot distribution of each study effect sizes measured with hazard ratio as primary endpoint.
Studies using Overall Survival were represented with the Triangle and Studies using Progressive-Free Survival were represented with the Circle. Color BLUE referred to Breakthrough therapy designation and the BLACK referred to Non- Breakthrough therapy designation.
Graph 4.
Breakthrough therapy designation (blue), Scatter plot distribution of each study effect sizes measured with risk ratio calculated from studies using response rate as primary endpoint.
All Studies (circle) were single-arms, response rate was the primary endpoint and we calculated the risk ratio estimated by using historical control. Color BLUE referred to Breakthrough therapy designation and the BLACK referred to Non- Breakthrough therapy designation.