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There is no upper limit on the maximum effect that can be detected in randomized trials
Benjamin Djulbegovic1, Iztok Hozo2, Renata Iskander3
1Division of Medical Hematology and Oncology, Department of Medicine, Medical University of South Carolina, Charleston, SC, USA.
Journal of Clinical Epidemiology
|May 14, 2025
Summary
Randomized controlled trials (RCTs) can discover dramatic cancer treatment effects with no upper limit. Increasing the number of RCTs significantly boosts the probability of identifying breakthrough therapies and accelerating medical advancements.
Area of Science:
- Oncology
- Clinical Trials
- Biostatistics
Background:
- Randomized controlled trials (RCTs) are crucial for detecting both small and large treatment benefits.
- Forecasting the potential for significant treatment effects aids in clinical trial resource allocation.
Purpose of the Study:
- To forecast the likelihood of future large treatment effects in cancer RCTs.
- To guide resource allocation for clinical trial conduct.
Main Methods:
- Analysis of 716 cancer RCTs from 1955-2022, involving approximately 350,000 patients.
- Application of Extreme Value Theory using a generalized Pareto distribution (GPD) to predict maximum treatment effects.
Main Results:
- The GPD analysis indicates no theoretical upper limit on maximum treatment effects.
- Historically, the largest observed odds ratio (OR) was 45; projected maximum OR is 23 over the next 50 years.
- A 20% probability exists for detecting treatments with OR >50 within 50 years; increasing RCTs can significantly enhance this likelihood.
Conclusions:
- There may be no absolute upper bound for discoverable treatment effects in cancer RCTs.
- Conducting more RCTs is essential for increasing the probability of identifying treatments with large effects.
- Sustained or expanded trial activity, coupled with public participation, is vital for accelerating therapeutic breakthroughs.
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