Test the drug in biomarker-negative patients too, so the biomarker can be validated
Trials that only enrol patients with a positive biomarker can never prove the biomarker matters. Including a smaller biomarker-negative group would show whether the test is really needed.
Enrichment designs enrol only biomarker-positive patients and cannot estimate the interaction between marker and treatment. Marker-stratified designs (all-comers with stratified randomisation and pre-specified interaction tests) are the accepted way to validate a predictive biomarker but are rarely used because they cost more. Regulators could require a marker-negative cohort (even under-powered but pooled across trials) whenever a biomarker is proposed to restrict a label, so the restriction is evidence-based.
- Biomarkers are not validated or standardised · Tests that decide who gets a drug are often not validated prospectively and are measured differently in every lab.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
Pages like this
not linked directly; found by shared links- IdeaA reliance pathway for companion diagnostics so the test arrives with the drug
Shares Companion diagnostic, Companion diagnostics, Biomarkers are not validated or standardised.
- IdeaDrug labels must state which biomarker assays were validated and how they compare
Shares Companion diagnostic, Companion diagnostics, Biomarkers are not validated or standardised.
- IdeaRegulators recognise each other's companion diagnostic approvals
Shares Companion diagnostic, Companion diagnostics, Biomarkers are not validated or standardised.
- TrialDESTINY-Breast06
Shares Randomised trials to test whether biomarker-negative patients really do not benefit, HER2-low and HER2-ultralow, Biomarkers are not validated or standardised.
- IdeaPublish the disagreement between trial doctors and independent reviewers for every trial
Shares Hazard ratio (HR), Biomarkers are not validated or standardised, Trial design, endpoints and cost.
- IdeaA fund for prospective validation of academic biomarkers and companion diagnostics
Shares Companion diagnostic, Companion diagnostics, Biomarkers are not validated or standardised.
- IdeaBayesian shrinkage for subgroup claims to stop false 'works in this group' stories
- IdeaPay for new biomarker tests only while evidence of clinical utility is being collected
Shares Companion diagnostic, Biomarkers are not validated or standardised.