{"entity":{"id":"idea-tr1-validate-real-world-progression-endpoints","kind":"idea","name":"Validate real-world progression endpoints so pragmatic trials can use them","aka":[],"tldr":"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.","summary":"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.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Trial design, endpoints and cost): Davis et al., Availability of evidence of benefits on survival and quality of life of cancer drugs approved by EMA 2009-13 (BMJ 2017)","url":"https://doi.org/10.1136/bmj.j4530"}],"tags":[],"related":["genie"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["pfs","recist","real-world-evidence"],"trials":[],"people":[],"bottlenecks":["b-trial-design","b-real-world-evidence","b-biomarker-validation"],"keyPapers":["paper-davis-bmj"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"In common solid tumours, rwPFS will yield hazard ratios within a pre-specified tolerance of RECIST-based PFS, enabling pragmatic trials to drop protocol imaging schedules in those settings.","rationale":"Retrospective comparisons of rwPFS and trial PFS show reasonable agreement in some cancers; prospective paired validation would allow protocol-free endpoints and make registry-embedded and pragmatic trials far cheaper.","test":"Embed routine-record endpoint capture in three ongoing phase 3 trials and report concordance of progression dates and hazard ratios.","maturity":"early-clinical","actor":"data","cost":"medium","horizonYears":3},"route":"/ideas/idea-tr1-validate-real-world-progression-endpoints/","neighbours":{"collection":[{"id":"genie","kind":"collection","name":"AACR Project GENIE","route":"/collections/genie/"}],"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-biomarker-validation","kind":"bottleneck","name":"Biomarkers are not validated or standardised","route":"/bottlenecks/b-biomarker-validation/"},{"id":"b-trial-design","kind":"bottleneck","name":"Trial design, endpoints and cost","route":"/bottlenecks/b-trial-design/"},{"id":"b-real-world-evidence","kind":"bottleneck","name":"Weak real-world evidence and registries","route":"/bottlenecks/b-real-world-evidence/"}],"paper":[{"id":"paper-davis-bmj","kind":"paper","name":"Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13","route":"/key-papers/paper-davis-bmj/"}],"roadmap":[{"id":"trial-modernisation-roadmap","kind":"roadmap","name":"Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on","route":"/roadmaps/trial-modernisation-roadmap/"}]}}