Risk and Benefit for Basket Trials in Oncology: A Systematic Review and Meta-Analysis.

Area of Science:

• Oncology
• Clinical Trials
• Precision Medicine

Background:

• Precision medicine is transforming oncology research with novel clinical trial designs.
• Basket clinical trials evaluate treatments across multiple tumor types based on molecular characteristics.
• These trials aim to identify patients likely to benefit from specific experimental therapies.

Purpose of the Study:

• To systematically evaluate the risks and benefits associated with basket clinical trials in oncology.
• To provide an evidence-based understanding of treatment-related adverse events and efficacy outcomes.

Main Methods:

• A systematic literature search was conducted on PubMed, Embase, and ClinicalTrials.gov.
• Data from basket clinical trials published between 2001 and 2023 were analyzed.
• Key metrics included objective response rate, progression-free survival, overall survival, and treatment-related adverse events (grades 3-5).

Main Results:

• The study analyzed 75 basket trials involving 7659 patients.
• The pooled objective response rate was 18.0%, with a treatment-related death rate of 0.7%.
• Grade 3/4 drug-related toxicity affected 30.4% of patients; median progression-free survival was 3.1 months and overall survival was 8.9 months.

Conclusions:

• Basket clinical trials offer a valuable framework for precision medicine in oncology.
• Results provide an empirical foundation for risk-benefit communication in these novel trial designs.
• Findings can inform the refinement of future clinical trial models for targeted therapies.