Win-ratio endpoints that weigh survival, toxicity and quality of life together
Every patient on the new drug is compared with every patient on the old one: who lived longer, and if equal, who had fewer serious side effects, and if still equal, who felt better. The share of 'wins' becomes the result.
Net benefit endpoints (win ratio, generalised pairwise comparisons, desirability of outcome ranking) rank prioritised outcomes hierarchically across all patient pairs. Cardiology adopted the win ratio for composite endpoints; oncology has used it only in exploratory analyses. The proposal is a pre-specified net-benefit endpoint with a published hierarchy (OS, then grade 3+ toxicity, then QoL deterioration) as a co-primary or key secondary in phase 3.
- 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.
- Toxicity and quality of life are undervalued · Trials measure how long people live, not how they live. Side-effects are under-reported and under-treated.
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not linked directly; found by shared links- IdeaPower trials to detect a benefit patients would value, not the smallest detectable one
Shares Nathan Cherny, Prasad: most surrogate endpoints in cancer trials correlate poorly with survival, European Society for Medical Oncology (ESMO), Overall survival (OS).
- IdeaTolerability as a co-primary endpoint with its own label claim
Shares Immune-related adverse events (irAEs), Progression-free survival (PFS), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaReport time toxicity, the days spent in healthcare, as a standard outcome for older patients
Shares Overall survival (OS), Progression-free survival (PFS), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- IdeaTime to treatment failure and quality of life as co-primary endpoints in non-curative trials
Shares Overall survival (OS), Progression-free survival (PFS), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.
- 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), Overall survival (OS), Progression-free survival (PFS).
- 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.
- IdeaPre-specified crossover-adjusted survival in every trial that allows crossover
Shares European Society for Medical Oncology (ESMO), Overall survival (OS), Trial design, endpoints and cost.
- IdeaDefine tolerability endpoints as rigorously as efficacy endpoints
Shares Immune-related adverse events (irAEs), Trial design, endpoints and cost, Toxicity and quality of life are undervalued.