This review study assessed if there is an efficacy between phase...

This review study assessed if there is an efficacy between phase ii and phase iii in oncology trials.

https://pubmed.ncbi.nlm.nih.gov/32034790/

What is already known about this subject

• Drugs are increasingly being evaluated based on a single pivotal trial, sometimes with only phase II data.
• Objective response rates reported in phase II trials are higher than in phase III trials in solid malignancies.

What this study adds

• There are no marked average differences in effect estimates between phase II and phase III trials in oncology, but differences are subject to large variations between indications.
• Differences in efficacy estimates measured in trials are associated with multiple covariates.
• Replication of oncology trials remains essential to confirm efficacy of oncology drugs and discern variables associated with demonstrated effects.

4.4 Conclusions

Overall, effect estimates in this dataset of trials measuring objective response rate and median progression free and/or overall survival did not show marked average differences between phase II and phase III trials for oncology drugs. These results may encourage decision‐makers such as clinicians, regulators or health technology assessment bodies to regard the quality of design and total body of evidence, including patient population and trial characteristics, instead of differentiating between phases of clinical development. The large variability in effect size differences throughout individual indications emphasizes that proper replication of study findings remains essential to confirm efficacy of oncology drugs and discern variables associated with demonstrated effects.