Real‑World Studies Outshine Single‑Arm Trials for Cancer Survival
Experts from European oncology societies and regulatory bodies compared different study designs to determine which provides the strongest evidence that a new cancer drug truly improves survival. Using conjoint analysis, they scored how convincing each study type was under various conditions, such as the quality of real‑world data and the magnitude of survival benefit.
Key Findings
| Group | Preference for Real-World Studies |
|---|---|
| Clinicians | 48 % positive rating |
| Regulators | 13 % positive rating |
- A high‑quality real‑world study showing a 1.5‑month overall survival benefit had odds of being judged stronger than a single‑arm trial 2.7× higher.
- If the benefit increased to 3 months, odds jumped to nearly 15×.
In contrast, randomized controlled trials (RCTs) outperformed single‑arm studies dramatically:
| Survival Benefit | Odds Ratio vs. Single‑Arm |
|---|---|
| 1.5 months | 36× |
| 3 months | >350× |
Risk Tolerance
The perceived strength of evidence correlated with regulators’ willingness to accept serious side‑effect risks. More convincing studies led to higher acceptable toxicity thresholds.
Bottom Line
Real‑world cohort studies—especially those with high data quality or a sizable survival advantage—are regarded as more reliable than single‑arm trials when assessing whether a new treatment genuinely extends patient survival.
