Power trials to detect a benefit patients would value, not the smallest detectable one
A trial can be designed to detect a tiny improvement that is statistically real but too small to matter. Protocols should state up front what size of benefit would be worth having, and be built to detect that.
Protocols pre-declare a minimal clinically important benefit using the ESMO-MCBS or ASCO Value Framework thresholds (e.g., a hazard ratio and absolute gain in median OS or PFS), justify the sample size against it, and report whether the observed effect met it. Regulators and HTA bodies use the pre-declared threshold in review, discouraging trials sized to detect trivial differences.
- 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.
- Prices and value · New cancer drugs routinely cost over $150,000 a year, often for months of benefit. Systems cannot afford them and patients go bankrupt.
Pages like this
not linked directly; found by shared links- IdeaWin-ratio endpoints that weigh survival, toxicity and quality of life together
Shares Nathan Cherny, Prasad: most surrogate endpoints in cancer trials correlate poorly with survival, European Society for Medical Oncology (ESMO), Overall survival (OS).
- IdeaPre-specified crossover-adjusted survival in every trial that allows crossover
Shares Hazard ratio (HR), European Society for Medical Oncology (ESMO), Overall survival (OS), Trial design, endpoints and cost.
- IdeaAn independent programme that validates surrogate endpoints, setting by setting
Shares Prasad: most surrogate endpoints in cancer trials correlate poorly with survival, European Society for Medical Oncology (ESMO), American Society of Clinical Oncology (ASCO), Overall survival (OS).
- IdeaLaunch prices indexed to the ESMO benefit scale, revisited when survival matures
Shares European Society for Medical Oncology (ESMO), American Society of Clinical Oncology (ASCO), Overall survival (OS), Progression-free survival (PFS).
- IdeaAgree in advance how to borrow evidence between similar rare cancers
Shares Hazard ratio (HR), European Society for Medical Oncology (ESMO), American Society of Clinical Oncology (ASCO), Trial design, endpoints and cost.
- IdeaCap public prices for new cancer drugs to tiers of the ESMO and ASCO value scales
Shares European Society for Medical Oncology (ESMO), American Society of Clinical Oncology (ASCO), Prices and value.
- IdeaSeamless phase 2/3 with pre-registered go rules as the default for new agents
Shares Overall survival (OS), Progression-free survival (PFS), Trial design, endpoints and cost.
- PersonMichael LeBlanc
Shares Hazard ratio (HR), Overall survival (OS), Progression-free survival (PFS).