Clinical Trial Size: Importance and Challenges
Author Information
Author(s): J. Haybittle, L. Freedman, P. Armitage, K. Kelly, M. Buyse, D. Spiegelhalter, R. Peto, M. Baum, R. Collins
Hypothesis
Do we need more large simple randomised trials in cancer?
Conclusion
Large clinical trials are essential to detect small but meaningful improvements in cancer treatment outcomes.
Supporting Evidence
- Large trials are necessary to detect small improvements in cancer treatment.
- Only about 2% of eligible cancer patients are currently entered into trials.
- Moderate sized trials can be justified under certain conditions.
Takeaway
To find out if new cancer treatments work, we need to test them on a lot of patients, like 1,000 or more, because even small improvements can be important.
Methodology
Discussion and recommendations from a meeting of statisticians involved in clinical trials.
Potential Biases
Clinicians may underestimate the importance of trial questions, affecting recruitment.
Limitations
Many trials have insufficient patient recruitment, limiting their power to detect treatment effects.
Participant Demographics
Patients with various cancers, with a focus on those eligible for trials.
Statistical Information
Confidence Interval
95% CI from about 10% to 30%
Statistical Significance
p<0.05
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