Mandatory real-world reporting for patients excluded from pivotal trials
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.
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.
- Weak real-world evidence and registries · We do not reliably know what happens to patients after approval, so we cannot tell which drugs deliver in practice.
- Older and multimorbid patients are excluded and undertreated · Most people with cancer are over 65 but most trial patients are younger and fitter. We guess how to treat the majority.
- Trials do not represent the people who get cancer · Older, Black, Hispanic, Asian, rural, poor and multimorbid patients are under-represented, so results may not apply to them.
Pages like this
not linked directly; found by shared links- IdeaParallel real-world cohorts for sicker patients alongside every pivotal trial
Shares Geriatric assessment, Older and multimorbid patients are excluded and undertreated, Trials do not represent the people who get cancer.
- IdeaA mandatory over-70s cohort with geriatric assessment in every pivotal trial
Shares Geriatric assessment, Older and multimorbid patients are excluded and undertreated, Trials do not represent the people who get cancer.
- IdeaRequire post-approval evidence in patients over 75 and update labels accordingly
Shares Older and multimorbid patients are excluded and undertreated, Weak real-world evidence and registries, Real-world evidence.
- IdeaPost-marketing safety monitoring stratified by ancestry and sex, with label updates
Shares Trials do not represent the people who get cancer, Weak real-world evidence and registries, Real-world evidence.
- IdeaAfter approval, a pragmatic trial in the patients the pivotal trial excluded
Shares Older and multimorbid patients are excluded and undertreated, Weak real-world evidence and registries, Real-world evidence.
- IdeaGeriatric assessment by default for every older patient, and trials that admit them
Shares Geriatric assessment, Older and multimorbid patients are excluded and undertreated, Trials do not represent the people who get cancer.
- IdeaGeriatrician co-management for older patients having cancer surgery
Shares Geriatric assessment, Older and multimorbid patients are excluded and undertreated.
- IdeaStop screening by life expectancy, not birthday, with a tool in the record
Shares Geriatric assessment, Older and multimorbid patients are excluded and undertreated.