Validate real-world progression endpoints so pragmatic trials can use them
Pragmatic trials want to use the progression dates recorded in ordinary clinic notes instead of expensive protocol scans. Checking how well those routine records match formal trial measurements would show when that shortcut is safe.
Paired studies in which patients enrolled in conventional trials also have their routine clinical progression (rwPFS from clinician notes and imaging reports, abstracted or NLP-derived) captured, quantifying agreement in progression dates and hazard ratios by tumour type. Regulators publish acceptable-use conditions for rwPFS and time-to-next-treatment as endpoints in pragmatic and registry-based trials.
- 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.
- Biomarkers are not validated or standardised · Tests that decide who gets a drug are often not validated prospectively and are measured differently in every lab.
Pages like this
not linked directly; found by shared links- IdeaValidate real-world progression against central imaging review
Shares RECIST, Weak real-world evidence and registries, Real-world evidence, Progression-free survival (PFS).
- IdeaEmulate the trial in real-world data first to decide which trials to run
Shares AACR Project GENIE, Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaPublish the disagreement between trial doctors and independent reviewers for every trial
Shares RECIST, Progression-free survival (PFS), Biomarkers are not validated or standardised, Trial design, endpoints and cost.
- IdeaOpen, benchmarked algorithms for lines of therapy and progression from routine data
Shares Weak real-world evidence and registries, Real-world evidence, Progression-free survival (PFS).
- 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.
- IdeaRandomise inside the registry that already follows every patient
Shares AACR Project GENIE, Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.