A pre-registered standard for emulating trials with real-world data
When researchers use hospital records to ask 'would drug A have beaten drug B in a trial', they should follow a published recipe and register their plan first, so the answer can be trusted.
Target trial emulation (specifying the hypothetical randomised trial, then mimicking it in observational data) is the accepted framework for causal inference from real-world data, but oncology emulations vary widely in rigour. The proposal is a formal standard: a protocol template (eligibility, treatment strategies, assignment, time zero, outcomes, causal contrast, analysis plan), mandatory pre-registration, and a requirement to report a positive control emulation of a known RCT in the same dataset, adopted by regulators and journals as a condition of considering RWE.
- Weak real-world evidence and registries · We do not reliably know what happens to patients after approval, so we cannot tell which drugs deliver in practice.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
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