{"entity":{"id":"idea-data-rwpfs-validation-programme","kind":"idea","name":"Validate real-world progression against central imaging review","aka":[],"tldr":"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.","summary":"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.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Weak real-world evidence and registries): Gyawali, Hey & Kesselheim, Assessment of the clinical benefit of cancer drugs receiving accelerated approval (JAMA Intern Med 2019)","url":"https://doi.org/10.1001/jamainternmed.2019.0462"}],"tags":[],"related":[],"cancers":[],"sections":["ai-computation","imaging"],"technologies":[],"targets":[],"drugs":[],"companies":["tempus"],"institutions":[],"pathways":[],"terms":["pfs","recist","real-world-evidence"],"trials":[],"people":[],"bottlenecks":["b-real-world-evidence"],"keyPapers":["paper-gyawali-jama-intern-med"],"journals":[],"dependsOn":[],"notes":[],"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.","test":"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","actor":"research","cost":"medium","horizonYears":2},"route":"/ideas/idea-data-rwpfs-validation-programme/","neighbours":{"section":[{"id":"ai-computation","kind":"section","name":"AI & Computation","route":"/fronts/ai-computation/"},{"id":"imaging","kind":"section","name":"Imaging","route":"/fronts/imaging/"}],"company":[{"id":"tempus","kind":"company","name":"Tempus AI","route":"/companies/tempus/"}],"term":[{"id":"pfs","kind":"term","name":"Progression-free survival (PFS)","route":"/terms/pfs/"},{"id":"real-world-evidence","kind":"term","name":"Real-world evidence","route":"/terms/real-world-evidence/"},{"id":"recist","kind":"term","name":"RECIST","route":"/terms/recist/"}],"bottleneck":[{"id":"b-real-world-evidence","kind":"bottleneck","name":"Weak real-world evidence and registries","route":"/bottlenecks/b-real-world-evidence/"}],"paper":[{"id":"paper-gyawali-jama-intern-med","kind":"paper","name":"Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval","route":"/key-papers/paper-gyawali-jama-intern-med/"}]}}