Clinical trials for glioblastoma, a type of aggressive brain tumor, have shown significant changes in the primary evaluation items in phase II trials. Researchers at the University of Tsukuba have conducted a study analyzing the trends in efficacy endpoints in phase II clinical trials for glioblastoma conducted worldwide from FY 2020 to 2022. The study compared the data with historical trends from FY 2017 to 2019, revealing interesting insights into the evolution of trial design and evaluation metrics.
Traditionally, response rate (ORR) has been the standard efficacy endpoint in early-stage clinical trials for cancer. However, due to the unique biological characteristics of glioblastoma, such as its invasive nature, using solid tumor efficacy criteria for evaluation has been challenging. The recent study found that the primary endpoints in glioblastoma trials are shifting towards more time-to-event measures, such as progression-free survival (PFS) and overall survival (OS). The use of ORR as a primary endpoint has decreased significantly, indicating a more comprehensive approach to trial design.
In FY 2020-2022, researchers identified 101 primary endpoints set in 88 trials, with PFS, OS, and PFS rate being the most commonly selected measures. This trend towards using time-to-event indicators reflects the need for more sensitive and clinically relevant metrics in evaluating glioblastoma treatments. The study also highlighted a diversification in the types of primary endpoints across trials and a move towards trial designs that incorporate multiple endpoints, rather than focusing on a single measure.
The findings from this research provide valuable insights for guiding future clinical trial design in glioblastoma treatment. By adapting trial design to reflect the complexity of evaluating glioblastoma therapies, researchers aim to establish more clinically relevant and effective metrics for assessing treatment outcomes. This evolution in trial design signifies a step towards improving the efficacy and safety assessment of treatments for glioblastoma, ultimately benefiting patients with this aggressive form of brain cancer.
This study, published in the journal Cancers, sheds light on the changing landscape of glioblastoma clinical trials and emphasizes the importance of evolving trial design to align with real-world clinical practice. The research conducted at the University of Tsukuba contributes to advancing regulatory science in glioblastoma treatment evaluation, paving the way for more effective and patient-centered clinical trials in the future.
