{"entity":{"id":"idea-data-excluded-populations-rwe-mandate","kind":"idea","name":"Mandatory real-world reporting for patients excluded from pivotal trials","aka":[],"tldr":"Older, frailer and sicker patients are usually kept out of trials but make up most of those treated. Require companies to report how these patients do in practice.","summary":"Pivotal trials exclude patients over 75, with poor performance status, organ dysfunction, brain metastases or HIV, yet these groups receive the drug after approval. The proposal is a post-marketing requirement: within two years of approval, sponsors report real-world effectiveness and toxicity in pre-specified excluded subgroups using structured registry data, with results added to the label.","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"],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["real-world-evidence","geriatric-assessment"],"trials":[],"people":[],"bottlenecks":["b-real-world-evidence","b-aging-comorbidity","b-trial-diversity"],"keyPapers":["paper-gyawali-jama-intern-med"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"Mandated reporting will reveal clinically meaningful differences in toxicity or benefit in excluded populations for a substantial share of new approvals, changing prescribing in those groups.","rationale":"Real-world studies of checkpoint inhibitors in performance status 2 patients found survival far below trial results; such findings currently depend on academic interest rather than requirement.","test":"Retrospectively produce excluded-population reports for ten recent approvals using existing federated data; measure how often the findings would have changed guideline recommendations.","maturity":"speculative","actor":"regulator","cost":"medium","horizonYears":3},"route":"/ideas/idea-data-excluded-populations-rwe-mandate/","neighbours":{"section":[{"id":"ai-computation","kind":"section","name":"AI & Computation","route":"/fronts/ai-computation/"}],"term":[{"id":"real-world-evidence","kind":"term","name":"Real-world evidence","route":"/terms/real-world-evidence/"}],"technology":[{"id":"geriatric-assessment","kind":"technology","name":"Geriatric assessment","route":"/technologies/geriatric-assessment/"}],"bottleneck":[{"id":"b-aging-comorbidity","kind":"bottleneck","name":"Older and multimorbid patients are excluded and undertreated","route":"/bottlenecks/b-aging-comorbidity/"},{"id":"b-trial-diversity","kind":"bottleneck","name":"Trials do not represent the people who get cancer","route":"/bottlenecks/b-trial-diversity/"},{"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/"}]}}