Emulate the trial in real-world data first to decide which trials to run
Before spending millions on a randomised trial, analyse existing patient records as if the trial had already happened. If the answer is obvious or the question is unanswerable, skip or redesign the trial.
Funders require a target-trial emulation in linked EHR or registry data as part of any application for a comparative-effectiveness trial: the emulation estimates the likely effect size, event rates, eligible population and recruitment feasibility, and identifies confounders to stratify on. Trials proceed where emulation is inconclusive or where residual confounding is plausible; the emulation also serves as a pre-registered comparator for the trial's result.
- 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.
- 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.
- Funding follows fashion, not burden · Money goes to the cancers and questions that are easy or popular, not the ones that kill most or where a dollar would do most.
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not linked directly; found by shared links- IdeaRandomise inside the registry that already follows every patient
Shares AACR Project GENIE, SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Real-world evidence.
- IdeaValidate real-world progression endpoints so pragmatic trials can use them
Shares AACR Project GENIE, Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaAutomatic weekly linkage of cancer registries to deaths, prescriptions and imaging
Shares SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Real-world evidence.
- IdeaCheap long-term survival follow-up by linking trial participants to registries
Shares SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Trial design, endpoints and cost.
- IdeaRandomise inside the cancer registry: registry-based trials for everyday questions
Shares SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaEmulate combination trials from real-world data to triage which ones to run
Shares AACR Project GENIE, Weak real-world evidence and registries, Real-world evidence.
- IdeaAfter approval, a pragmatic trial in the patients the pivotal trial excluded
Shares Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaStandards for external control arms built from federated real-world data
Shares Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.