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Collaborative clinical trials: quality or quantity?
1Department of Medical Oncology and Hematology, Princess Margaret Hospital and University of Toronto, Toronto, Canada. ian.tannock@uhn.on.ca
Purpose:
To review the merits of using the limited available resources--patients, money, clinical scientists, and ideas--in various types of clinical trial.
Conclusions:
Two types of trial represent a poor use of resources: (a) nonrandomized trials that provide no insight into biologic mechanisms and are not precursors to testing new strategies in comparison with standard treatment in randomized trials; and (b) small randomized trials that are difficult to interpret because of a high rate of false-positive and false-negative trials. Very large trials that can detect small differences in survival for patients with common tumors are appropriate, but a similar design to detect transient improvements due to palliative therapy represent a poor use of resources. Larger gains in therapeutic index will require the recognition of tumor heterogeneity and the conduct of small trials that are based on biologic hypotheses, and which provide mechanistic information in patients; two examples are provided. Ultimately, strategies that may be individualized among a group of patients with histologically similar tumors will need to be evaluated against the current standard (and homogeneous) treatment.
Insights
Clinical trial design must optimize resource allocation. Non-randomized and small randomized trials often waste resources, while large trials for common tumors and hypothesis-driven small trials focusing on tumor heterogeneity are more effective.
Area of Science:
- Oncology
- Clinical Trial Design
- Biostatistics
Background:
- Limited resources necessitate careful consideration in clinical trial design.
- Evaluating the efficiency of various clinical trial types is crucial for advancing cancer research.
Discussion:
- Non-randomized trials lacking mechanistic insights and small randomized trials with interpretation challenges represent poor resource utilization.
- Large trials are effective for detecting minor survival differences in common tumors, but not for transient palliative therapy improvements.
Key Insights:
- Optimizing resource allocation in clinical trials is paramount.
- Biologically-driven, mechanistic small trials are essential for identifying therapeutic index gains.
- Recognizing tumor heterogeneity is key for developing effective, individualized treatment strategies.
Outlook:
- Future research should focus on hypothesis-driven, mechanistic trials to uncover novel therapeutic strategies.
- Individualized treatment strategies require rigorous evaluation against current standards of care.