# Mandatory real-world reporting for patients excluded from pivotal trials

Source: https://onco.cc/ideas/idea-data-excluded-populations-rwe-mandate/  
OnCo record `idea-data-excluded-populations-rwe-mandate` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- 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.
- Proposed 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

## Sources

- 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): https://doi.org/10.1001/jamainternmed.2019.0462

## Connected records

- fronts: [AI & Computation](https://onco.cc/fronts/ai-computation/)
- terms: [Real-world evidence](https://onco.cc/terms/real-world-evidence/)
- technologies: [Geriatric assessment](https://onco.cc/technologies/geriatric-assessment/)
- bottlenecks: [Older and multimorbid patients are excluded and undertreated](https://onco.cc/bottlenecks/b-aging-comorbidity/), [Trials do not represent the people who get cancer](https://onco.cc/bottlenecks/b-trial-diversity/), [Weak real-world evidence and registries](https://onco.cc/bottlenecks/b-real-world-evidence/)
- key papers: [Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval](https://onco.cc/key-papers/paper-gyawali-jama-intern-med/)

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