Emulate combination trials from real-world data to triage which ones to run
Many combinations are already used off-label. Careful analysis of what happened to those patients can rule out the pairs that clearly do not help before spending money on trials.
Target trial emulation with clinico-genomic databases can estimate effects of combinations that are used in practice but never randomised. Emulations cannot replace trials but can deprioritise pairs with no signal and flag those with large effects. The proposal is a standing programme that emulates every combination used in over 200 patients in the database and publishes ranked results with pre-registered protocols.
- Too many combinations to test · There are thousands of possible drug pairs and sequences. Trials can test a few dozen a year.
- 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.
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