OnCo
ideasIdea

Validate real-world progression against central imaging review

Real-world studies say a drug 'stopped working' based on clinic notes. Check how often that matches a proper scan review, and fix the definitions so the two agree.

Real-world PFS (rwPFS) and real-world response are derived from clinician notes and imaging reports, with definitions that differ by vendor. Concordance with RECIST-based central review is only moderately studied. The proposal is a programme in which a sample of real-world progression events in federated datasets is re-read by blinded central radiology, error rates are published by cancer type and data source, and a harmonised definition with known operating characteristics is adopted by regulators.

Hypothesis
Harmonised, validated rwPFS definitions will show agreement with RECIST-based progression within one month in more than 80 percent of events for solid tumours and will be accepted as a supportive endpoint in regulatory submissions.
Rationale
Endpoint validity is the main reason regulators discount RWE for effectiveness; a one-time validation programme is far cheaper than the trials RWE is meant to supplement.
What would test it
Re-read 1,500 real-world progression events across three cancers with blinded RECIST review; publish sensitivity, specificity and timing bias by data source.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
2
Bottlenecks it attacks

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