Risk-Benefit of Phase 2 Monotherapy Trials in Adult Solid Cancers: A Systematic Review and Meta-Analysis.

In 30 seconds

This systematic review and meta-analysis evaluated the efficacy and safety of monotherapies in phase 2 versus phase 3 trials for adult solid cancers. Analyzing 130 phase 2 and 52 phase 3 trial arms with 6,665 and 18,694 patients respectively, the study found a pooled objective response rate of 7% in phase 2 compared to 24% in phase 3 (p < 0.0001).

Key findings

• The pooled objective response rate was 7% in phase 2 versus 24% in phase 3 (p < 0.0001).
• Median progression-free survival was 3.23 months in phase 2 compared to 5.43 months in phase 3 (p < 0.0001).
• Median overall survival was 9.46 months in phase 2 versus 14.44 months in phase 3 (p = 0.0001).
• The rate of drug-related grade 3-4 adverse events was 30% in phase 2 and 25% in phase 3.

Why it matters

Understanding the risk-benefit profile of phase 2 monotherapy trials is crucial for clinicians when considering treatment options and ethical implications of drug administration in early-phase trials.